Atamyo Therapeutics has partnered with the Dion Foundation for Children with Rare Diseases to expand into the U.S.
Articles by Andrea Lobo
The U.S. Food and Drug Administration (FDA) has given WVE-N531, an investigational therapy for Duchenne muscular dystrophy (DMD) patients…
A Phase 3 trial testing an experimental therapy for limb-girdle muscular dystrophy type 2i (LGMD2i) has surpassed patient enrollment for…
The gene therapy developed by Pfizer called fordadistrogene movaparvovec failed to significantly improve motor function in boys with…
The U.S. Food and Drug Administration (FDA) has approved the first generic version of Emflaza (deflazacort) oral suspension for…
After nearly three months of treatment with pitolisant, adults with myotonic dystrophy type 1 (DM1) experienced a reduction of…
AMO Pharma has announced it will conduct a Phase 3 trial of AMO-02 (tideglusib), its investigational oral therapy…
Most people with myotonic dystrophy type 1 (DM1) develop heart problems, according to a Danish study that followed patients for about 10…
Oral treatment with EDG-5506, now known as sevasemten, continued to stabilize muscle function after two years in men with…
The U.S. Food and Drug Administration (FDA) has granted rare pediatric disease status to SGT-003, a next-generation gene therapy candidate…