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

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

Marcos Reyes and Valeria Martínez are in a tough spot, unsure which of their twins should receive the treatment they ...

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

Make-A-Wish and the Buffalo Bills are helping one Bills fan’s dreams come true. Sixteen-year-old Tommy Parzymieso tells us he’s a manager for the Orchard Park High School Football team, and his goal ...

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

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

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

EveryONE Medicines, a company pioneering a scalable, global framework to design, develop, and manufacture individualized medicines for children with life-threatening neurodegenerative diseases, ...

Pratteln, Switzerland, April 16, 2025 – Santhera Pharmaceuticals (SIX: SANN) announces the latest progress on the commercial rollout of AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD).

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

NBC News’ chief Washington and chief foreign affairs correspondent Andrea Mitchell has been tapped to receive this year’s ...