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Data on the RAISE Clinical Trial Program of Zilucoplan for the Treatment of Myasthenia Gravis

by Madaline Spencer | Nov 13, 2025

Michael Weiss, MD, Neurologist at the University of Washington, discusses data on the RAISE and RAISE-XT clinical trials of zilucoplan for the treatment of generalized myasthenia gravis (gMG).     gMG is a chronic autoimmune neuromuscular disease...

New Data on Vyvgart for Patients With AChR-Ab Seronegative Myasthenia Gravis

by Madaline Spencer | Nov 12, 2025

James Howard, Jr., MD, Professor of Neurology at the University of North Carolina, discusses new data on the safety and efficacy of Vyvgart (efgartigimod alfa) for patients with AChR-Ab seronegative generalized myasthenia gravis (gMG).   gMG is a chronic...

FDA Approves First Treatment for Thymidine Kinase 2 Deficiency

by Madaline Spencer | Nov 10, 2025

The U.S. Food and Drug Administration (FDA) has approved Kygevvi for the treatment of adults and pediatric patients, with an age of symptom onset on or before 12 years, with thymidine kinase 2 deficiency (TK2d). TK2d is a rare genetic disease characterized by...

A Patient’s Diagnostic Journey With Systemic Mastocytosis

by Madaline Spencer | Nov 7, 2025

Joan Smith, a patient with systemic mastocytosis, discusses her diagnostic journey with systemic mastocytosis (SM).   SM  is a rare disease, usually caused by mutations in the KIT D816V gene. The disorder is characterized by uncontrolled mast cell proliferation...

Results From the DeFi Clinical Trial of Nirogacestat in Patients With Desmoid Tumors

by Madaline Spencer | Nov 6, 2025

Bernd Kasper, MD, PhD, University of Heidelberg Cancer Center, discusses results from the DeFi clinical trial of nirogacestat in patients with desmoid tumors.     Desmoid tumors are abnormal growths that arise from connective tissue. The tumors can occur...
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