Data from DELIVER and ACHIEVE Clinicals Trial to be Presented -- Presentation on ACHIEVE Trial in DM1 and Company Symposium to Feature Data on ...

Understanding the behavior of tiny droplets in our cells could aid the search for new treatments. A team of Leiden ...

Duchenne muscular dystrophy (DMD) is a rare hereditary disease that is associated with progressive muscle wasting. The ...

Managed Healthcare Executive provides C-suite executives at health plans and provider organizations with news, analysis, ...

Results show improved functional outcomes for people with Duchenne muscular dystrophy who are ambulatory. Elevidys, which is ...

Utrophin increase in muscle cells after transcriptional adaptation normalises cell function in Duchenne muscular dystrophy ...

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 ...

Despite the obstacles, Riley Herrera had a life filled with family, sports, travel, faith and hope before his death on Nov.

Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company with development efforts focused on new products for rare diseases, today announced positive top-line results from ...

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 ...

Roche and Sarepta Therapeutics have shared positive top-line results from a late-stage study of Elevidys (delandistrogene moxeparvovec) in children with the rare muscle-wasting disorder Duchenne ...