Two-year study shows lasting gains from Elevidys gene therapy for DMD
A single gene therapy dose helped young boys with Duchenne muscular dystrophy keep their strength longer than expected.
News
-
-
International study reveals high caregiver burden in DMD
Caregivers of individuals with DMD report substantial disruptions to work and everyday life across the U.S., Europe and Japan, study finds.
-
Researchers examine how to improve safety of gene therapy for Duchenne
Gene therapy for Duchenne muscular dystrophy (DMD) shows real promise, but high doses have caused rare serious side effects.
-
Study finds environmental factors lower participation in daily life in DMD
Children with DMD take part in fewer daily activities than peers, and physical environmental obstacles appear to be the key factor.
-
Study reveals effects of work and income disruptions among DMD caregivers
Caregivers of individuals with DMD face substantial impacts on employment, productivity and income, new research finds.
-
Case report: Gastric dilation in a teenager with DMD
Gastrointestinal manifestations remain relatively overlooked in terms of possible symptoms of DMD.
-
HHS adds Duchenne to newborn screening panel
Adding DMD and metachromatic leukodystrophy to newborn screening can help identify affected babies before symptoms appear.
-
Study finds zoledronic acid improves bone density in boys with DMD
Zoledronic acid increased spine and hip bone density in boys with Duchenne muscular dystrophy, but fracture risk remained during follow-up.
-
DMD RNA imbalance may limit benefits of genetic therapy
Uneven dystrophin RNA production is common in DMD and may limit how much dystrophin current genetic therapies can restore in muscle.
-
Telerehabilitation is a useful and effective approach in DMD, study finds
Telerehabilitation can expand the delivery and use of physical rehabilitation services in DMD.
