Northern Minnesota boy is first American to receive groundbreaking treatment for muscular dystrophy
An 8-year-old northern Minnesota boy is one of the first in the country to receive a specific type of gene therapy treatment.
MedPage Today on MSN5d
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 ...
Clinical Trials Arena on MSN3d
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 ...
BioSpace3d
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 ...
Teen’s death following Sarepta DMD gene therapy underscores a risk seen for decades
The death of a 16-year-old boy evokes a lengthy battle to realize the benefit of cutting-edge gene therapy while also ...
Regenxbio Touts Positive Biomarker Data From Duchenne Muscular Dystrophy Gene Therapy Trial
Regenxbio's RGX-202 gene therapy shows promising results in Duchenne muscular dystrophy, with high microdystrophin levels and ...
Sarepta's gene therapy under scrutiny after patient death
A patient has died while receiving Elevidys, a gene therapy for Duchenne muscular dystrophy, marking the first known death ...
Teenager who received Elevidys, Sarepta’s Duchenne gene therapy, dies
Elevidys was approved after intense debate at the FDA, as officials and reviewers disagreed over how strong the evidence was ...
Managed Healthcare Executive4d
Promising Early Results of Investigational Duchenne Muscular Dystrophy Therapy
Avidity Biosciences Inc. says will submit an application for accelerated approval to the FDA for del-zota before the end of ...
U.S. News & World Report5d
Patient Dies Following Muscular Dystrophy Gene Therapy, Sarepta Reports
It’s the first known patient death with the therapy, which has been ... about its effectiveness in treating Duchenne muscular dystrophy. It’s the first gene therapy approved in the U.S ...