by Madaline Spencer | Sep 17, 2026
Carol Roberts, President of The PBCers Organization, discusses results from the Voice of PBC patient survey. PBC is a chronic, progressive autoimmune liver disease in which the bile ducts become inflamed and damaged. This leads to the buildup of bile...
by Madaline Spencer and Scott Harwood | Sep 16, 2026
Karen A. Jones, EdD, President and CEO of wAIHA Warriors, discusses the approval of Imaavy (nipocalimab) and its impact on patients with warm autoimmune hemolytic anemia (wAIHA). wAIHA is a rare hematologic autoimmune condition characterized by antibodies that...
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...
