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Lives hang in the balance while the FDA bureaucracy churns – Washington Examiner

Posted: Published on August 10th, 2017

The McLinn family of Indianapolis is still fighting for their seven-year-old son, Jordan, who was diagnosed at the age of 3 with Duchenne muscular dystrophy. This is an aggressive disease that results in muscle weakness and loss first attacking the extremities, eventually moving to the heart and other internal organs. There is no known cure, yet, but there are treatments that can help manage symptoms and slow progression of the disease. For people like Jordan McLinn, failure or needless delay of the Food and Drug Administration to grant a compassionate use request (also known as expanded access) can mean the difference between walking or being wheelchair-bound. Currently, the FDA has the authority to not approve access to potentially life-saving medicine based on the agency's criteria meaning, the FDA must determine that "the potential patient benefit justifies the potential risks of the treatment use and those potential risks are not unreasonable in the context of the disease or condition to be treated." In a country that prides itself on upholding individual liberties, it's contradictory that the government reserves the right to grant or deny individuals who are fighting for their lives access to potentially life-saving medicine simply based on the agency's … Continue reading →

Posted in Muscular Dystrophy Treatment | Comments Off on Lives hang in the balance while the FDA bureaucracy churns – Washington Examiner

Multiple sclerosis vs. fibromyalgia: Similarities, differences and treatments – Herald Palladium

Posted: Published on August 10th, 2017

There is no known cure for fibromyalgia or multiple sclerosis.Both are life-altering and have similar symptoms. New advancements in science and technology have producednew treatments and exercises that can help remedy symptoms of these disorders. Similarities and differences in symptoms Multiple sclerosis and fibromyalgia are similar, yetdifferent, diseases. Theyhave similar symptoms, which can make a proper diagnosis difficult. According to the National Multiple Sclerosis Society, 400,000 Americans currently suffer from MS. According to the Centers for Disease Control, 4million Americans havefibromyalgia. Women are two to three times more likely to develop MS or fibromyalgia than men, according to reports from the CDC and healthline.com. Overlapping symptoms include chronic pain, chronic fatigue, depression, anxiety, headaches, systemic lupus, rheumatoid arthritis and memory issues. In addition to these symptoms, both have uniquesymptoms. A person with fibromyalgia may experience mood swings, sleep disorders and nausea, while those with MS can havedifficulty walking, slurred speech and vision problems. Diagnosis Currently, there is little known about fibromyalgia other than it is a musculoskeletal disease that increases a persons sensitivity to pain. Itwas not considered to be a disease by the American Medical Association until 1987, which contributes to the more limited research. A New York Times … Continue reading →

Posted in MS Treatment | Comments Off on Multiple sclerosis vs. fibromyalgia: Similarities, differences and treatments – Herald Palladium

TG Therapeutics, FDA Agree on Phase 3 Program to Evaluate Relapsing MS Therapy TG-1101 – Multiple Sclerosis News Today

Posted: Published on August 10th, 2017

TG Therapeuticsand the U.S. Food and Drug Administration (FDA) have agreed on a special protocol assessment for a Phase 3 trial program evaluating TG-1101 (ublituximab)to potentially treatrelapsing forms of multiple sclerosis (MS). A special protocol assessment (SPA) is a procedure by which the agency officially evaluates the design and size of proposed protocols meant to ground a new drug application (NDA). The SPA marks the conditions under which the trial design adequately addresses objectives that, if met, will support consequent regulatory submission for approval of TG-1101. Stanford Universitys Dr. Lawrence Steinman will lead the program, which is expected to begin this year. Itincludes two Phase 3 clinical trials (ULTIMATE I and ULTIMATE II) both randomized, double-blind, active-controlled studies comparing TG-1101 to Aubagio (teriflunomide) in patients with relapsing MS. By targeting relapsing MS, TG-1101 could potentially be approved to two particular forms of the disease:relapsing-remitting MS (RRMS) and secondary progressive MS (SPMS), which often follows RRMS. TG-1101 is TGs novel glyco-engineered monoclonal antibody that targets a specific and unique epitope on the CD20 proteinfound on mature B-cells. The primary endpoint of the trials will be changes inannualized relapse rate after 96 weeks of treatment in about 440 randomized patients in each … Continue reading →

Posted in MS Treatment | Comments Off on TG Therapeutics, FDA Agree on Phase 3 Program to Evaluate Relapsing MS Therapy TG-1101 – Multiple Sclerosis News Today

