Advancements in gene therapy for Duchenne muscular dystrophy (DMD) have improved the outlook for some patients, but further ...

Duchenne is caused by mutations of the DMD gene, which affects the production of the muscle ... Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by ...

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 ...

DMD Long-term functional outcomes ... Evrysdi is a survival motor neuron 2 (SMN2) pre-mRNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to survival motor ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is ...

Duchenne muscular dystrophy (DMD) is a genetic condition that causes progressive muscle weakness and wasting. It is an X-linked recessive disorder that occurs due to changes in the DMD gene ...

An early transcriptomic investigation highlighted SMN2 and SMN1 expression as a possible indicator of nusinersen efficacy in spinal muscular atrophy (SMA), according to a recently published study in ...

Their son, Michael, was diagnosed with Duchenne muscular dystrophy (DMD) at four years old. In the years following, Robert and his family established Michael's Cause, a nonprofit dedicated to ...

mL. Treatment-naïve SMA individuals, control individuals, and individuals with DMD and DM1 showed baseline plasma pNF-H levels < BLQ, as well as asymptomatic siblings identified through newborn ...

A recent study suggests virtual reality and biofeedback training may be feasible and effective for patients with Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD). Virtual ...

I think one of the programs that can bring about significant value would be the ability for it to advance the use of AOC 1044 for the treatment of patients with Duchenne Muscular Dystrophy (DMD ...