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 ...

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

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

After escaping a clinical hold several years back, Dyne Therapeutics has revealed new phase 1/2 data for its Duchenne muscular dystrophy (DMD) therapy DYNE-251. The readout notes several serious ...

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 ...

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

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 ...

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 ...

Adult men with DMD have over twice the fracture risk compared to non-DMD counterparts, with corticosteroids increasing this risk further. Bone health monitoring is inadequate, with less than half of ...

Duchenne muscular dystrophy (DMD) occurs as a result of genetic changes on the X chromosome. If someone has a gene change that can cause DMD, their children may inherit that change. DMD is a ...