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.
Sportschosun (English) on MSN2d
New Treatment Strategy for Rare Disease Dutsenic Muscle Dystrophy...EZH2 inhibitor steroid effective in combination
Korean researchers have proposed a new treatment strategy that can increase the effectiveness of the genetic rare disease ...
MedPage Today on MSN4d
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 Care1d
Delandistrogene Moxeparvovec Shows Long-Term Impacts at 3 and 5 Years in DMD
Study results support the role of functional dystrophin and suggest that delandistrogene moxeparvovec stabilizes or slows ...
Gene therapy loses luster as investors eye quicker returns from weight-loss drugs
(Reuters) -Gene therapy, with its offer of a possible cure for rare diseases like sickle cell, is losing early investors to ...
Becker's Hospital Review3d
1st patient death linked to muscular dystrophy treatment
Cambridge, Mass.-based Sarepta Therapeutics reported the first known death from its Duchenne muscular dystrophy gene therapy ...
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 ...
The American Journal of Managed Care4d
How Access to SMA Treatment Varies Globally and by Insurance Type
Posters presented at the 2025 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference show that therapeutic ...
MPR1d
Patient Dies Following Treatment With DMD Gene Therapy Elevidys
The Company is currently analyzing the case and will update the prescribing information for Elevidys to note this development.
GEN - Genetic Engineering and Biotechnology News4d
DMD Patient Dies After Treatment with Sarepta Gene Therapy
The patient, whom Sarepta described as a young man and later told news outlets was 16 years old, suffered acute liver failure ...
Sarepta says teen died after its gene therapy treatment
Sarepta Therapeutics said on Tuesday a young man has died due to acute liver failure after treatment with its gene therapy for a rare muscular dystrophy.
Sarepta falls after notifying of patient death following treatment with Elevidys
Therapeutics are down 24.95, or 25%, to $76.40 in pre-market trading after the company shared a safety update related ...