Category Archives: Muscular Dystrophy Treatment

Suzy Cohen, Columnist Published 5:02 a.m. ET Dec. 30, 2019 Dear Readers, its time for my annual article regarding new medications of the year. Continue reading →

Sarepta Therapeutics and Roche inked a licensing deal that gives Roche exclusive commercial rights to SRP-9001, Sareptas investigation gene therapy for Duchenne muscular dystrophy (DMD), outside the U.S. DMD is a muscle wasting disease caused by mutations in the dystrophin gene. Continue reading →

GERMANTOWN, Md., Dec. 27, 2019 /PRNewswire/ -- The HealthWell Foundation, an independent non-profit that provides a financial lifeline for inadequately insured Americans, has opened a new fund to provide copayment and premium assistance to people living with Lambert-Eaton Myasthenic Syndrome (LEMS). Continue reading →

Jay Smith, a Central Bucks West graduate now living in Texas, continues his fight against ALS, with which he was diagnosed in 2014. Continue reading →

For biotech, 2019 ended like the penultimate episode of a prestige TV show. We got answers to some weighty questions, but mostly, the year left a breadcrumb trail to some major reveals. Continue reading →

What a difference a decade makes. Continue reading →

The newly approved therapyVyondys 53(golodirsen) is available for immediate distribution to treat people with Duchenne muscular dystrophy (DMD) amenable toexon 53 skipping, according to the medicines developer Sarepta Therapeutics. In an email reply toMuscular Dystrophy News Today,a spokeswoman added that, similar toExondys 51(eteplirsen, also marketed by Sarepta), the price ofVyondys 53 will be based on a patients weight, with an estimated annual cost of $300,000 for a child weighing 20 kg (44 pounds). Continue reading →

What happened Shares of Solid Biosciences (NASDAQ:SLDB) are getting hammered today, down by 14% as of 11:54 a.m. Continue reading →

In January 2019, then-FDA commissioner Scott Gottlieb ushered in the new year with a bold prediction: The agency, he said, would be approving between 10 and 20 gene and cell therapies per year by 2025. At the time, there were a whopping 800 such therapies in the biopharma pipeline and the FDA was aiming to hire 50 new clinical reviewers to handle the development of the products. Continue reading →

Details Category: More News Published on Friday, 20 December 2019 13:54 Hits: 143 Selecta eligible to receive upfront and milestone payments of over $240 million AskBios AAV gene therapy combined with ImmTOR could prevent the formation of neutralizing antibodies and potentially enable re-treatment of patients with Pompe disease WATERTOWN, MA and RESEARCH TRIANGLE PARK, NC, USA I December 19, 2019 ISelecta Biosciences, Inc. Continue reading →