Page 21234..1020..»

Brain chemical lost in Parkinson’s may contribute to its own demise – Science News Magazine

Posted: September 7, 2017 at 5:47 pm

The brain chemical missing in Parkinsons disease may have a hand in its own death. Dopamine, the neurotransmitter that helps keep body movements fluid, can kick off a toxic chain reaction that ultimately kills the nerve cells that make it, a new study suggests.

By studying lab dishes of human nerve cells, or neurons, derived from Parkinsons patients, researchers found that a harmful form of dopamine can inflict damage on cells in multiple ways. The result, published online September 7 in Science, brings multiple pieces of the puzzle together, says neuroscientist Teresa Hastings of the University of Pittsburgh School of Medicine.

The finding also hints at a potential treatment for the estimated 10 million people worldwide with Parkinsons: Less cellular damage occurred when some of the neurons were treated early on with antioxidants, molecules that can scoop up harmful chemicals inside cells.

Study coauthor Dimitri Krainc, a neurologist and neuroscientist at Northwestern University Feinberg School of Medicine in Chicago, and colleagues took skin biopsies from healthy people and people with one of two types of Parkinsons disease, inherited or spontaneously arising. The researchers then coaxed these skin cells into becoming dopamine-producing neurons. These cells were similar to those found in the substantia nigra, the movement-related region of the brain that degenerates in Parkinsons.

After neurons carrying a mutation that causes the inherited form of Parkinsons had grown in a dish for 70 days, the researchers noticed some worrisome changes in the cells mitochondria. Levels of a harmful form of dopamine known as oxidized dopamine began rising in these energy-producing organelles, reaching high levels by day 150. Neurons derived from people with the more common, sporadic form of Parkinsons showed a similar increase but later, beginning at day 150. Cells derived from healthy people didnt accumulate oxidized dopamine.

This dangerous form of dopamine seemed to kick off other types of cellular trouble. Defects in the cells lysosomes, cellular cleanup machines, soon followed. So did the accumulation of a protein called alpha-synuclein, which is known to play a big role in Parkinsons disease.

Those findings are direct experimental evidence from human cells that the very chemical lost in Parkinsons disease contributes to its own demise, says analytical neurochemist Dominic Hare, of the University of Technology Sydney. Because these cells churn out dopamine, they are more susceptible to dopamines potential destructive forces, he says.

When researchers treated neurons carrying a mutation that causes inherited Parkinsons with several different types of antioxidants, the damage was lessened. To work in people, antioxidants would need to cross the blood-brain barrier, a difficult task, and reach the mitochondria in the brain. And this would need to happen early, probably even before symptoms appear, Krainc says.

Without this human model, we would not have been able to untangle the pathway, Krainc says. In dishes of mouse neurons with Parkinsons-related mutations, dopamine didnt kick off the same toxic cascade, a difference that might be due to human neurons containing more dopamine than mice neurons. Dopamine-producing neurons in mice and people have some very fundamental differences, Krainc says. And those differences might help explain why discoveries in mice havent translated to treatments for people with Parkinsons, he says.

Over the past few decades, scientists have been accumulating evidence that oxidized dopamine can contribute to Parkinsons disease, Hastings says. Given that knowledge, the new results are expected, she says, but still welcome confirmation of the idea.

These toxic cellular events occurred in lab dishes, not actual brains. Cell cultures arent the perfect re-creation of whats going on in the human brain, Hare cautions. But these types of experiments are the next best thing for monitoring the chemical changes in these neurons, he says.

See the rest here:
Brain chemical lost in Parkinson’s may contribute to its own demise – Science News Magazine

Posted in Parkinson's Treatment | Comments Off on Brain chemical lost in Parkinson’s may contribute to its own demise – Science News Magazine

Sumitomo Dainippon Seeks Japanese Approval for Trerief to Treat Parkinsonism in Dementia Patients – Parkinson’s News Today

Posted: at 5:47 pm

Sumitomo Dainippon Pharma has asked Japanese authorities to approve itsTrerief (zonisamide) as a new therapy for parkinsonismin dementia patients with Lewy bodies, the company announced in a press release.

Trerief went on sale in Japan in March 2009 as a treatment for Parkinsonspatients who saw insufficient results with levodopa and other Parkinsons-specific drugs. After a 2013 expansion of its originalapproval, Trerief is now accepted as a treatment option inJapan, where a2014 Patient Surveyreported some144,000 patients suffering from vascular dementia and unspecified dementia,including dementia with Lewes bodies (DLB).

