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.
Morningstar13d
New data on investigational therapy for thymidine kinase 2 deficiency presented at Muscular Dystrophy Association (MDA) 2025 Conference
Data from untreated participants were pooled from literature reviews of case series and reports, and a retrospective chart review study. Subgroups were stratified by age of TK2d symptom onset ...
Medical News Today5d
Nutritional considerations for Duchenne muscular dystrophy (DMD)
The symptoms appear early in life, and most affected individuals require the use of a wheelchair by the age of 12. Muscular dystrophy, loss of mobility, and complications from treatment can affect ...
Sarepta Therapeutics Reports Patient Death Post Elevidys Gene Therapy Treatment, Stock Plunges
Sarepta Therapeutics, Inc. (NASDAQ:SRPT) stock is sliding. The company shared an update on Elevidys (delandistrogene moxeparvovec-rokl), the only approved gene therapy in patients with Duchenne ...
The Boston Globe5d
Wave Life Sciences to submit Duchenne drug for approval after Phase 2 success
This story is republished from STAT, the health and medicine news site that’s a partner to the Globe. Sign up for STAT’s free Morning Rounds newsletter here. Wave Life Sciences’ experimental ...
La Grande Observer28d
CureDuchenne and Children’s Hospital of Orange County Partner to Bring World-Class Care to Families with Duchenne Muscular Dystrophy
March 3, 2025 /PRNewswire/ — CureDuchenne, a global leader in Duchenne muscular dystrophy (DMD ... partnership with CureDuchenne will help us chart a bright future,” said Dr. Julian Thomas ...