Muscular dystrophy (MD) represents a group of inherited muscle disorders that primarily cause muscle weakness and muscle wasting, though some types of the disease also present with other ...
The FDA has granted fast track designation to DYNE-101, a therapy for myotonic dystrophy type 1 (DM1) now in testing in a ...
An investigational higher dose of spinal muscular atrophy drug nusinersen gains attention as the FDA and European Medicines Agency (EMA) considerate it as an alternative than the current lower ...
Application is supported by results from the DEVOTE study, which suggested that two doses of Spinraza (nusinerse) 50 mg taken ...
We are pleased to announce that our applications for the higher dose regimen of nusinersen are now under review in the US and Europe,” said Stephanie Fradette, Pharm.D., Head of the Neuromuscular ...
CHARLESTOWN, Mass. - Solid Biosciences Inc. (NASDAQ:SLDB), a $130.65 million market cap biotechnology company, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) ...
Regulators in the U.S. and the European Union agreed to review Biogen’s applications for approval of a higher dose Spinraza ...
The FDA has granted fast-track designation to an investigational antisense oligonucleotide to treat individuals with myotonic ...
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Jan. 21, 2025 — A new paper led by Professor Imanuel Lerman of UC San Diego provides a review of the field of bioelectronic medicine and the most promising opportunities for life-changing new ...