by Madaline Spencer | Apr 15, 2026
Jennifer Ibrahim, Head of North America, Rare Disease Medical Affairs at Sanofi, discusses the Breakthrough Therapy designation of venglustat for the treatment of neurological manifestations in Gaucher disease type 3 (GD3). Gaucher disease refers to a...
by Madaline Spencer | Apr 14, 2026
Stevie Ringel, CEO of Nome Therapeutics, discusses the US Food and Drug Administration’s (FDA) Plausible Mechanism Framework and its effect on rare disease therapy development. On November 12, 2025, the US Food and Drug Administration (FDA) released the...
by Madaline Spencer | Apr 10, 2026
Systemic mastocytosis is a rare disease in which excess mast cells are produced and activated, resulting in chronic, severe, and heterogenous symptoms. CheckRare spoke with Patrick C. Foy, MD, a Clinical Hematologist practicing at Froedtert Hospital, Milwaukee, and...
by Madaline Spencer and Joe Haddad | Apr 9, 2026
The US Food and Drug Administration (FDA) has approved Tecartus (brexucabtagene autoleucel) for the treatment of adults with relapsed or refractory (R/R) mantle cell lymphoma (MCL). MCL is a rare form of malignant non-Hodgkin lymphoma affecting B lymphocytes in the...
by Madaline Spencer | Apr 7, 2026
Miquel Vila-Perello, PhD, co-founder and CEO of SpliceBio, discusses an investigational dual adeno-associated virus (AAV) gene therapy in development for patients with Stargardt disease. Stargardt disease is a genetic eye disorder characterized by...
