rarediseaseadvisor3d
Optimizing Diet and Physical Therapy Is Important in Patients With Neuromuscular Diseases
DALLAS—The careful optimization of diet and physical therapy is critical for the care of patients with neuromuscular diseases such as Duchenne muscular ... challenges in pediatric obesity. Oral ...
BioSpace1d
In Wake of Elevidys Death, Duchenne Patient Community Vows to Push On
After Sarepta reported the death of a patient who had recently taken the gene therapy Elevidys, patient advocacy group Parent ...
Patient dies following muscular dystrophy gene therapy, Sarepta reports
A patient has died while taking a closely watched gene therapy for muscular dystrophy. Sarepta Therapeutics announced the ...
MedPage Today on MSN2d
Young Patient Dies After Receiving New Gene Therapy for Duchenne Muscular Dystrophy
A young person with Duchenne muscular dystrophy died following treatment with the recently approved gene therapy ...
The American Journal of Managed Care3d
Solving the Transition Conundrum as More Children With Muscular Dystrophy Live to Adulthood
Learning from examples like congenital heart disease and cystic fibrosis can help health systems and clinicians prepare to care for an influx of patients with neuromuscular diseases as they reach ...
STAT3d
Teenager who received Elevidys, Sarepta’s Duchenne gene therapy, dies
Sarepta Therapeutics said that a young man died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy ...
TwinCities.com2d
Patient dies following muscular dystrophy gene therapy, Sarepta reports
In 2023, Elevidys received expedited U.S. approval despite concerns from some Food and Drug Administration scientists about its effectiveness in treating Duchenne muscular dystrophy. It’s the ...
The Boston Globe3d
Teenager who received Elevidys, Sarepta’s Duchenne gene therapy, dies
Sarepta Therapeutics said Tuesday that a 16-year-old boy died after receiving Elevidys, its gene therapy for Duchenne muscular dystrophy ... largely in young children and the risks of gene ...