Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-08-21EMA Authorised: Zolgensma (onasemnogene abeparvovec) — Novartis Europharm Limited
EMA authorised Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy under conditional approval and orphan designation.
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EMA approval · 2026-08-20EMA Authorised: Zolgensma (onasemnogene abeparvovec) — Novartis Europharm Limited
The EMA has authorised Zolgensma (onasemnogene abeparvovec) from Novartis Europharm Limited with conditional approval for spinal muscular at…
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CLINICAL_TRIALS trial_result · 2026-08-11Clinical Trial Update: A Study to Learn About the Long-Term Safety of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Took Part in an Earlier Nusinersen Trial (ONWARD) [Phase 3]
A phase 3 trial update assessing long-term safety of higher doses of nusinersen (BIIB058) in SMA participants from a prior ONWARD trial.
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EMA approval · 2026-08-04EMA Authorised: Spinraza (nusinersen sodium) — Biogen Netherlands B.V.
EMA authorises Spinraza (nusinersen) for spinal muscular atrophy, granted orphan medicine status with accelerated assessment.
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EMA approval · 2026-06-15EMA Authorised: Zolgensma (onasemnogene abeparvovec) — Novartis Europharm Limited
The EMA authorises Zolgensma (onasemnogene abeparvovec) for spinal muscular atrophy under a conditional approval as an orphan medicine.
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CLINICAL_TRIALS research · 2026-04-24Clinical Trial Update: Adrenal& Bone Complications in Paediatric Patients Living With Spinal Muscular Atrophy (SMA)
A pediatric clinical trial investigating adrenal and bone complications in children with spinal muscular atrophy; currently NOT_YET_RECRUITI…
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
A clinical trial update describes development of non-invasive prenatal diagnosis for single-gene disorders. The study, based in Paris, is cu…
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CLINICAL_TRIALS trial_result · 2026-04-23Clinical Trial Update: Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback [NA]
A completed clinical trial assessed the feasibility of virtual reality in children with neuromuscular diseases and evaluated the effectivene…
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CLINICAL_TRIALS research · 2026-04-23Clinical Trial Update: Effects of Developmental Support for Mothers of Infants With Spinal Muscular Atrophy [NA]
A clinical trial update noting an ongoing study on developmental support for mothers of infants with spinal muscular atrophy, with status AC…
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CLINICAL_TRIALS research · 2026-04-22Clinical Trial Update: A Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of Intravenous Administration of ARGX-119 in Pediatric Participants Aged 5 to Less Than 18 Years With Spinal Muscular Atrophy [Phase 2]
This document reports a Phase 2 clinical trial evaluating ARGX-119 administered intravenously in children aged 5 to under 18 with Spinal Mus…
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EMA approval · 2026-04-21EMA Authorised: Evrysdi (risdiplam) — Roche Registration GmbH
The European Medicines Agency authorised Evrysdi (risdiplam) for spinal muscular atrophy with accelerated assessment. The decision date was …
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
This clinical trial update describes the Active-NBS study in the UK, evaluating an international federated model for wearable-derived remote…
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CLINICAL_TRIALS research · 2026-04-21Clinical Trial Update: A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years With Spinal Muscular Atrophy (SMA) Who Are Either New to SMA Treatment or Were Previously Treated With Risdiplam [Phase 3]
Phase 3 clinical trial evaluating Salanersen's (BIIB115) effects on movement and safety in people aged 15 to 60 with spinal muscular atrophy…
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CLINICAL_TRIALS research · 2026-04-20Clinical Trial Update: Invasive Home Ventilation in Denmark
A Danish clinical trial sponsored by Rigshospitalet is studying invasive home ventilation for patients with neuromuscular diseases, includin…
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CLINICAL_TRIALS trial_result · 2026-04-20Clinical Trial Update: Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) [Phase 4]
Phase 4 long-term follow-up study of risdiplam in Spinal Muscular Atrophy; the trial is currently active but not recruiting.
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CLINICAL_TRIALS trial_result · 2026-04-16Clinical Trial Update: Gut Peptides and Bone Remodeling in Children With Neuromuscular Disorders [NA]
A terminated clinical trial update investigating gut peptides and bone remodeling in children with neuromuscular disorders, sponsored by the…
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CLINICAL_TRIALS research · 2026-04-16Clinical Trial Update: Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy [NA]
This clinical trial update indicates ongoing recruitment for a study of an intrathecal port and catheter system in people with spinal muscul…
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CLINICAL_TRIALS trial_result · 2026-04-15Clinical Trial Update: A Study of Risdiplam in Participants With Type I and Type II Spinal Muscle Atrophy (SMA)
A Roche-sponsored clinical trial evaluating risdiplam for spinal muscular atrophy types I and II is listed as NOT_YET_RECRUITING.
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CLINICAL_TRIALS trial_result · 2026-04-13Clinical Trial Update: Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy [Phase 2]
Phase 2 study evaluating the safety and efficacy of NMD670 in ambulatory adult patients with Type 3 spinal muscular atrophy; the trial is sp…
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CLINICAL_TRIALS research · 2026-04-13Clinical Trial Update: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered in Pediatric Patients With Spinal Muscular Atrophy Who Experienced a Plateau or Decline in Function After Gene Therapy [Phase 4]
A Phase 4 trial is evaluating the effectiveness and safety of risdiplam in pediatric spinal muscular atrophy patients who experienced a plat…
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CLINICAL_TRIALS research · 2026-04-09Clinical Trial Update: Establishing Walking-related Digital Biomarkers in Rare Childhood Onset Progressive Neuromuscular Disorders
A clinical trial update describing a study to establish walking-related digital biomarkers in rare childhood-onset progressive neuromuscular…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy [Phase 4]
A Phase 4 clinical trial is evaluating whether risdiplam provides effective and safe benefits when given as an early intervention after gene…
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CLINICAL_TRIALS research · 2026-04-03Clinical Trial Update: A Study to Investigate the Pharmacokinetics and Safety of Risdiplam in Infants With Spinal Muscular Atrophy [Phase 2]
Phase 2 study evaluating pharmacokinetics and safety of risdiplam in infants with spinal muscular atrophy, currently recruiting.
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FDA approval · 2026-03-27FDA Approved: SPINRAZA — Supplemental Application (Biogen Inc.)
The FDA approved Biogen's supplemental application for Spinraza (nusinersen). This approval updates the drug's labeling and/or indication.
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CLINICAL_TRIALS research · 2026-03-25Clinical Trial Update: A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular Atrophy [Phase 2]
A Phase 2 clinical trial is evaluating how apitegromab works in subjects under 2 years old with spinal muscular atrophy (SMA). The study is …