Interim clinical study results for the BB-301 Phase 1b/2a Treatment Study including 12-month follow-up results for the first four Cohort 1 completers, 24-month clinical study results for the first ...

Roche’s autoimmune drug Enspryng is making solid progress as a treatment for neuromyelitis optica spectrum disorder (NMOSD).

KYOTO--Utilizing genome editing technology, a team of researchers has developed a much more effective treatment for a certain type of muscular dystrophy. The method has recently been completed by the ...

Del-desiran reduced DMPK mRNA levels, but led to two serious adverse events ...

ITF Therapeutics LLC, the U.S. rare disease affiliate of Italfarmaco, today announced that one oral and nine poster presentations have been accepted at the Muscular Dystrophy Association (MDA) ...

Late-breaking poster to feature new positive cardiopulmonary function results from the DELIVER trial of z-rostudirsen in DMD -- Oral ...

Duchenne muscular dystrophy (DMD) is a rare neurological condition that causes severe muscle weakness and intellectual disability. DMD is an inherited (passed down) disorder. The condition is linked ...

Muscular dystrophy can appear in infancy up to middle age or later, and its form and severity are determined in part by the age at which it occurs. Some types of muscular dystrophy typically affect ...

A lower court had previously ruled for Sarepta in the companies’ long-running dispute, finding that REGENXBIO’s AAV patent ...

Muscular dystrophy is a type of disease that causes the muscles in your body to lose strength and mass. As your muscles become weaker over time, it may be hard for you to do normal activities.

Regenxbio filed a lawsuit in 2020 alleging that Elevidys infringed a gene-therapy patent that the company licenses from ...