emflaza
(deflazacort)PTC Therapeutics, Inc.
Usage: EMFLAZA is indicated for treating Duchenne muscular dystrophy (DMD) in patients aged 2 years and older.
PTC Therapeutics, Inc. drugs
2 results
kebilidi
(eladocagene exuparvovec-tneq)PTC Therapeutics, Inc.Usage: KEBILIDI (eladocagene exuparvovec-tneq) is gene therapy for adults and pediatric patients with aromatic L-amino acid decarboxylase (AADC) deficiency, approved under accelerated approval based on motor milestone improvements at 48 weeks. Continued approval may depend on further clinical trial outcomes.