The amazing story of Kyle Miller overcoming cerebral palsy to play … – GolfDigest.com

Posted: Published on August 10th, 2017

Kyle Miller picked up the phone in his office at GolfTEC Calgary Midnapore a few weeks ago and called Colin Lavender, tournament director of the Mackenzie Tour-PGA Tour Canadas ATB Financial Classic. Miller had already written a letter to Lavender requesting a sponsors exemption into the tournament. Having been rejected 14 previous times for spots into other eventsand told all his life as someone with cerebral palsy that he couldnt achieve thingsMiller figured a call would go a long way to pitch his story. It did. Three weeks later, on the same day he had turned professional in 2013, Miller found out he had been given a spot in the field. On Thursday, at 2:30 p.m., the 26-year-old will tee it up at Country Hills Country Club in his hometown of Calgary, becoming the first golfer with cerebral palsy to tee it up in a PGA Tour-sanctioned event. In that conversation, I pled my case for an hour and a half, Miller said in a phone interview on Wednesday. And I think that time, rather than trying to get a Yes, I just made it clear that I wanted to know why hed say No in this point in my … Continue reading →

Posted in Cerebral Palsy | Comments Off on The amazing story of Kyle Miller overcoming cerebral palsy to play … – GolfDigest.com

Report: Inmate accused of punching man with cerebral palsy claims … – PhillyVoice.com

Posted: Published on August 10th, 2017

Barry Baker, the man chargedwith sucker punching a disabled manoutside a local 7-Eleven, claims that he was recently attacked by a corrections officer at the Chester County Prison, according to a letter obtained by The Daily Local News. The incident, which occurred May 10 in West Chester, provoked viral outrage after the Chester County District Attorney's Office released surveillance video of it, calling on the public to help locate the "bully" who mocked a 22-year-old man with cerebral palsy. Authorities said Baker, a resident of Georgetown, Delaware, had twice mocked the way the man walked before punching him in the face in the parking lot of the convenience store. Baker fled the scene, failed to turn himself inafter he was briefly in police custody and was eventually captured June 5 following a "two-week manhunt" by the U.S. Marshals and Chester County Sheriffs, according to authorities. In a letter dated Aug. 2, Baker, identifying himself as "the person whom you guys love writing about," told a reporter at The Daily Local that he was "roughed up" by an officer in the protective custody block where he has been held since his arrest. Baker reportedly claimed the assault took place July 30 … Continue reading →

Posted in Cerebral Palsy | Comments Off on Report: Inmate accused of punching man with cerebral palsy claims … – PhillyVoice.com

Fearing stigmatization, patient’s father seeks retraction of paper on rare genetic mutation – Retraction Watch (blog)

Posted: Published on August 9th, 2017

The father of a boy with a rare genetic mutation has accused a scientist of exploiting his child by proclaiming the defect a genetic syndrome and naming it after herself. At an impasse with scientists investigating, publicizing, and interpreting his sons condition, the father seems willing to use any leverage he can muster to remove the syndrome entry in an online genetic disease database. Based solely on an email he obtained from the database director, the father became convinced that if the paper underpinning the entry were retracted, the syndrome would go down with it. So earlier this year, he withdrew his consent and asked the journal that published the paper for a retraction, based on improper patient consent. He has also threatened to lob accusations of research misconduct at the papers last author. Marc Pieterse, of The Netherlands, is the father of Vincent, a teenager who has a mutation in the RPS23 gene that has only been found in one other person, so far. In March, an international team of researchers published a paper on Vincents RPS23 mutation in the American Journal of Human Genetics (AJHG), linking it to defective ribosomes, organelles involved in protein synthesis. One of the … Continue reading →

Posted in Human Genetics | Comments Off on Fearing stigmatization, patient’s father seeks retraction of paper on rare genetic mutation – Retraction Watch (blog)

Modification of genes in human embryos could mark turning point in human evolution – The Globe and Mail

