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

Michael Rankin, 12, who is desperately waiting for access to the drug, said: “Every second they don’t get this through, one ...

A trial found magnetic resonance imaging correlated with physical function tests in patients with Duchenne muscular dystrophy ...

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

The remote measurement tool enables Duchenne muscular dystrophy (DMD) assessments through analysis of caregiver-recorded ...

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

A major trial of a promising gene therapy for Duchenne muscular dystrophy has fallen short, but researchers say the lessons learned could pave the way for better, more effective treatments.

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 ...

A Southern Oregon family is embarking on a remarkable journey to support their son, who is facing a life-threatening disease.

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