by Madaline Spencer | Sep 16, 2026
The US Food and Drug Administration (FDA) has approved Isembyld (apitegromab-mstn) for the treatment of spinal muscular atrophy (SMA) in patients ages 2 years and older who are currently receiving a survival motor neuron 2 (SMN2)-targeted treatment. SMA is a genetic...
by Madaline Spencer | Sep 11, 2026
Tushar Tangsali, father to two sons with Duchenne muscular dystrophy (DMD), discusses how he’s working to develop a gene therapy for a unique genetic variation of DMD for his sons. DMD is a rare genetic, neuromuscular condition characterized by...
by Madaline Spencer | Sep 10, 2026
Amélie Lothe, PhD, Head of Global Medical Community of Rare Epilepsies at UCB, discusses results from a post hoc analysis of trajectories of Fintepla (fenfluramine) effectiveness and safety in patients with Lennox-Gastaut syndrome (LGS). LGS is a...
by Madaline Spencer | Sep 9, 2026
The US Food and Drug Administration (FDA) has approved Zanvastro (zilganersen) for the treatment of pediatric and adult patients with Alexander disease. Alexander disease is a rare leukodystrophy that impairs neuronal signalling. Most cases of Alexander disease begin...
by Madaline Spencer | Sep 8, 2026
Neil Inhaber, MD, FRCPC, Therapeutic Area Head of Global Medical, Rare Disease at Takeda, discusses data on maribavir in post-transplant cytomegalovirus (CMV) presented at the American Transplant Congress 2026. Post-transplant CMV is one of the most common...