Parkinsonism is a general term used to describe a group of neurological disorders that cause movement problems similar to those observed in Parkinsons disease, like tremors, slow movement and stiffness.

The DLB form of dementia causes progressive cognitive impairment. Parkinsonism is one of the four core features of DLB, alongside fluctuating cognition, recurrent visual hallucinations and rapid eye movement (REM) sleep behavior disorder.

DLB is classified as part of the Lewy body disease spectrum, which also includes Parkinsons disease. Since symptoms of parkinsonism virtually mimic those of Parkinsons disease, Sumitomo Dainippon is now seeking approval for Trerief as another therapeutic option for treating parkinsonism in DLB, under the assumption that its action will be equally effective.

If approved, Trerief will be the worlds first drug to treatparkinsonism in DLB. Sumitomo Sainipponbased its application on data from a Phase 3, multicenter, placebo-controlled, randomized, double-blind, 12-week study of Trerief in patients with parkinsonism accompanying DLB. Topline results were disclosed in early April.

The study evaluated the efficacy and safety of Trerief in 351 patients randomized to receive the active compound at 25 mg/daily, 50 mg/daily, or placebo.

Using the Unified Parkinsons Disease Rating Scale a rating scale thatmeasures disability and impairment in Parkinsons and also the primary outcome measure in most clinical trials of Parkinsons therapeutics the primary endpoint of the study at 12 weeks was significantly higherin the groups receiving Trerief, compared to placebo.

According to the April report of topline results, the incidence of treatment emergent adverse effects was no different than those previously reported. Incidence at 25 mg/daily was 48.7 percent, rising to 54.5 percent at 50 mg/daily, while in the placebo group, it was 47.1 percent.

Originally posted here:
Sumitomo Dainippon Seeks Japanese Approval for Trerief to Treat Parkinsonism in Dementia Patients – Parkinson’s News Today

Posted in Parkinson's Treatment | Comments Off on Sumitomo Dainippon Seeks Japanese Approval for Trerief to Treat Parkinsonism in Dementia Patients – Parkinson’s News Today

Spreading depolarizations trigger early brain injury after subarachnoid hemorrhage, researchers find – Medical Xpress

Posted: September 6, 2017 at 5:45 pm

The study of Hartings and colleagues found that bleeding onto the surface of the brain (a subarachnoid clot) can directly cause the death (infarct) of the affected brain gray matter the cerebral cortex. Progression to death is mediated by repetitive brain tsunamis (cortical spreading depolarizations), shown here by blue waves and arrows, that spread through the cortex. Brain tsunamis reduce blood supply to the brain (cortical spreading ischemia) and impair brain function, resulting in flatline (terminal depolarization) as the tissue dies. Brain tsunamis were first described in 1944 by Aristides Leo and today are measured in patients with electrodes placed on the brain surface. Credit: Illustration by Tonya Hines, Mayfield Clinic

The phrase “time is brain” could take on new meaning when applied to the treatment of subarachnoid hemorrhage, a type of bleeding stroke, thanks to research partially funded by the Mayfield Education & Research Foundation and the United States government.

Subarachnoid hemorrhage, caused by the rupture of a brain aneurysm, affects an estimated 10 to 15 of every 100,000 individuals each year. An international team of researchers has established that the blood from this type of hemorrhage launches deadly “brain tsunamis” within hours, leading to permanent brain damage. This damage, known as infarction, occurs in the cerebral cortex near the hemorrhage and can cause permanent disability and in some cases death.

Brain tsunamis, scientifically known as cortical spreading depolarizations, are travelling waves of brain dysfunction that spread out from an injury site and contribute to worse outcomes in patients. They affect patients who suffer trauma to the brain as well as those who suffer various types of stroke. In subarachnoid hemorrhage, blood from a ruptured aneurysm or shredded artery pools in the space between the arachnoid membrane and the brain itself.

A team of researchers led by Principal Investigator Jed Hartings, PhD, Research Associate Professor in the Department of Neurosurgery at the University of Cincinnati, used a novel animal model to study what happens immediately after a subarachnoid hemorrhage. The team also monitored 23 patients who were surgically treated for subarachnoid hemorrhage at hospital locations of Charit University Medicine in Berlin. Monitoring was accomplished by placing electrode strips on the surface of the brains of animal models as well as patients.

The team’s findings of secondary damage within 6 hours were published online today in the journal Brain.

