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...
by Madaline Spencer | Sep 7, 2026
Christine Gustafson, Executive Director and CEO of the TED Community Organization, discusses the new Graves’ Community website. Graves’ disease is an autoimmune disease characterized by generalized overactivity of the thyroid gland. Thyroid eye disease...
