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First Charcot-Marie-Tooth Patient Dosed in Phase 2 Clinical Trial of Muscle Impairment Therapy ACE-083 – Muscular Dystrophy News

Posted: Published on August 11th, 2017

The first patient has been dosed in a Phase 2 clinical trial of ACE-083as a treatment for the muscle impairment inCharcot-Marie-Tooth disease, according to the therapys developer,Acceleron Pharma. Acceleron is already evaluating ACE-083 in another Phase 2 trial this one as a treatment forfacioscapulohumeral muscular dystrophy. People diagnosed with CMT currently have no drug therapy options to address the major consequences of their disease, such as impaired walking and falls due to progressive muscle weakness in the lower leg, Dr. Colin Quinn, the new Phase 2 trials principal investigator,said in a press release. He is anassistant professor of clinical neurology at the University of Pennsylvanias Perelman School of Medicine. ACE-083 has the potential to increase muscle growth and strength in the lower leg muscles we are targeting, and could improve patients ability to walk, Quinn said. The therapy inhibits agents that impair muscle growth and strength. Acceleron is developing it for muscular dystrophies with what scientists call focal muscle loss. That kind of loss occurs in a muscle or group of muscles in a specific part of the body. A Phase 1 clinical trial (NCT02257489) showed that injecting ACE-083 into the legs of healthy volunteers increased their muscle volume. Researchers … Continue reading →

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Americans want a say in human genome editing, survey shows – Los Angeles Times

Posted: Published on August 11th, 2017

When it comes to CRISPR, our society has some important decisions to make. Just last week, scientists reported a new first in the journal Nature: They edited heritable cells in human embryos to treat an inherited form of heart disease. The day after the research was published, a group of genetics experts published a statement calling for further debate before applications of the technology are taken any further in humans. According to a new survey of 1,600 adults published in the journal Science today, much of the American public shares this desire for engagement in decision-making. Led by Dietram Scheufele, a professor of science communication at the University of Wisconsin - Madison, the study found that while support for gene editing applications varies, a majority of respondents think the public should be consulted before genome editing is used in humans. Gene editing presents the potential for remarkable benefits. The potential to cure genetic disease and to ensure the safety of the world's food supply in the face of climate change are perhaps the most exciting opportunities, said Jennifer Doudna, a chemist at UC Berkeley who was an early pioneer of the powerful gene-editing technique CRISPR-Cas9 and was not involved in … Continue reading →

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New test for screening of Duchenne muscular dystrophy in newborn babies – Medical Xpress

Posted: Published on August 11th, 2017

Researchers at Cardiff University and Cardiff and Vale University Health Board have developed a more reliable method of screening for Duchenne muscular dystrophy (DMD) in newborn babies. In collaboration with biotechnology company PerkinElmer, they have developed a diagnostic kit that can accurately screen for the disorder by analysing neonatal dried blood spots. Professor Ian Weeks, Dean of Clinical Innovation and Head of Cardiff University's School of Medicine, said: "The successful outcome of this study is an example of the impact that can be achieved through collaboration between academic, clinical and industry colleagues in developing improvements in human healthcare." The new method screens for DMD by detecting an enzyme that is released into the blood when muscle fibres are damaged from the condition. Previous tests of this type were less accurate as they also detected the activity of other forms of this enzyme, two of which are also produced in high levels due to muscle trauma-related injuries, which can lead to false results during screening. Dr Stuart J. Moat, Consultant Clinical Biochemist and Director of the Wales Newborn Screening Laboratory at the University Hospital of Wales, said: "We found that CK-MM can be reliably quantified in blood spots and believe that … Continue reading →

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Diabetes Medication Exenatide Shows Promise In Treating … – MedicalResearch.com (blog)