“We found that patients who suffered brain damage in their frontal lobes were more likely to have experienced spreading depolarizations than those who had no damage,” Dr. Hartings says. “The animal studies showed that these pathological changes can arise as a direct result of the blood accumulation in the grooves of the brain that the presence of blood in the wrong place is toxic to the brain.”

In the past, researchers have focused primarily on delayed complicationssuch as the narrowing of major cerebral arteries known as vasospasm that occur 5 to 14 days after subarachnoid hemorrhage. Those complications account for only a minority of deaths (13 percent), however; the majority of deaths from subarachnoid hemorrhage (86 percent) occur in the early hours and days after an aneurysm rupture occurs. The present study sheds new light on these early events and suggests that they may be preventable.

“This is the first evidence that brain tsunamis are a clinical marker, and mechanism, of early brain injury,” Dr. Hartings says. “As such, they provide us with an opportunity to pursue therapeutic interventions that could improve outcomes for patients. By treating or preventing brain tsunamis, we could potentially stop many victims of bleeding stroke from suffering additional, often catastrophic, brain damage.”

The early damage caused by subarachnoid hemorrhage has been underappreciated, Hartings says, because it often cannot be observed with routine CT brain imaging. “There has also been a sense that early damage could not be prevented, but perhaps that perception is starting to change.”

Looking ahead, the researchers propose the investigation in animal models of therapies that target spreading depolarizations in an effort to interrupt or prevent secondary injury processes.

When a brain injury occurs, nerve cells in the brain (which act like batteries by storing electrical and chemical energy) malfunction and effectively short-circuit. Because all nerve cells in the brain are connected, this depolarization causes all the neighboring cells to short-circuit as well; this subsequent leakage of precious electrical charge moves like a tsunami through the brain, with the potential to cause additional permanent tissue damage.

To document the cascade of events that immediately follows subarachnoid hemorrhage, the research team used a novel swine model, whose brain, with grooves and fissures, more closely resembles the human brain than previously studied rodent models. The researchers found that clots that quickly formed in the grooves of the swine brain mirrored the results in the human brain; in both situations, the clots caused repetitive spreading depolarizations and lesions (infarcts) in the cerebral cortex shortly after an aneurysm rupture and subarachnoid hemorrhage.

The study’s collaborating scientists and clinicians are members of COSBID (Co-Operative Studies on Brain Injury Depolarizations.

Explore further: Subarachnoid hemorrhage and the need for expert treatment

More information: Ayata C, Lauritzen M. Spreading Depression, Spreading Depolarizations, and the CerebralVasculature. Physiological Reviews. 2015;95(3):953-93.

Baechli H, Behzad M, Schreckenberger M, Buchholz HG, Heimann A, Kempski O, et al. Blood constituents trigger brain swelling, tissue death, and reduction of glucose metabolism early after acute subdural hematoma in rats. J Cereb Blood Flow Metab. 2010;30(3):576-85.

Bosche B, Graf R, Ernestus RI, Dohmen C, Reithmeier T, Brinker G, et al. Recurrent spreading depolarizations after subarachnoid hemorrhage decreases oxygen availability in human cerebral cortex. Ann Neurol. 2010;67(5):607-17.

Bretz JS, von Dincklage F, Woitzik J, Winkler MKL, Major S, Dreier JP, et al. The Hijdra scale has a significant prognostic value for the functional outcome of Fisher grade 3 patients with subarachnoid hemorrhage. Clin Neuroradiol. 2016:in press

Hartings JA. Spreading depolarization monitoring in neurocritical care of acute brain injury. Curr Opin Crit Care. 2017;23(2):94-102.

Hartings JA, Shuttleworth CW, Kirov SA, Ayata C, Hinzman JM, Foreman B, et al. Thecontinuum of spreading depolarizations in acute cortical lesion development: Examining Leao’s legacy. J Cereb Blood Flow Metab. 2017;37(5):1571-94.

Excerpt from:
Spreading depolarizations trigger early brain injury after subarachnoid hemorrhage, researchers find – Medical Xpress

Posted in Brain Injury Treatment | Comments Off on Spreading depolarizations trigger early brain injury after subarachnoid hemorrhage, researchers find – Medical Xpress

Carlson offers veteran a new start after brain injury – Minnesota Daily

Posted: at 5:45 pm

On March 19, 2012, Brian Grundtner fell 50 feet during an airborne training jump, sustaining multiple broken bones and a traumatic brain injury.

