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An FDA expansion of Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) Elevidys has sparked a new surge in sales for the ...

Researchers created a mouse model to study liver symptoms of myotonic dystrophy type 1, revealing how toxic RNA in the liver ...

People with Myotonic Dystrophy Type 1 (DM1), the most common adult-onset form of muscular dystrophy, progressively lose ...

New Gene Therapy Approach Shows Promise for Duchenne Muscular Dystrophy July 24, 2024 — Researchers have made a significant breakthrough in developing a new gene therapy approach that restores ...

Sarepta Therapeutics (Nasdaq: SRPT) has announced it will cease development of SRP-5051, an experimental drug for Duchenne ...

Elevidys sales have increased since the FDA made a controversial choice to expand the therapy's use. Meanwhile, Sarepta is abandoning a successor to its drug Exondys 51, citing an “evolving" treatment ...

Sarepta Therapeutics is advancing SRP-9003 gene therapy for LGMD, with phase 3 study results expected in early 2025. Learn ...

(AskBio), a gene therapy company wholly owned and independently operated as a subsidiary of Bayer AG, today announced that AB-1003 (also known as LION-101) has received rare pediatric disease ...

Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, announced today that the United States (U.S.) Food and Drug Administration (FDA) granted Orphan Drug Designation and ...

Analyst Joseph Schwartz from Leerink Partners maintained a Buy rating on Solid Biosciences (SLDB – Research Report) and keeping the price ...

Research Triangle Park, North Carolina Saturday, November 9, 2024, 13:00 Hrs [IST] ...