Despite significant unmet need, there are currently no disease-modifying therapies available for the treatment of STXBP1 encephalopathy, although there are at least six assets in preclinical development, according to findings [...]

U.S. physicians report that the majority of their patients on Teva Pharmaceuticals’ Austedo and Neurocrine Biosciences’ Ingrezza achieve some degree of symptom relief, according to findings from REACH Market Research.   [...]

LQTS types 2 and 3 have the highest need for novel pharmacologic treatments of all subtypes, according to findings from REACH Market Research. NEWTON, Mass., Nov. 7, 2023 /PRNewswire/ -- Long QT syndrome [...]

~70% of physicians surveyed by REACH Market Research report there is an extremely high unmet medical need for novel treatments for ACM. NEWTON, Mass., Nov. 7, 2023 /PRNewswire/ -- Arrhythmogenic Cardiomyopathy (ACM) is [...]

Ultragenyx's AAV8 vector gene therapy, DTX401, is advancing through clinical trials as potentially the first ever therapy to treat Glycogen Storage Disease Type 1a (GSD-1a). NEWTON, Mass., Sept. 28, 2023 /PRNewswire/ -- [...]

Less than half of Graves' Disease patients achieve remission with anti-thyroid drugs, and nearly half of those that do will relapse. NEWTON, Mass., Aug. 16, 2023  /PRNewswire/ - Graves' Disease (GD) is [...]

Apellis's anti-C3 Syfovre (pegcetacoplan intravitreal injection) and Astellas' anti-C5 Izervay (avacincaptad pegol intravitreal injection) were both recently FDA-approved to treat Geographic Atrophy (GA), but physicians are eager for more treatment [...]