Significant unmet needs in SMA management include therapy access, fatigue, reproductive health, and emerging phenotypes in older patients. Intrathecal gene therapy offers potential for older SMA ...
Discusses MissionEB Phase III Trial and CORDStrom as a Disease-Modifying Therapy for RDEB February 26, 2026 1:00 PM ...
Currently, there is no cure for amyotrophic lateral sclerosis (ALS) and the foundation of ALS management revolves around symptomatic and palliative care. Early diagnosis offers the best prognosis for ...
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