Four years later, the former special operations staff sergeant overcame his TBI to graduate from the Carlson School of Management with his Masters of Business Administration.

Grundtner, a native of White Bear Lake, Minnesota, said he chose the University for its military veterans MBA program.

I think the biggest factor for me was the veterans initiative and the emphasis on integrating veterans into the whole MBA program, Grundtner said. It really helped with the transition out of the military and into the civilian world.

Retired naval officer Charles Altman, the MBA military veterans program director, said financial assistance make the University’s one of the best programs for veterans.

Twenty percent of Carlsons full-time MBA students are veterans and all of them get scholarships, Altman said. Those who complete the program have a 100 percent employment rate after graduation.

In a lot of cases what veterans need more than anything else is a leg up this funding provides them that, Altman said, adding he spent time getting to know Grundtner while he was a student.

Dave Hopkins, a professor in Carlson, said Grundtner stood out in his classes as a personable student and hard worker.

I knew that he had some challenges in overcoming some injuries, but it didnt impact him with me in any way, Hopkins said. He was a great asset to the program.

Veterans, especially those struggling with trauma and injuries, often struggle to return and translate their military and leadership skills to civilian life, Altman said.

Grundtners accident was the start of a long recovery process.

I was knocked out after I landed. When I woke up, my eyes didnt work right away, he said.

Though he didnt immediately realize the extent of the brain injury, he said he began to notice difficulties with everyday tasks.

I would be reading a book and the words would all just turn into a bunch of letters, Grundtner said. Or if I was sitting in front of a computer screen for more than 30 minutes I would just get an immense headache.

He received treatment for over a year at a concussion care clinic at Fort Bragg in North Carolina, where he focused on vision therapy and concentration. As a result, he said he hasnt had a migraine since June 2013.

Grundtner now works in software sales at IBM and spreads the word about TBIs with the Defense and Veterans Brain Injury Center’s initiative A Head for the Future.

Hopefully my participation in this program can connect me with other TBI victims or concussion victims, Grundtner said, adding he wants to encourage veterans to pursue recovery and careers.

Read the original here:
Carlson offers veteran a new start after brain injury – Minnesota Daily

Posted in Brain Injury Treatment | Comments Off on Carlson offers veteran a new start after brain injury – Minnesota Daily

This Is Why You Want to Steer Clear of Antidepressants If You Have Alzheimer’s – Reader’s Digest

Posted: at 5:45 pm

Photographee.eu/ShutterstockSome of the early symptoms of Alzheimers Disease are the same as those of depression, including apathy and irritability. As a result, many patients with Alzheimers Disease (AD) are prescribed antidepressant medications. A group of scientists from the University of Eastern Finland were concerned that this practice could be increasing the risk of head and brain injuries among AD patients. Heres why:

First, AD is a disease associated primarily with the elderly. Second, antidepressant use among the elderly is associated with an increased risk of falling down. Finally, falling down is the number one cause of head and brain injuries in the elderly (compare this to younger people, who more often experience head injuries as a result of motor vehicle, sports, and other accidents).

Since there were no existing studies investigating the risk of head or brain injuries associated with antidepressant use, let alone studies that focused on the AD patients or even the elderly, the Finnish scientists took it upon themselves to investigate whether antidepressant use is associated not only with an increased risk of falling, but also with an increased risk of head and brain injury in Alzheimers patients. What they found is that the use of antidepressants by AD patients is, in fact, associated with a higher risk of head injuries.

Their study, the results of which were recently published in the journal, Alzheimers Research & Therapy, included 32,700 AD patients living in nursing homes, 10,910 of whom used antidepressants and 21,820 of whom did not. Each of the people who used antidepressant medication was matched with two users who did not, based on age, gender, and time since AD-diagnosis. Head and brain injuries were recorded, numbers were crunched, and the association became clear: the risk of head injury goes up immediately upon starting the medication and continues to be elevated for up to two years.

Antidepressant use has been previously associated with an increased risk of falls, the study authors report, but our novel findings indicate that they are associated with severe injurious falls, i.e., those resulting in head or brain injuries among persons with Alzheimers disease.

Future studies will be required to confirm the association between antidepressant use and head and brain injuries, and the study authors would like to conduct further research to determine whether the risk of head injury increases in all elderly people who use antidepressant medications, as opposed to just elderly AD patients.

