New phase 3 data show meaningful improvement in antibody-negative myasthenia gravis, for which treatment options are limited ...

FDA Approves KYGEVVI® (doxecitine and doxribtimine), the First and Only Treatment for Adults and Children Living with Thymidine Kinase 2 deficiency (TK2d), a Rare and Devastating Mitochondrial Disease ...

Sarepta stock crashed late Monday after the biotech company said two of its muscular dystrophy drugs failed their ...

Detailed price information for Solid Biosciences Inc (SLDB-Q) from The Globe and Mail including charting and trades.

The ESSENCE trial was intended to prove the efficacy of Vyondys 53 (golodirsen) and Amondys 45 (casimersen), which received ...

That research led to the FDA granting compassionate use of the doxecitine and doxribtimine treatment in Arturito Estopinan, ...

Smartphones can accurately assess neuromuscular diseases, using video and free apps that provide faster and easier way to detect diseases earlier.

Two of Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) candidates have failed to reach their primary endpoint in a ...

Sarepta shares are plunging in extended trading Monday after the company reports third-quarter results and provided an update on its ESSENCE study.

Columnist Betty Vertin shares how she explained Duchenne muscular dystrophy to her sons, who received their DMD diagnosis at a young age.

Duchenne (SGT-003): 23 participants have been dosed in the INSPIRE DUCHENNE trial as of October 31, 2025; Solid expects to have dosed 30 ...

Learn more about whether ACADIA Pharmaceuticals Inc. or BioMarin Pharmaceutical Inc. is a better investment based on AAII's A+ Investor grades, which compare both companies' key financial metrics.