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 ...
Clinical Trials Arena on MSN1d
Regenxbio’s DMD gene therapy drives 122.3% biomarker uptick in three-year-old
The interim data found one child in the Duchenne trial produced more than double the amount of the protein needed to maintain ...
MedPage Today2d
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 ...
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 ...