Genetic mechanisms underlying Duchenne muscular dystrophy point to potential treatments
Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...
Max Planck Society2d
Mechanism for treating muscle wasting discovered
Utrophin increase in muscle cells after transcriptional adaptation normalises cell function in Duchenne muscular dystrophy ...
Managed Healthcare Executive2d
Entrada Therapeutics On Track For New DMD Study in the U.K.
Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, ...
Managed Healthcare Executive2d
Sarepta Announces Positive Results for Elevidys, Gene Therapy for Duchenne
Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...
Dyne Therapeutics Announces Upcoming Presentations at the 2025 MDA Clinical & Scientific Conference
Data from DELIVER and ACHIEVE Clinicals Trial to be Presented -- Presentation on ACHIEVE Trial in DM1 and Company Symposium to Feature Data on ...
Medscape17d
Calpain 3 Is Important for Muscle Regeneration
To be able to determine if mutations in specific domains of CAPN3 ... we included five clinically matched patients with Becker muscular dystrophy (BMD) (age 31 ± 16 years) and five patients ...
Cumberland Pharmaceuticals' Duchenne Candidate Shows Improved Heart Function In Patients With Associated Heart Disease
Cumberland Pharmaceuticals Inc. (NASDAQ:CPIX) released topline results from its Phase 2 FIGHT DMD trial. The study evaluated ...
BioWorld8d
Repurposing CFTR corrector to treat muscular dystrophies caused by misfolded α-sarcoglycan
Limb-girdle muscular dystrophy type 2D (LGMD2D/R3) is a rare genetic disorder caused by mutations in the SGCA gene, leading to defective folding and the loss of functional α-sarcoglycan, with ...