The Disease-Modifying Era in Angelman Syndrome
MARKETVUE® REPORT: More than half of STXBP1 Encephalopathy patients experience refractory seizures with currently available antiepileptics
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 [...]
MARKETVUE® REPORT: Over 80% of Tardive Dyskinesia patients are currently prescribed Austedo or Ingrezza, according to U.S. Neurologists
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. [...]
MARKETVUE® REPORT: Surgery remains the only option for the ~50% of LQTS patients refractory or intolerant to beta blockers
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 [...]
MARKETVUE® REPORT: Arrhythmogenic Cardiomyopathy Gene Therapy Research Gains Momentum
~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 [...]
MARKETVUE® REPORT: Gene therapies for treatment of Glycogen Storage Disease Type 1a offer hope for overburdened patients
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/ -- [...]
Graves’ Disease patients face limited treatment options that do not irreversibly destroy the thyroid (MARKETVUE® REPORT)
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 [...]
Ophthalmologists estimate 10% of Geographic Atrophy patients are now treated with Syfovre since its February 2023 approval (MARKETVUE® REPORT)
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 [...]