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 ...

Thanks in part to federal funding for newborn hearing screening, about 98% of newborns are screened for hearing loss before ...

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

Scholar Rock Holding Corporation's apitegromab shows promise for SMA treatment, aims for 2025 launch. Click for my updated ...

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

Medcare Women & Children Hospital (MWCH), a leading premium multi-speciality healthcare provider in the UAE dedicated to ...