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 ...
Levodopa is the main treatment for symptoms of Parkinson’s disease. Determining whether levodopa also has a disease-modifying effect could provide guidance as to when in the course of the disease the ...
Discusses MissionEB Phase III Trial and CORDStrom as a Disease-Modifying Therapy for RDEB February 26, 2026 1:00 PM ...
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