CDMO giant Catalent inked a deal with Sarepta Therapeutics to manufacture what looks to become the first gene therapy to treat Duchenne muscular dystrophy (DMD). In November, the FDA gave its ...

Genethon highlights latest developments in gene therapies for diseases once considered incurable, including Duchenne muscular dystrophy ...

Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® ...

Under the agreement, Santhera will receive an initial payment of $40m from Nxera.

Taiho Pharmaceutical’s investigational treatment for Duchenne muscular dystrophy (DMD) has failed to impact the time it takes patients to rise from the floor over 52 weeks, missing the phase 3 study’s ...

Sarepta Therapeutics said it will update its prescribing information for Elevidys ® (delandistrogene moxeparvovec-rokl) after acknowledging the sudden death of a patient with Duchenne muscular ...

When it comes to children born with rare diseases, early diagnosis can make the difference between life and death. Since many ...

Don't catch a falling knife.

Jefferies analysts forecast a $1 billion market opportunity for each of Sarepta’s siRNA programs for facioscapulohumeral muscular dystrophy and myotonic dystrophy type 1.

One patient with Duchenne muscular dystrophy (DMD) has passed away following treatment with Sarepta Therapeutics’ gene therapy Elevidys, the biotech said (PDF) Tuesday. The patient suffered acute ...