Clinical and pharmacokinetic evidence supported approval of zilganersen across pediatric and adult age groups, providing the first therapy designed to reduce production of the abnormal GFAP protein...
The FDA has approved Avlayah (tividenofusp alfa-eknm) for certain pediatric patients with Hunter syndrome, marking the first approval targeting the disease's neurologic complications and offering clinicians...
The FDA initiated approval of leucovorin calcium tablets to treat cerebral folate deficiency (CFD) following a systematic analysis demonstrating promise in this patient population.
The FDA approved the first treatment for patients with congenital thrombotic thrombocytopenic purpura (cTTP), which is a rare and life-threatening blood clotting disorder that may be fatal if left...
Following two 12-week studies, the FDA has approved the first ever medication for treating agitation in patients with dementia due to Alzheimer disease.
The FDA has approved a treatment for Friedreich ataxia after the results of randomized, placebo-controlled study indicate a strong effectiveness in treatment.
