An experimental gene therapy from Sarepta Therapeutics increased levels of the gene missing in an ultra-rare form of muscular dystrophy, according to data the company presented Friday. The company has said it plans to file for approval in the disease, known as limb girdle muscular dystrophy (LGMD) 2E. That would make it the first approved…
In a setback for Sarepta Therapeutics, a New York panel has voted unanimously to recommend the state Medicaid program should pause coverage of a treatment for Duchenne muscular dystrophy in response to a high-profile safety controversy this past summer. The Drug Utilization Review Board late last week suggested the state should wait for the Food…
Sanofi Commits USD 625 Million to Sanofi Ventures to Boost Biotech and Digital Health Startups We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
Stealth BioTherapeutics has claimed FDA approval for Forzinity, the first treatment for ultra-rare disease Barth syndrome, just a few months after it was turned down by the regulator. Forzinity (elamipretide) can be used to treat patients weighing at least 30 kg with Barth syndrome, an X-linked, inherited mitochondrial disorder that causes an enlarged and weakened…
The summer has been filled with debates about the safety of Elevidys, Sarepta’s gene therapy for Duchenne muscular dystrophy. Four prominent researchers are now raising a fundamental question about its efficacy: How much gene does the gene therapy actually deliver? In principle, the question should be settled. Since its first trial in 2017, Sarepta has…
Brain-eating amoeba claims 2 more lives, including infant in Kerala We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
Sanofi’s BTK inhibitor Wayrilz has become the first drug in the class to be approved by the FDA for immune thrombocytopenia (ITP), a rare autoimmune blood disorder. Wayrilz (rilzabrutinib) has been cleared by the US regulator for adults with persistent or chronic ITP who have had an insufficient response to a previous treatment. In ITP,…
AMCH Dibrugarh designated as Centre of Excellence for rare diseases We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
Kerala launches campaign against Amoebic Meningoencephalitis amid 18 active cases We use cookies for analytics, advertising and to improve our site. You agree to our use of cookies by continuing to use our site. To know more, see our Cookie Policy and Cookie Settings.Ok Source link
The Food and Drug Administration has agreed to decide by late next month whether to approve an ultra-rare disease drug developed by Stealth BioTherapeutics, a significant step after the company claimed it may have to close its doors if an agency endorsement is not made in coming weeks. The move comes after protracted uncertainty surrounding…