Sarepta Therapeutics SRPT announced updates from its clinical programs, which focus on targeting different subtypes of ...

Duchenne Muscular Dystrophy: diagnosis, clinical development and global researchMuscular dystrophy refers to a group of rare, genetic, progressive diseases that primarily affect the body’s muscles, ...

Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular dystrophy (DMD), after the treatment failed to show significant benefit in a ...

A new article argues dystrophin immunogenicity is an under-examined issue in the treatment of Duchenne muscular dystrophy.

Discover how an emerging technology called tRNA therapy could become the next big thing in genetic disease treatment.

Spinal muscular atrophy (SMA) is a disorder affecting the motor ... Most forms of SMA are caused by mutations of the survival motor neuron 1 gene (SMN1) on the fifth chromosome, resulting in ...

New research led by the University of Portsmouth has revealed how Duchenne muscular dystrophy (DMD), best known for causing severe muscle degeneration, also profoundly affects the brain, leading to ...

Becker muscular dystrophy (BMD) is a type of muscular dystrophy ... Dystrophinopathies (including BMD and DMD) happen due to certain mutations in the genetic code. One gene, for instance, controls ...

The beginning to the year has been eventful in the Duchenne muscular dystrophy (DMD ... administered intravenously to DMD patients with mutations amenable to Exon 44 skipping. The trial is due to ...

Sarepta Therapeutics said Tuesday that a patient died while taking its closely watched gene therapy for muscular dystrophy, sending company shares plummeting more than 25%. A recent study ...