MedPage Today on MSN2d
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 ...
Selling shamrocks for Muscular Dystrophy Awareness
While plenty of people are going green in honor of Saint Patrick's Day, one student at East Stroudsburg University is turning ...
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 ...
The American Journal of Managed Care3d
MRI Could Aid in Early Differential Diagnosis of DMD, BMD
Multiparametric quantitative MRI could potentially help differentiate between Duchenne muscular dystrophy and Becker muscular dystrophy early and improve the management of these conditions.
Managed Healthcare Executive1d
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 ...
The American Journal of Managed Care2d
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 ...
Business Insider10d
FDA Approves Expanded Use of Eculizumab (Soliris) for Pediatric Myasthenia Gravis, Bringing New Treatment Options for Children
“The availability of Soliris for children underscores the importance of continued ... thanks to donors and supporters who generously give to organizations like Muscular Dystrophy Association because ...