Spinal Muscular Atrophy

Biogen Ushers Spinraza Follow-Up (Salanersen) Into Pivotal SMA Studies

6 days ago talkbio0Tagged Antistreptolysin O, Biogen, Clinical Trials, motor milestones, Nerve Degeneration, Novartis, Pediatric brand name, Phase III, salanersen, Spinal Muscular Atrophy, Spinraza, Zolgensma

Biogen; SMA; Spinraza; salanersen; Phase III; clinical trials; neurodegeneration; motor milestones; pediatric; Novartis Zolgensma; ASO therapy

Scholar Rock’s SMA drug helps Zepbound patients retain muscle in phase 2 trial

2 weeks ago talkbio0Tagged apitegromab, GDF8 protein, human, Inhibitor, Lean Mass, muscle preservation, Obesity, Phase 2 trial, Scholar Rock, Spinal Muscular Atrophy, Weight Loss, Zepbound

Scholar Rock; apitegromab; SMA drug; Zepbound; tirzepatide; muscle preservation; obesity; weight loss; phase 2 trial; lean mass; myostatin inhibitor

Novartis Prepares to File Intrathecal Zolgensma for Older SMA Patients by Mid-2025

3 months ago talkbio0Tagged gene therapy, intrathecal delivery, older, Spinal Muscular Atrophy, Zolgensma

Zolgensma, intrathecal delivery, spinal muscular atrophy, gene therapy, older patients

FDA Approves Evrysdi Tablets: A Breakthrough in Convenient SMA Treatment

5 months ago talkbio0Tagged Approved, Drug Formulation Process, Evrysdi, genentech, Oral treatment, Risdiplam, Roche (company), Spinal Muscular Atrophy, Tablet Dosage Form, United States Food and Drug Administration

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

6 months ago talkbio0Tagged Approved, intrathecal formulation, Novartis, older, Phase 3 trial, Spinal Muscular Atrophy, United States Food and Drug Administration, Zolgensma

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

7 months ago talkbio0Tagged Alfa, Body Composition, Bone and Bones, density, GDF8 protein, human, Inhibitor, lean, Obesity, Phase 3 trial, Spinal Muscular Atrophy

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

7 months ago talkbio0Tagged Discussion (procedure), Indication of (contextual qualifier), Obesity, Phase 3 trial, Spinal Muscular Atrophy, Talditercept alfa, United States Food and Drug Administration

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

9 months ago talkbio0Tagged Biogen, Clinical Trials, DEVOTE, Dose Regimen, High, Improvement, Motor function (observable entity), Nerve Degeneration, nusinersen, Spinal Muscular Atrophy, Spinraza

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

9 months ago talkbio0Tagged Clinical Trials, pipeline, reshuffle, Scholar Rock, SMA, Spinal Muscular Atrophy, Vincerx

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

9 months ago talkbio0Tagged apitegromab, Biologics License Application, MAA, Phase 3 Clinical Trials, Scholar Rock, SMA, Spinal Muscular Atrophy, Stock Surge

Scholar Rock, Spinal Muscular Atrophy (SMA), Apitegromab, Phase III Trial, Stock Surge, Biologics License Application (BLA), Marketing Authorisation Application (MAA)