Children suffering from a rare disease may soon be able to benefit from a new treatment, which was previously not available ...

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 ...

Investors who adopt systems thinking when approaching the space economy can enhance their ability to understand and influence ...

Make-A-Wish and the Buffalo Bills are helping one Bills fan’s dreams come true. Sixteen-year-old Tommy Parzymieso tells us he ...

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 ...

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