Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS research · 2026-03-23Clinical Trial Update: HABIT-ILE + FST in Children With SMA: Preliminary Effectiveness [NA]
Clinical trial update for HABIT-ILE plus FST in children with spinal muscular atrophy, noting preliminary effectiveness and current recruitm…
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: A Real-World Study of Clinical Use of Available Treatments for Pediatric Patients With Spinal Muscular Atrophy
Completed real-world study conducted by Novartis evaluating the use of available treatments in pediatric spinal muscular atrophy patients.
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CLINICAL_TRIALS trial_result · 2026-03-18Clinical Trial Update: ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM) [Phase 4]
A U.S. pragmatic Phase 4 study (STREAM) is evaluating real-world safety and effectiveness of intrathecal onasemnogene abeparvovec-brve (OAV1…
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CLINICAL_TRIALS research · 2026-03-18Clinical Trial Update: A Study to Find Out How Nusinersen is Processed in the Body When Given Through the ThecaFlex DRx™ System in Adult and Pediatric Participants With Spinal Muscular Atrophy (PIERRE-PK) [Phase 1]
A Phase 1 clinical trial evaluating pharmacokinetics of nusinersen when delivered through the ThecaFlex DRx system in adults and pediatric p…
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CLINICAL_TRIALS research · 2026-03-18Clinical Trial Update: Gait and Bone Health in SMA
A recruiting clinical study assessing gait and bone health in ambulatory individuals with Spinal Muscular Atrophy Type 3.
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CLINICAL_TRIALS research · 2026-03-17Clinical Trial Update: Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease [NA]
A clinical trial is evaluating the effects of whole-body electrical muscle stimulation on adults with various neuromuscular diseases and is …
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CLINICAL_TRIALS trial_result · 2026-03-16Clinical Trial Update: Spinal Cord Stimulation in Spinal Muscular Atrophy [NA]
A completed clinical trial evaluating a spinal cord stimulator in patients with Spinal Muscular Atrophy (Types 3 and 4) has posted results a…
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CLINICAL_TRIALS research · 2026-03-16Clinical Trial Update: HABIT-ILE + FST in Children With SMA [NA]
This clinical trial evaluates HABIT-ILE plus FST in children with spinal muscular atrophy and is currently active but not recruiting.
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CLINICAL_TRIALS research · 2026-03-13Clinical Trial Update: A Study to Investigate the Safety and Efficacy of RO7204239 in Combination With Risdiplam (RO7034067) in Participants With Spinal Muscular Atrophy [Phase 2, Phase 3]
A Phase 2/Phase 3 clinical trial is evaluating the safety and efficacy of RO7204239 in combination with risdiplam (RO7034067) for spinal mus…
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EMA approval · 2026-03-11EMA Authorised: Spinraza (nusinersen sodium) — Biogen Netherlands B.V.
The European Medicines Agency authorised Spinraza (nusinersen) by Biogen Netherlands B.V. for spinal muscular atrophy, with orphan medicine …
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CLINICAL_TRIALS research · 2026-03-09Clinical Trial Update: A Study to Learn How Nusinersen (Spinraza) Affects Participants With Spinal Muscular Atrophy (SMA) Who Took it Before or During Pregnancy And About The Health of Their Babies
This clinical trial investigates nusinersen (Spinraza) exposure before or during pregnancy in people with spinal muscular atrophy and assess…
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CLINICAL_TRIALS research · 2026-03-03Clinical Trial Update: A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Onasemnogene Abeparvovec [Phase 3]
Phase 3 clinical trial evaluating the safety and effects of Salanersen (BIIB115) in babies with spinal muscular atrophy who were previously …
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CLINICAL_TRIALS research · 2026-03-02Clinical Trial Update: Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 [Phase 1, Phase 2]
A Phase 1/2 clinical trial is evaluating the safety, tolerability, and efficacy of GB221 in infants with Spinal Muscular Atrophy Type I; the…
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CLINICAL_TRIALS trial_result · 2026-02-23Clinical Trial Update: Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration
A completed clinical trial studying neuromuscular junction defects in spinal muscular atrophy following restoration of SMN protein in centra…
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CLINICAL_TRIALS trial_result · 2026-02-23Clinical Trial Update: Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA)
This clinical trial update reports the completion of a study assessing Motor Unit Number Estimation (MUNE) in adults with Spinal Muscular At…
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CLINICAL_TRIALS research · 2026-02-18Clinical 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 assesses the long-term safety of higher doses of nusinersen administered by injections in spinal muscular atrophy pat…
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CLINICAL_TRIALS trial_result · 2026-02-11Clinical Trial Update: A Real-world Study of Disease-modifying Therapy Treatment Outcomes in Patients With Spinal Muscular Atrophy
This completed real-world clinical trial evaluates outcomes of disease-modifying therapy for patients with spinal muscular atrophy.
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FDA approval · 2026-02-10FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
The FDA approved a supplemental application for Evrysdi (risdiplam) submitted by Genentech, Inc., under NDA 213535.
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CLINICAL_TRIALS research · 2026-02-05Clinical Trial Update: Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA
A new clinical trial is recruiting to characterize perceived physical fatigability in individuals with spinal muscular atrophy treated with …
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CLINICAL_TRIALS research · 2026-02-04Clinical Trial Update: A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy [Phase 3]
This is an update on a Phase 3 trial evaluating Taldefgrobep Alfa for Spinal Muscular Atrophy, with the study currently active and not recru…
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CLINICAL_TRIALS research · 2026-01-30Clinical Trial Update: Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy [NA]
This clinical trial investigates new phenotypes among Spinal Muscular Atrophy patients treated with SMN-restoring therapy. The study is curr…
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CLINICAL_TRIALS trial_result · 2026-01-30Clinical Trial Update: A Study of Dosing Patterns and Costs in Patients With Spinal Muscular Atrophy Receiving Disease Modifying Therapies
A completed clinical trial examining dosing patterns and costs in spinal muscular atrophy patients receiving disease-modifying therapies.
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CLINICAL_TRIALS trial_result · 2026-01-22Clinical Trial Update: Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam [Phase 3]
Phase 3 trial update evaluating the efficacy and safety of Apitegromab in patients with later-onset spinal muscular atrophy who are treated …
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CLINICAL_TRIALS research · 2026-01-22Clinical Trial Update: Self-Efficacy Enhancement Using a Multicomponent Support Group for Caregivers of Children With DMD/SMA [NA]
This clinical trial update describes a study to enhance self-efficacy in caregivers of children with Duchenne Muscular Dystrophy and Spinal …
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FDA approval · 2026-01-21FDA Approved: EVRYSDI — Supplemental Application (Genentech, Inc.)
The FDA approved a supplemental application for Evrysdi (risdiplam) from Genentech, Inc.