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.
Clinical Trials Arena on MSN2d
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 ...
BioSpace2d
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 ...
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 ...
Becker's Hospital Review3d
1st patient death in muscular dystrophy treatment
Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...