Panelists discuss how significant knowledge gaps among nonspecialist providers—particularly regarding differential diagnosis, interpretation of cognitive assessments, and awareness of treatment options—are being addressed through targeted educational initiatives, embedded clinical decision support tools, and collaborative care models to enhance understanding of early Alzheimer diagnosis and treatment importance.
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STEER Data Open Door to SMA Gene Therapy for Wider Age Range of Children
March 19th 2025Delivery of onasemnogene abeparvovec into the intrathecal space was safe and effective for children with spinal muscular atrophy (SMA) aged 2 to 17 years, who had previously been shut out of receiving gene therapy.
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Bustling Gene Therapy Pipeline for Neuromuscular Diseases Brings Thorny Questions to the Clinic
March 18th 2025The rapid development of gene therapy options for treating neuromuscular diseases has created new therapeutic options but also logistical hurdles and a need for complex discussions between clinicians and families.
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