Systemic lupus erythematosus (SLE) is a heterogeneous autoimmune disorder marked by unpredictable organ involvement, clinical variability, and a high burden of chronic therapy. For biopharma teams evaluating opportunity in [...]
Why therapeutic window, and patient segmentation will become increasingly important in Angelman syndrome The Angelman syndrome (AS) treatment landscape is on the verge of a fundamental shift from symptomatic [...]
Gene therapies, shifting diagnosis paradigms, and a new calculus for patient eligibility are changing how companies must approach Huntington’s disease Huntington’s disease (HD) has long been recognized for its [...]
How multisystem involvement, patient heterogeneity, and new modalities are redefining the DM1 opportunity for drug developers Myotonic dystrophy type 1 (DM1) stands apart on the rare neuromuscular landscape as [...]
What evolving risk definitions, shifting treatment expectations, and B cell immunomodulation mean for drug developers IgA nephropathy (IgAN) is no longer a niche renal indication defined by slow progress and [...]
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/ -- [...]