by Madaline Spencer | Dec 2, 2025
Michael A. Levine, MD, ML, Children’s Hospital of Philadelphia, discusses positive topline results from the CALIBRATE trial of encaleret in patients with autosomal dominant hypocalcemia type 1 (ADH1). ADH1 is a rare form of hypoparathyroidism caused by...
by Madaline Spencer | Dec 1, 2025
Nolan Campbell, PhD, U.S. Medical Director at Johnson & Johnson, discusses the EPIC clinical trial design that will evaluate the efficacy of nipocalimab versus efgartigimod in patients with generalized myasthenia gravis (gMG). gMG is a chronic...
by Madaline Spencer | Nov 26, 2025
Sindhu Ramchandren, MD, Executive Medical Director of Neuroscience at Johnson & Johnson, discusses long-term efficacy of nipocalimab in patients with generalized myasthenia gravis (gMG). gMG is a chronic autoimmune neuromuscular disease characterized...
by Madaline Spencer | Nov 25, 2025
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib) for the treatment of adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN). Selumetinib was first approved in 2020 for pediatric...
by Madaline Spencer | Nov 24, 2025
The U.S. Food and Drug Administration (FDA) has updated the prescribing information for Elevidys (delandistrogene moxeparvovec) for Duchenne muscular dystrophy (DMD). DMD is a rare genetic, neuromuscular condition characterized by progressive muscle wasting. Symptoms...
