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 [...]