After nearly three months of treatment with pitolisant, adults with myotonic dystrophy type 1 (DM1) experienced a reduction of…
Articles by Andrea Lobo
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…
Long-term treatment with AOC 1001 continues to be safe and may reverse the progression of myotonic dystrophy type…
SAT-3247, an oral therapy candidate for rebuilding muscle tissue, improved muscle function in a mouse model of facioscapulohumeral muscular…
Epic Bio’s epigenetic therapy EPI-321 has been granted an orphan drug designation by the U.S. Food and Drug Administration…
Fulcrum Therapeutics has completed patient enrollment for its Phase 3 clinical trial evaluating losmapimod as a potential treatment…
More than 420 “Fill the Boot” events will raise money throughout Labor Day weekend to advance research and care…