For now, the study authors recommend that the use of antidepressants be carefully considered in those with AD, and in the elderly in general. Instead, practitioners may prescribe anti-psychotics, which have been the traditional treatment of choice for the behavioral and psychological symptoms of dementia. The use of anti-psychotics comes with its own set of risks, including life-threatening brain events such as stroke and hemorrhage. However, knowing the risks of the available treatments for AD will allow for more-informed prescribing.

Even if you dont have AD, your meds might be causing you problems. For example, here are some warning signs youre taking too many prescription drugs.

Read this article:
This Is Why You Want to Steer Clear of Antidepressants If You Have Alzheimer’s – Reader’s Digest

Posted in Brain Injury Treatment | Comments Off on This Is Why You Want to Steer Clear of Antidepressants If You Have Alzheimer’s – Reader’s Digest

How to avoid, recognize and treat concussion in sports – National Post

Posted: at 5:45 pm

This article was originally published on The Conversation, an independent and nonprofit source of news, analysis and commentary from academic experts. Disclosure information is available on the original site.

Author: Kathryn Schneider, Assistant Professor, Clinician Scientist (Physiotherapist), Faculty of Kinesiology, University of Calgary

Recognize, remove, rest and recover before returning to sport. Those are key points about sport-related concussion stated in the 5th International Consensus Statement on Concussion in Sport.

The Canadian Guidelines on Concussion in Sport have been developed based on this statement and were recently released by Parachute Canada, a charitable organization focused on injury prevention, and its expert advisory group.

The good news is that most people who suffer a concussion will recover in the initial days and weeks following injury. However, some will have ongoing symptoms. While concussions may occur in sport, there are many benefits to physical activity and sport participation for both youth and adults.

I am a physiotherapist and researcher (assistant professor and clinician scientist) at the Sport Injury Prevention Research Centre, Faculty of Kinesiology, University of Calgary. My research focuses on the prevention and treatment of sport-related concussion in children, youth and adults, with special emphasis on the role of the neck and balance systems.

A concussion is a type of a brain injury that occurs following a trauma to the head or body. Symptoms can come on immediately or may take hours to gradually evolve. The most common symptom following concussion is a headache. However, a number of other symptoms such as dizziness, nausea, fatigue, difficulty with concentration, neck pain and other complaints may also occur. Lying motionless, clutching the head, being slow to get up, wobbling and appearing dazed are some of the observable signs.

In the event that a concussion may have occurred, it is important that the player is removed from the activity and has follow-up medical evaluation as soon as possible. A tool called the Concussion Recognition Tool 5 (CRT5) has been developed by the Concussion in Sport Group and is meant to help coaches, officials, parents and players recognize the signs of concussion.

How are concussions treated?

All individuals with a suspected concussion should see a health care professional with knowledge of concussion. The typical treatment is a short period of rest (24 to 48 hours, both mental and physical) followed by a gradual return to the typical activities that are done throughout the day.

Following this, individuals may slowly increase their activity level using a graduated return-to-school strategy and return-to-sport strategy.

The return-to-school strategy includes steps to gradually increase the amount of mental activity prior to returning to school.

The return-to-sport strategy includes a series of steps that are performed sequentially, with each step taking a minimum of 24 hours. These include light aerobic exercise, sport-specific exercises, non-contact training drills and full practice prior to returning to play or sport.

These two strategies can be performed simultaneously under the supervision of a health care professional. They take approximately one week to complete as long as the person with the concussion does not have any recurrence of symptoms. It is recommended that individuals return to school prior to returning to sport. Medical clearance should be obtained prior to returning to sport.

Most people get better in the initial days to weeks following a concussion. However, for those who have ongoing symptoms and difficulties, there are some treatments that have been shown to help.

My research has found that people with ongoing headaches, neck pain and/or dizziness and balance problems who are treated with physiotherapy techniques aimed at treating the neck and balance systems are more likely to be medically cleared to return to sport in eight weeks.

There is also some research that low-level aerobic exercise may be beneficial following a concussion. Our research in this area is ongoing and we currently have a number of studies that are evaluating different types of treatments to help with recovery.

Is there any way to prevent a concussion?

The best way to minimize the impact of concussions is to prevent them. To do this, we first need to understand who is at the greatest risk.

Research has shown that individuals who have had a previous concussion, who play games (as opposed to practice) and engage in contact sports all increase the risk of concussion.