Posted: Published on August 9th, 2017

It appears, by all accounts, to be a momentous scientific achievement and possibly a turning point in human evolution. In a study released last week, scientists at Oregon Health and Science University confirmed they were able to modify genes in viable human embryos, proving the potential to permanently alter the makeup of a genetic line. In this case, that meant replacing and repairing a mutated gene that causes a common and deadly heart disorder. But the possibilities heralded by gene-editing technology are endless, the scenarios as divided as they are bold. In some visions, it leads to a population of designer babies or consumer eugenics. Others imagine a utopia of scientific advancement where humans live free of disease, and devastating conditions are eradicated for the betterment of humanity. What direction the technology will take is the topic of much debate. The big thing which is making the scientific and ethics community get excited, and on the other hand a little bit hot and bothered, is its a mechanism to change genes for multiple generations, says Dr. Alice Virani, a genetic counsellor and director of ethics at British Columbias Provincial Health Services Authority. There are two ways to look at it, … Continue reading →

Posted in Human Genetics | Comments Off on Modification of genes in human embryos could mark turning point in human evolution – The Globe and Mail

The best way to fix broken bones might be with glass – BBC News

Posted: Published on August 9th, 2017

In 2002, Ian Thompson, a specialist in facial reconstruction at Kings College, London, received an urgent phone call. A patient in his late 20s had been struck by an out-of-control car mounting the pavement. The impact had sent him catapulting over the bonnet of the car, smashing his face and shattering the fragile orbital floor the tiny bone, no more than 1mm thick, which holds the eyeball in place in the skull. Without the orbital floor, your eye moves backwards into the skull, almost as a defensive mechanism, Thompson explains. But this results in blurred vision and lack of focus. This patient had also lost the ability to perceive colour. His job involved rewiring aircraft and as he could no longer detect a red wire from a blue one, hed barely been able to work in three years. The accident had happened three years earlier. Since then, surgeons had desperately tried to reconstruct the bony floor and push the eye back into position, first using material implants and then bone from the patients own rib. Both attempts had failed. Each time, infection set in after a few months, causing extreme pain. And now the doctors were out of ideas. You … Continue reading →

Posted in Future Medicine | Comments Off on The best way to fix broken bones might be with glass – BBC News

Gene Therapy Is Now Available, but Who Will Pay for It? – Scientific American

Posted: Published on August 9th, 2017

By Ben Hirschler LONDON (Reuters) - The science of gene therapy is finally delivering on its potential, and drugmakers are now hoping to produce commercially viable medicines after tiny sales for the first two such treatments in Europe. Thanks to advances in delivering genes to targeted cells, more treatments based on fixing faulty DNA in patients are coming soon, including the first ones in the United States. Yet the lack of sales for the two drugs already launched to treat ultra-rare diseases in Europe highlights the hurdles ahead for drugmakers in marketing new, extremely expensive products for genetic diseases. After decades of frustrations, firms believe there are now major opportunities for gene therapy in treating inherited conditions such as haemophilia. They argue that therapies offering one-off cures for intractable diseases will save health providers large sums in the long term over conventional treatments which each patient may need for years. In the past five years, European regulators have approved two gene therapies - the first of their kind in the world, outside China - but only three patients have so far been treated commercially. UniQure's Glybera, for a very rare blood disorder, is now being taken off the market given … Continue reading →

Posted in Gene Therapy | Comments Off on Gene Therapy Is Now Available, but Who Will Pay for It? – Scientific American

Silverstein-backed startup will test gene therapy for Parkinson’s – FierceBiotech

Posted: Published on August 9th, 2017

Regenxbio has joined forces with investment firm OrbiMed and a new nonprofit foundation to create Prevail Therapeutics, a startup focused on new biologics and gene therapiesfor Parkinson's disease (PD). Prevail will draw on the expertise of the Silverstein Foundation for Parkinson's with GBA, which concentrates on a particular form of the disease caused by mutations in the glucocerebrosidase gene. The foundation was set up this year by OrbiMed's co-head of private equity Jonathan Silverstein, who was diagnosed with GBA-linked PD in February and is mobilizing efforts to discover a cure for the disease. Silverstein backed the foundation with $10 million of his own money, and is intent on accelerating research into PD with GBA as well as other forms of the disease. Prevail says it will focus initially on research coming out of the lab of its co-founder and CEO Asa Abeliovich, M.D., Ph.D., who is on the faculty of Columbia University as well as being a scientific adviser to the Silverstein Foundation and co-founder of neurodegenerative disease biotech Alector. By joining forces with Regenxbio, Prevail launches with an exclusive license to the gene therapy specialist's adeno-associated virus (AAV) based vector technology NAV AAV9 for PD and other neurodegenerative disorders. … Continue reading →

Posted in Gene Therapy | Comments Off on Silverstein-backed startup will test gene therapy for Parkinson’s – FierceBiotech

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