Spinal Muscular Atrophy
Novartis Prepares to File Intrathecal Zolgensma for Older SMA Patients by Mid-2025
Zolgensma, intrathecal delivery, spinal muscular atrophy, gene therapy, older patients
FDA Approves Evrysdi Tablets: A Breakthrough in Convenient SMA Treatment
Evrysdi, risdiplam, spinal muscular atrophy (SMA), FDA approval, tablet formulation, oral treatment, Genentech, Roche
Novartis Achieves Pivotal Trial Success for Intrathecal Zolgensma in Older SMA Patients After FDA Delay
Novartis, Zolgensma, Intrathecal formulation, Spinal muscular atrophy (SMA), FDA approval, Pivotal phase 3 trial, Older SMA patients
Biohaven’s Taldefgrobep Alfa Shifts Focus to Obesity After Missing Primary Endpoint in Spinal Muscular Atrophy Phase 3 Trial
Biohaven, taldefgrobep alfa, spinal muscular atrophy (SMA), obesity, myostatin inhibitor, phase 3 trial, body composition, lean muscle mass, bone density.
Biohaven’s Taldefgrobep Alfa Misses Primary Endpoint in Phase 3 SMA Trial, Eyes FDA Discussions and Obesity Indication
Biohaven, Taldefgrobep Alfa, Spinal Muscular Atrophy (SMA), Phase 3 Trial, FDA Discussions, Obesity Indication
Biogen’s Higher Dose Spinraza Shows Enhanced Efficacy in Treating Spinal Muscular Atrophy
Biogen, Spinraza, Nusinersen, Spinal Muscular Atrophy (SMA), Higher Dose Regimen, Clinical Trial Results, DEVOTE Study, Motor Function Improvement, Neurodegeneration Reduction
Scholar Rock Seeks $275M Funding Following Successful SMA Therapy Trial
Scholar Rock, funding, SMA therapy, clinical trial, Vincerx, pipeline reshuffle
Scholar Rock’s Stock Skyrockets 300% Following Successful Phase III Trial of Apitegromab for Spinal Muscular Atrophy
Scholar Rock, Spinal Muscular Atrophy (SMA), Apitegromab, Phase III Trial, Stock Surge, Biologics License Application (BLA), Marketing Authorisation Application (MAA)
Scholar Rock’s Apitegromab Shows Significant Improvement in Spinal Muscular Atrophy Treatment
Spinal Muscular Atrophy (SMA), Apitegromab, Phase 3 Clinical Trial, Motor Function Improvement, Myostatin Inhibition
Voyager Therapeutics Secures $100M Upfront Payment in Expanded Gene Therapy Partnership with Novartis
Gene therapy, Novartis, Voyager Therapeutics, Huntington’s disease, Spinal muscular atrophy (SMA), TRACER capsid discovery platform, Biotechnology collaboration