December 2024

Novartis Intrathecal Gene Therapy Onasemnogene Abeparvovec Phase III Study Meets Primary Endpoint in Children and Young Adults with SMA

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Novartis today announced positive topline results from the Phase III STEER study. This pivotal study assessed the efficacy and safety of investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in treatment-naïve patients with spinal muscular atrophy (SMA) Type 2, aged two to less than 18 years who are able to sit but have never walked independently. Efficacy and […]

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Cure SMA Celebrates New DOT Rule for Accessible Air Travel

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Cure SMA is thrilled to share a major win for the SMA community: the U.S. Department of Transportation (DOT) has finalized its rule on ensuring safe accommodations for air travelers with disabilities using wheelchairs. This significant advancement underscores the importance of equity in air travel and represents a critical step toward making flights safer and

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