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...
A 52-week trial evaluated apitegromab-mstn as an adjunct to SMN2-targeted treatment in patients with spinal muscular atrophy who could not move or walk independently, demonstrating improved motor function...
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 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...
The US Food and Drug Administration has approved a new option for the treatment of a specific form of Batten disease in pediatric patients aged 3 years and older.
