by Madaline Spencer | May 4, 2026
Tuan Vu, MD, Professor, Department of Neurology at the University of South Florida, discusses positive results of cemdisiran in patients with generalized myasthenia gravis (gMG). MG is a chronic autoimmune neuromuscular disease characterized by weakness...
by Madaline Spencer | Apr 30, 2026
Dareen D. Siri, MD, FAAAAI, FACAAI is a board-certified allergist and immunologist practicing at Midwest Allergy Sinus Asthma, based in central Illinois. She talked with CheckRare about a unique, rare disorder called systemic mastocytosis. Systemic...
by Madaline Spencer | Apr 29, 2026
The US Food and Drug Administration (FDA) has granted accelerated approval for Otarmeni (lunsotogene parvec) for the treatment of patients with otoferlin (OTOF)-related hearing loss. The indication includes pediatric and adult patients with severe-to-profound and...
by Madaline Spencer | Apr 29, 2026
A study published in the Journal of Clinical Medicine evaluated the improvement of bone mineral density in patients with type 1 Gaucher disease (GD1) treated with velaglucerase alfa. GD1 is the most common form of Gaucher disease, characterized by a deficiency of...
by Madaline Spencer | Apr 28, 2026
Arthur Beisang, MD, Department of Pediatrics at Gillette Children’s Specialty Healthcare in Saint Paul, Minnesota, discusses Daybue (trofinetide) Stix, a new formulation of the treatment for Rett syndrome. Rett syndrome is a neurodevelopmental condition...