Many different sport associations are working to use research to inform rule changes to decrease the risk of concussion, including rules to disallow bodychecking in youth ice hockey nationally, in leagues for 11-to-12-year-olds (based on research led by Carolyn Emery).

Another focus of my research program is to evaluate different pre-training strategies that may be used to prevent concussions.

Concussions in sport are an area of much discussion. It is imperative that individuals with a suspected concussion are removed from play until they can be medically evaluated and do not return until they are cleared to do so.

At this time, the literature on the long-term consequences of exposure to head trauma is inconsistent. Thus, more research is required to answer these important questions.

The good news is that most people recover well following a concussion when managed appropriately. Awareness of the signs and symptoms of concussion, and appropriate initial management when a concussion may have occurred, are of utmost importance.

This article was originally published on The Conversation. Disclosure information is available on the original site. Read the original article:

https://theconversation.com/how-to-avoid-recognize-and-treat-concuss https://theconversa

More here:
How to avoid, recognize and treat concussion in sports – National Post

Posted in Brain Injury Treatment | Comments Off on How to avoid, recognize and treat concussion in sports – National Post

New AstraZeneca, Amgen Biotech Drug Offers Broad Asthma Relief – New York Times

Posted: at 5:45 pm

Injections for severe asthma have opened up a multibillion-dollar market as competing drugmakers have raced to develop antibody-based medicines for the 15 percent or more of patients who do poorly even on the latest inhalers.

Despite treatment advances in recent decades, their asthma is still not well controlled by standard therapy, which consists of inhaled steroids and drugs to open the airways.

Nucala and Teva’s Cinqair are two recently approved new injectable drugs and AstraZeneca’s benralizumab is likely to join them soon, since it is awaiting approval in the fourth quarter of this year.

Sanofi’s Dupixent, already approved for severe eczema, is a bit further behind but is widely seen as a strong contender.

However, all these new medicines only appear to help people with certain types of severe asthma, by targeting specific inflammatory chemicals made in the body that drive asthma, making them suitable for subgroups of patients.

Tezepelumab is different because it acts further upstream in the inflammatory cascade responsible for asthma by blocking the action of a cell-signalling protein called thymic stromal lymphopoietin (TSLP).

That means it can help a wider range of patients and could be a “game-changer”, according Tom Keith-Roach, head of AstraZeneca’s respiratory, inflammation and autoimmune business.

Biotech drugs for severe asthma are already worth $2 billion (1.53 billion pounds) in annual sales and Keith-Roach believes there is significant scope for growth since currently only about 10 percent of patients who might benefit are getting them.

Tezepelumab, like Dupixent, is also being developed for eczema.

The results of the Phase IIb asthma study, which were published online by the New England Journal of Medicine, will also be presented at the European Respiratory Society annual meeting in Milan next week.

(Reporting by Ben Hirschler; Editing by Mark Potter)

Go here to see the original:
New AstraZeneca, Amgen Biotech Drug Offers Broad Asthma Relief – New York Times

Posted in Eczema | Comments Off on New AstraZeneca, Amgen Biotech Drug Offers Broad Asthma Relief – New York Times

Shares of Sarepta Therapeutics soar on positive drug study results – CNBC

Posted: at 5:44 pm

Shares of Sarepta Therapeutics soared 12 percent in early trading Wednesday after the biopharmaceutical company reported positive results from a clinical trial of an experimental medicine for Duchenne muscular dystrophy.

The drug, golodirsen, would be Sarepta’s second to treat the rare, genetic disease, which causes muscle wasting and can be fatal before patients turn 30. Sarepta focuses on the discovery and development of precision genetic medicines to treat rare neuromuscular diseases.

The new study, conducted in Europe, involved 25 boys with confirmed deletions of the DMD gene amenable to skipping exon 53. Exons are part of the DNA code. The treatment targets a genetic mutation affecting about 8 percent of patients with DMD.

Sarepta’s first drug for DMD, Exondys 51 approved on a conditional basis by the FDA last year pending more testing to confirm results treats a mutation affecting about 13 percent. Exondys 51 costs about $300,000 per year.

“Our goal is to treat 100 percent” of DMD suffers, Sarepta CEO Doug Ingram told CNBC’s “Squawk Box.” “The data that we have this morning shows we’re on the right path.”

The results, announced before Wall Street’s open bell, showed that golodirsen increased production of the protein dystrophin to 1.02 percent of normal levels from about 0.095 percent without the drug. Analysts said those results were higher than expected, but scientists wonder whether that’s enough to increase muscle strength and have a clinical benefit.