Posted: Published on August 11th, 2017

MedicalResearch.com Interview with:Dr Dilan Athauda MRCPSobell Department of Motor Neuroscience and Movement DisordersUCL Institute of Neurology & The National Hospital for Neurology and NeurosurgeryLondon MedicalResearch.com: What is the background for this study?Response: Exenatide is a synthetic version of a naturally occurring protein exendin-4 that was originally discovered by Dr John Eng in the early 1990s in the saliva of the Gila Monster, a venomous lizard native to the Southwestern United states. He and his team were looking for bio-active peptides in insect and lizard venom that could be useful for people with Type 2 diabetes. They discovered that exendin-4 was extremely similar to a human hormone called Glucagon-like peptide-1 (GLP-1). In humans, GLP-1 is secreted after you eat a meal to stimulate insulin secretion (and inhibit glucagon production) of which the end result is a lowering of blood sugar. Unfortunately human GLP-1 is rapidly broken down by a circulating enzyme called dipeptidyl peptidase IV (DPP-IV) and its effects only last minutes. Importantly, it was discovered that exendin-4 is naturally resistant to the actions of this enzyme, meaning its effects on blood sugar control lasts much longer in the body. These properties made it very attractive to people trying to treat … Continue reading →

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Company Spotlight: Adamas Pharma – Markets Insider

Posted: Published on August 11th, 2017

(RTTNews) - Shares of Adamas Pharmaceuticals Inc. (ADMS) have lost more than 9% of their value year to date while the iShares Nasdaq Biotechnology Index (ETF) (IBB) is up more than 14% during the same period. Adamas is focused on developing new medicines for chronic neurologic conditions like Parkinson's disease, multiple sclerosis, epilepsy and Alzheimer's disease. The Company's lead program is ADS-5102, a high-dose *Amantadine, taken once-daily at bedtime, in development for levodopa-induced dyskinesia (LID) in people with Parkinson's disease. *Amantadine is an antiviral medication used to prevent or treat certain influenza infections and is also prescribed to relieve symptoms of Parkinson's disease. The drug is also effective in relieving fatigue in multiple sclerosis. Parkinson's disease is a degenerative disorder of the central nervous system characterized by tremor, or shaking often in a hand or leg, and stiff muscles. It is estimated that over 1.5 million people in the U.S. have Parkinson's disease. Levodopa remains the gold standard to treat Parkinson's symptoms. A common and severe complication of the Levodopa therapy is dyskinesia, which refers to non-purposeful, fragmented or jerky motions. Approximately 200,000 Parkinson's disease patients suffer from Levodopa-induced dyskinesia, or LID. Adamas' drug candidate for levodopa-induced dyskinesia (LID) in … Continue reading →

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Therapy improves mobility for Parkinson’s disease patients – Victoria Advocate

Posted: Published on August 11th, 2017

Mary Blanton, 74, noticed years ago she was dragging her foot a little bit. "It just kept on getting worse," she said. Three years ago, she was diagnosed with Parkinson's disease by a Houston doctor. The neurodegenerative brain disorder progresses slowly in most people and, in her case, affects her gait and balance. So when her doctor recommended a specific type of therapy called LSVT BIG, she turned to a physical therapist she has trusted for the past 20 years or so. Mary Drost, 55, was working on a doctorate degree at the University of Texas Medical Branch at the time. Drost's professor recommended she become certified in the therapy for her last semester because it complemented her coursework. When Blanton learned Drost would be able to provide the specialized treatment locally, she was able to breathe a sigh of relief. Drost said with an aging population, this protocol will become more crucial. Laura Guse, chief clinical officer of LSVT BIG, said the program should be prescribed early in the course of Parkinson's disease. "Don't wait till you're falling over or having severe problems with your mobility or speech. Do something now," she said. Physical and occupational therapists can be … Continue reading →

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Music festival to help raise cash for youngsters’ treatment – Rotherham Advertiser

Posted: Published on August 11th, 2017

Festival committee members, (from left to right), Cllr Gill Shaw, Cllr Peter Blanksby and Cllr Wendy Fowkes with Daisy Garforth, dad, Lee and mum, Hollie. 171296-4 A TWO-day music festival will raise money to treat two young girls with cerebral palsy from the same village. The charity event on Wales Recreation Ground will help fund treatments for seven-year-old Daisy Garforth and Taylor Dobbs (3). A variety of music acts will be performing in the marquee between 6pm and 11.30pm on Friday and from noon until midnight on the Saturday. Cash raised will help fund therapy for both Kiveton Park girls, whose brain conditions were caused by complications at birth. Daisy was born with cord presentation and was starved of oxygen for about 20 minutes, resulting in brain damage which caused her athetoid dystonic cerebral palsy. Mum Hollie (32) said: It means her muscles can be very, very floppy or very, very tight and it affects all four limbs. Several of Daisys treatments are unavailable on the NHS, including the three times a week she has hyperbaric oxygen therapy in Leeds. Since Daisys been going for oxygen therapy in the last 18 months, she hasnt suffered a cold, chest infection or … Continue reading →

