Spinal Muscular Atrophy
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Sign up freeTop drugs studied for this disease
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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 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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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 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 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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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 …
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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 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 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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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 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 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 …