Gene therapy can effectively treat various diseases, but for some debilitating conditions like muscular dystrophies there is ...
has partnered with MDA to support individuals and families affected by muscular dystrophy. The SAFD is proud to continue this ...
According to the Mayo Clinic, muscular dystrophy is a group of diseases that cause loss of muscle mass and progressive ...
"For Duchenne muscular dystrophy, a devastating genetic disorder affecting young boys, securing both Orphan Drug and Rare Pediatric Disease Designations for Ifetroban from the FDA is a critical ...
[3] National Institutes of Health — National Center of Advancing Translational Sciences: Genetic and Rare Diseases Information Center. Limb-Girdle Muscular Dystrophy Type 2I – Summary.
AskBio Receives FDA Rare Pediatric Disease and Orphan-Drug Designations for AB-1003 for the Treatment of Limb-Girdle Muscular Dystrophy Type 2I/R9 Back to video Asklepios BioPharmaceutical, Inc.