Author name: Zach Galati

New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy

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Below is an excerpt from a Scholar Rock press release: Scholar Rock, a Phase 3, clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced new data from the Phase 2 TOPAZ trial extension period evaluating patient outcomes at 36 months of treatment with […]

New 36-Month Apitegromab Extension Data Reinforce Long-Term Substantial and Sustained Improvement of Motor Function in Phase 2 TOPAZ Trial Patients with Nonambulatory Spinal Muscular Atrophy Read More »

Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA

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• Alcyone’s ThecaFlex DRx™ System is an implantable medical device in development to enable routine subcutaneous administration of therapeutics to the cerebrospinal fluid• First stage of the pivotal study expected to initiate in summer of 2023 for first 10 patients followed by enrollment of the remaining 80 patients in a second stage beginning in 2024

Alcyone Therapeutics Receives FDA IDE Approval to Initiate Clinical Study of the ThecaFlex DRx™ System for Administration of SPINRAZA Read More »

Congressional Committee Approves Legislation to Improve Air Travel for People with SMA and other Disabilities

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This week, a key committee in the U.S. House of Representatives unanimously approved (63-0) aviation legislation that will make air travel more accessible for individuals with SMA and other disabilities. Accessible air travel is a top priority for Cure SMA and the SMA community, which were recognized by the U.S. House committee for their advocacy and support of the legislation.  

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Erin Trainor Memorial Fund will Match Donations for the funding of Care Centers

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Double your donation to help double Cure SMA’s Care Center Network Care Centers guide our understanding of how SMA is changing, help us understand how care is being delivered, and help identify best practices in SMA treatment. We currently have nearly 30 Care Centers across the U.S., and with your gift and the generous support

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