In boys with Duchenne muscular dystrophy (DMD), executive skills — essentially the brain’s self-management tools, such as self-control, emotional…
Articles by Patricia Inácio, PhD
Satellos Bioscience asked the U.S. Food and Drug Administration (FDA) for clearance to conduct a Phase 2 clinical trial testing…
The U.S. Food and Drug Administration (FDA) has granted breakthrough therapy designation to Dyne Therapeutics’ DYNE-251 for the treatment…
People with myotonic dystrophy (DM) use more healthcare resources and face higher associated costs than patients without the disease,…
The progression of myotonic dystrophy type 1, known as DM1, differs according to sex and age at symptom onset,…
Losmapimod, an investigational medication from Fulcrum Therapeutics, failed to significantly outperform a placebo at improving upper limb function…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to RAG-18, being developed as a potential…
Satellos Bioscience has established a clinical advisory board, with experts in drug development and genetic muscle disorders, to help…
Patients and experts in limb-girdle muscular dystrophy (LGMD), along with drug developers, community leaders, and regulatory agency personnel, came…
The U.S. Food and Drug Administration (FDA) has given orphan drug designation to JUV-161, Juvena Therapeutics’ lead treatment candidate for…