Raredr

Musculoskeletal

Another Duchenne Boy Fighting to Get Approved Drug (Exondys 51)
Dr Koop noted, I think the conversation has to be not through us but between those who create and market the drugs and those who pay the bills.
Excellus BlueCross BlueShield is denying to provide the drug on the basis that the boys are not ambulatory and the drug is 'medically unnecessary'.
PTC said treatment for the average Duchenne patient will cost about $35,000 per year with Emflaza. Most Duchenne boys weigh more than the average.
Did PTC just replace Marathon as the most hated pharma company out there?
In preclinical studies, Dr. Olson’s lab used adeno-associated virus (AAV) and CRISPR/Cas9 technology to identify and correct exon mutations in the dystrophin gene.
I am guessing that the CEO of Marathon got some hate mail this weekend.
At this time, the company plans to continue assessing the data and also plans to continue with its open label extension of the study.
Levi is 3-years-old and has LCMD, rare form of congenital muscular dystrophy (CMD).
Gay and Steve Grossman, co-founders of ADCY5.org provide some advice for finding (and retaining) researchers to help patient advocacy groups
Priya Kishnani, MD, PhD was instrumental in getting an orphan drug approved for Pompe disease. But the management of Pompe disease requires a multidisciplinary approach and enzyme replacement therapy is only one part of that care.
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