Multidisciplinary coordination across prescribing teams, nursing, laboratory medicine, finance, and infusion centers is crucial for gene therapy delivery in Duchenne muscular dystrophy (DMD).

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, ...

July 17, 2024 — A new gene therapy treatment for Duchenne muscular dystrophy (DMD) shows promise of not only arresting the decline of the muscles of those affected by this inherited genetic ...

Newborn screening is also cost-effective. Testing that takes just a few dollars per baby can save families and the health ...

DMD was first described by French neurologist Guillaume Benjamin Amand Duchenne in the 1860s. However, until the 1980s, ...

It can worsen with age. However, its symptoms are less severe than those of Duchenne muscular dystrophy (DMD), which is more common. Becker muscular dystrophy (BMD) is a type of muscular ...

A Phase 3 clinical trial investigating Elevidys (delandistrogene moxeparvovec), a gene therapy for Duchenne muscular dystrophy, did not achieve its primary endpoint, according to findings ...

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

Doing more to incorporate caregiver burden into value assessments and cost-effectiveness research of Duchene muscular ...

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 ...