According to Sarepta, the underlying cause of DMD is a mutation in the gene for dystrophin, which is an essential protein involved in muscle fiber function. DMD occurs in one in every 3,500 to 5,000 males worldwide. Symptoms usually start in early childhood, usually between 3- and 5-years old. It primarily affects boys. But in rare cases can affect girls.

“Sarepta is a small company. We have already invested $1 billion fighting Duchenne muscular dystrophy. And we’re not done yet,” said Ingram, who was appointed as CEO in July. Ahead of Wednesday, Sarepta had a stock market value of $2.6 billion.

See original here:
Shares of Sarepta Therapeutics soar on positive drug study results – CNBC

Posted in Muscular Dystrophy Treatment | Comments Off on Shares of Sarepta Therapeutics soar on positive drug study results – CNBC

Early movers: XOM, SRPT, NAV, HDS, NWL, TRVG, GIII, HPE & more – CNBC

Posted: at 5:44 pm

Check out which companies are making headlines before the bell:

Exxon Mobil UBS upgraded the stock to “neutral” from “sell,” noting the underperformance of the stock this year and an improved macro outlook for the oil industry.

Sarepta Therapeutics The drugmaker announced positive study results for a new treatment for Duchenne Muscular Dystrophy.

Navistar The truck and engine maker earned 37 cents per share for its latest quarter, beating estimates by nine cents a share. Revenue also topped forecasts. The return to profitability for Navistar comes as it increases market share and profit margins.

HD Supply Holdings The industrial distributor matched forecasts with adjusted quarterly profit of 64 cents per share, with revenue very slightly above Street projections.

Newell Brands The consumer products maker cut its full-year forecast, due to the impact of Hurricane Harvey on its resin suppliers. Newell’s Rubbermaid division is among its substantial users of resins.

Trivago The travel website operator cut its full-year outlook, based on slower-than-expected revenue growth. Trivago said revenue per qualified referral a key metric fell too quickly to prevent overspending on ad purchases.

G-III Apparel The manufacturer of licensed apparel lost 18 cents per share for its latest quarter, eight cents a share less than analysts had anticipated. Revenue beat forecasts and G-III raised its full-year forecast. The company said strong brand names like Tommy Hilfiger and Calvin Klein are helping it overcome significant marketplace headwinds.

Hewlett Packard Enterprise The enterprise technology company reported adjusted quarterly profit of 30 cents per share, beating estimates by four cents a share. Revenue easily beat forecasts on the strength of improved networking equipment sales.

Dave & Buster’s Dave & Buster’s beat estimates by two cents a share, with adjusted quarterly profit of 59 cents per share. The restaurant chain’s revenue fell below forecasts and it also cut full-year comparable restaurant sales guidance.

Wal-Mart The retailer kicked off its holiday layaway plan, hoping to provide a boost to slow toy sales. Under the program, customers can put as little as $10 down to hold items with a minimum sales price of $50.

Novo Nordisk Novo Nordisk settled a Food and Drug Administration (FDA) investigation into its diabetes drug Victoza. The Danish drugmaker will pay about $58.7 million to resolve claims that it downplayed FDA-mandated warnings about the cancer risks for users of the treatment.

Royal Caribbean, Carnival, Norwegian Cruise Line Cruise line stocks are on watch once again after falling the past two sessions on concerns about the financial impact of Hurricanes Harvey and Irma. Also on hurricane watch: airline stocks, including American Airlines, United Continental, Delta Air Lines, and Southwest Airlines, among others.

MGM Resorts MGM announced a $1 billion stock buyback program, and also said it would sell the MGM National Harbor casino’s real estate to MGM Growth Properties for $1.19 billion. MGM Resorts will continue to operate that property.

Duluth Holdings Duluth reported quarterly profit of 13 cents per share, three cents a share above estimates. The casual wear company also saw revenue exceed forecasts. The parent of Duluth Trading saw a better than 30 percent improvement in net sales over a year earlier, as it opened more stores and attracted new customers.

Fiat Chrysler The automaker was upgraded to “overweight” from “equal-weight” at Barclays, as speculation continues about the possible sale of the Jeep division or the company as a whole.

