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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).
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 ...
A trial found magnetic resonance imaging correlated with physical function tests in patients with 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, ...
Two dose levels of a single-administration gene therapy were well-tolerated and led to functional improvements in ambulatory ...
RFK Jr.'s Health Department shut down an advisory group that recommends how to test newborns for genetic disorders ...
Advocates fear the U.S. could return to a patchwork system in which richer states provide more comprehensive screenings than ...
A Southern Oregon family is embarking on a remarkable journey to support their son, who is facing a life-threatening disease.