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 ...

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

Sarepta Therapeutics has ended development of a late-phase Duchenne muscular dystrophy (DMD) drug candidate over a safety problem. The safety signal deprived Sarepta of a near-term shot at accelerated ...

REGENXBIO Inc. (Nasdaq: RGNX) today reported financial results and recent operational highlights for the third quarter ended September 30, 2024.

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

Brian Skorney, an analyst from Robert W. Baird, maintained the Buy rating on RegenXBio (RGNX – Research Report). The associated price ...

CHARLESTOWN, Mass., Nov. 06, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB) (the "Company” or "Solid”), a ...

Gain insight on Rigel Pharmaceuticals, AbbVie and more in the latest Market Talks covering the Health Care sector.

Sarepta has stopped developing SRP-5051, its exon 51-skipping therapy for Duchenne MD, due to long-term safety concerns and ...

“We need help from other people,” pleaded Pavlina. “We saw videos from the children who were already there, it [the treatment ...

EBR Systems is among ASX healthcare stocks with upcoming catalysts, expecting feedback on a FDA substantive review expected ...