Go here to read the rest:
Early movers: XOM, SRPT, NAV, HDS, NWL, TRVG, GIII, HPE & more – CNBC

Posted in Muscular Dystrophy Treatment | Comments Off on Early movers: XOM, SRPT, NAV, HDS, NWL, TRVG, GIII, HPE & more – CNBC

Mid-Afternoon Market Update: Crude Oil Up Over 1%; Sarepta Shares Jump After Positive Results In DMD Treatment … – Benzinga

Posted: at 5:44 pm

Toward the end of trading Wednesday, the Dow traded up 0.42 percent to 21,845.34 while the NASDAQ climbed 0.41 percent to 6,401.95. The S&P also rose, gaining 0.42 percent to 2,468.27.

Leading and Lagging Sectors

Wednesday afternoon, the energy shares surged 1.23 percent. Meanwhile, top gainers in the sector included Frontline Ltd. (NYSE: FRO), up 5 percent, and JA Solar Holdings Co., Ltd. (ADR) (NASDAQ: JASO), up 6 percent.

In trading on Wednesday, utilities shares fell 0.25 percent. Meanwhile, top losers in the sector included NRG Energy Inc (NYSE: NRG), down 3 percent, and Entergy Corporation (NYSE: ETR) down 1 percent.

Top Headline

G-III Apparel Group, Ltd. (NASDAQ: GIII) reported stronger-than-expected results for its second quarter and raised its FY18 forecast.

G-III Apparel reported a Q2 adjusted loss of $0.15 per share on revenue of $538 million. However, analysts were expecting a loss of $0.26 per share on sales of $522 million.

Equities Trading UP

Verastem Inc (NASDAQ: VSTM) shares shot up 30 percent to $5.00 after the company disclosed that its Phase 3 DUO study evaluating the efficacy and safety of duvelisib showed statistically significant improvement.

Shares of Voyager Therapeutics Inc (NASDAQ: VYGR) got a boost, shooting up 20 percent to $13.10 after the company announced ‘positive’ results from ongoing Phase 1b trial of VY-AADC01 for advanced Parkinson’s disease.

Sarepta Therapeutics Inc (NASDAQ: SRPT) shares were also up, gaining 16 percent to $47.52 following the announcement of positive results in its study for the treatment of Duchenne Muscular Dystrophy.

Equities Trading DOWN

Trivago NV – ADR (NASDAQ: TRVG) shares dropped 18 percent to $12.29 after the company lowered its guidance.

Shares of At Home Group Inc (NYSE: HOME) were down 10 percent to $23.19. At Home Group reported Q2 adjusted earnings of $0.18 per share on revenue of $232.1 million.

Dave & Buster’s Entertainment, Inc. (NASDAQ: PLAY) was down, falling around 10 percent to $52.38. Dave & Buster’s posted upbeat earnings for its second quarter, while sales missed expectations. The company also cut its comparable-store sales forecast for its fiscal year.

Commodities

In commodity news, oil traded up 1.07 percent to $49.18 while gold traded down 0.42 percent to $1,338.90.

Silver traded down 0.03 percent Wednesday to $17.935, while copper rose 0.83 percent to $3.154.

Eurozone

European shares closed mostly higher today. The eurozones STOXX 600 rose 0.06 percent, the Spanish Ibex Index declined 0.48 percent, while Italys FTSE MIB Index gained 0.35 percent. Meanwhile the German DAX climbed 0.75 percent, and the French CAC 40 rose 0.29 percent while U.K. shares fell 0.25 percent.

Economics

The MBAs index of mortgage application activity rose 3.3 percent for the latest week.

The U.S. trade deficit rose to $43.7 billion in July, compared to $43.5 billion in June. However, economists were expecting a $44.8 billion deficit. Imports declined 0.2 percent to $238.1 billion in July, while exports dropped 0.3 percent to $194.4 billion.

The Johnson Redbook Retail Sales Index rose 0.3 percent during the first week of September versus August.

The ISM non-manufacturing index climbed to 55.30 in August, versus prior reading of 53.90. Economists projected a reading of 55.40.

The Federal Open Market Committee released its latest Beige Book report.

Posted-In: Mid-Afternoon Market UpdateNews Eurozone Commodities Global Intraday Update Markets

2017 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.

Continue reading here:
Mid-Afternoon Market Update: Crude Oil Up Over 1%; Sarepta Shares Jump After Positive Results In DMD Treatment … – Benzinga

Posted in Muscular Dystrophy Treatment | Comments Off on Mid-Afternoon Market Update: Crude Oil Up Over 1%; Sarepta Shares Jump After Positive Results In DMD Treatment … – Benzinga

Page 21234..1020..»