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

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

A new paper published in Gene Therapy raises serious concerns about the effectiveness of gene therapy for Duchenne muscular ...

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

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

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

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, ...

Advocates fear the U.S. could return to a patchwork system in which richer states provide more comprehensive screenings than ...

Suneel Ram's caregivers share some of the lessons they have learned while caring for the 28-year-old who was diagnosed with ...

RFK Jr.'s Health Department shut down an advisory group that recommends how to test newborns for genetic disorders ...

Global Duchenne Muscular Dystrophy Drugs Market value attained USD 3.9 Billion in 2025. The market will achieve USD 6.5 ...

Is dystrophin immunogenicity a barrier to advancing gene therapy for Duchenne muscular dystrophy? Gene Therapy , 2025; DOI: 10.1038/s41434-025-00531-y Cite This Page : ...