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Judge dismisses suit over disabled kids’ treatment under Fla. Medicaid system; case heads to appeal court – The Ledger

Posted: Published on August 11th, 2017

Federal judge dismisses suit against care provided by Florida's Medicaid system TALLAHASSEE A legal battle will continue about whether Florida's Medicaid program has provided proper services to children with severe medical conditions. Attorneys for children and the U.S. Department of Justice gave notice this week that they will take the five-year battle to the 11th U.S. Circuit Court of Appeals in Atlanta. The move came after a federal district judge in June sided with the state Agency for Health Care Administration and the state Department of Health, and dismissed the case. The case has centered on allegations the state violated the Americans with Disabilities Act and other laws by failing to provide services that would allow medically complex children to stay in their homes and communities. It was filed on behalf of children who had been placed in nursing homes or who were considered at risk of going into such facilities. Senior U.S. District Judge William Zloch in the June ruling approved the recommendations of a magistrate judge, who focused on changes the Medicaid program had made. U.S. Magistrate Judge Patrick Hunt wrote that the plaintiffs could no longer show they were in danger of being denied medically necessary private-duty … Continue reading →

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Ulcerative Colitis Investigational Therapy TD-1473 Shows Encouraging Results in Early Trial – IBD News Today

Posted: Published on August 11th, 2017

Early data from a Phase 1b clinical trial show that the investigational therapy TD-1473 for patients with ulcerative colitis (UC) has demonstrated targeted action and effectiveness, according to the therapys developer and study sponsor,Theravance Biopharma. Preliminary data are from four weeks of treatment. The ongoing study (NCT02818686), which currently is recruiting participants, is for patients with moderate to severe active UC. TD-1473 is an orally administered, highly specific inhibitor of the Janus kinase (JAK) family of enzymes, which plays a key role in inflammation. Most importantly, TD-1473s activity is restricted to the intestinal tract, which prevents the risk of systemic exposure and unwanted side effects. The study was designed to evaluatethe treatments safety, tolerability, and pharmacokinetics (a therapys behavior in the body) in UC patients during a 28-day period. Additional endpoints include assessing TD-1473s biologic effects by analyzing disease biomarkers and performing a thorough evaluation of clinical and endoscopic parameters. In the exploratory study, 10 patients from the first cohort received 80 mg of TD-1473 once a day for 28 days, while a control group of three patients received a placebo. Preliminary results from the first cohort revealed that TD-1473 induced a localized response (restricted to the intestine) with minimal … Continue reading →

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Paediatric ulcerative colitis drug gets orphan designation – Drug Target Review

Posted: Published on August 11th, 2017

The U.S. Food and Drug Administration has granted an orphan-drug designation to cobitolimod for the treatment of ulcerative colitis in pediatric patients The U.S. Food and Drug Administration (FDA) has granted an orphan-drug designation to cobitolimod for the treatment of ulcerative colitis in paediatric patients. We are pleased that the FDA has granted orphan-drug designation for cobitolimod for treatment of ulcerative colitis in children, which may provide seven years of market exclusivity in this indication on the US market, saidPeter Zerhouni, CEO of InDex Pharmaceuticals. Cobitolimod is a new type of drug that can help patients with moderate to severe ulcerative colitis back to a normal life. It is a so-called Toll-like receptor 9 (TLR9) agonist, that can provide an anti-inflammatory effect locally in the large intestine, which may induce mucosal healing and relief of the clinical symptoms in ulcerative colitis. Cobitolimod has achieved clinical proof-of-concept in moderate to severe active ulcerative colitis, with a very favourable safety profile. Data from four placebo-controlled clinical trials indicate that cobitolimod has statistically significant effects on those endpoints that are most relevant in this disease, both from a regulatory and clinical perspective. These endpoints include the key clinical symptoms such as blood in … Continue reading →

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