| Not Yet Recruiting | ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathec NCT07448610 | Novartis Pharmaceuticals | Phase 4 |
| Not Yet Recruiting | A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 NCT07444476 | Biogen | Phase 3 |
| Recruiting | Effects of Whole-body Electrical Muscle Stimulation Exercise on Adults With Neuromuscular Disease NCT07478172 | University of Missouri-Columbia | N/A |
| Recruiting | Exploring the Physiologic, Pharmacodynamic, and Clinical Responses of Skeletal Muscle in Patients With Spinal NCT06532474 | St. Jude Children's Research Hospital | — |
| Recruiting | A Study to Evaluate How Apitegromab Works in Subjects Who Are Less Than 2 Years Old and Have Spinal Muscular A NCT07047144 | Scholar Rock, Inc. | Phase 2 |
| Recruiting | Characterizing Perceived Physical Fatigability in Nusinersen-treated SMA NCT06955897 | Columbia University | — |
| Enrolling By Invitation | A Long-term Follow-up Study Evaluating Intravenous Injection of EXG001-307 in Patients With Type 1 SMA NCT06588803 | Guangzhou Jiayin Biotech Ltd | — |
| Recruiting | A Registry Based Randomized-Controlled Trial of an Upper Limb Exergaming Intervention for Children and Adolesc NCT06396325 | Maryam Oskoui, MD, MSc, FRCPC, FAAN | N/A |
| Recruiting | An Open Label Study of Gene Therapy Product (Vesemnogene Lantuparvovec) in Spinal Muscular Atrophy NCT06288230 | Lantu Biopharma | Phase 1 / Phase 2 |
| Enrolling By Invitation | Development and Validation of the FBIndex to Determine the Risk of Falls for Patients With Neuromuscular Disor NCT06605612 | LMU Klinikum | — |
| Recruiting | Characterization of New Phenotypes of Patients With Spinal Muscular Atrophy Treated With SMN Restoring Therapy NCT06321965 | Hospices Civils de Lyon | N/A |
| Active Not Recruiting | Acceptability, Feasibility, Safety and Efficacy of a Optimized Rehabilitation Program for Treated Patients Wit NCT06419322 | University of Oxford | N/A |
| Active Not Recruiting | Spinal Cord Stimulation for the Treatment of Motor Deficits in People With Spinal Muscular Atrophy - Upper Lim NCT06300996 | Marco Capogrosso | N/A |
| Recruiting | Respiratory Functions, Thoracoabdominal Movements and Exercise Capacity in Neuromuscular Diseases NCT06756633 | Lokman Hekim University | — |
| Recruiting | High-intensity Interval Training in Patients With Spinal Muscular Atrophy NCT06368076 | Rigshospitalet, Denmark | N/A |
| Recruiting | A Review of the Management and Outcomes of Children With SMA in the West Midlands During 2017-2022 NCT05994950 | Robert Jones and Agnes Hunt Orthopaedic and District NHS Trust | — |
| Completed | Safety and Usability of a Robotic Gait Device for Children and Adolescents With Neurological or Neuromuscular NCT06167954 | MarsiBionics | N/A |
| Recruiting | Study of an Intrathecal Port and Catheter System for Subjects With Spinal Muscular Atrophy NCT05866419 | Alcyone Therapeutics, Inc | N/A |
| Recruiting | Spinal Muscular Atrophy Neonatal Screening Program NCT06310421 | IRCCS Burlo Garofolo | — |
| Active Not Recruiting | Safety and Efficacy of NMD670 in Ambulatory Adult Patients With Type 3 Spinal Muscular Atrophy NCT05794139 | NMD Pharma A/S | Phase 2 |
| Unknown | Development of a Multidisciplinary Network for Clinical and Laboratory Research for SMA NCT05866939 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Unknown | Motor Function and Activity of Daily Life in Spinal Muscular Atrophy NCT05931211 | National Taiwan University Hospital | — |
| Active Not Recruiting | Long-Term Safety & Efficacy of Apitegromab in Patients With SMA Who Completed Previous Trials of Apitegromab NCT05626855 | Scholar Rock, Inc. | Phase 3 |
| Unknown | MAP THE SMA: a Machine-learning Based Algorithm to Predict THErapeutic Response in Spinal Muscular Atrophy NCT05769465 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Recruiting | Evaluation of Safety and Efficacy of Gene Therapy Drug in the Treatment of Spinal Muscular Atrophy (SMA) Type NCT05824169 | GeneCradle Inc | Phase 1 / Phase 2 |
| Completed | Exploratory Study of Respiratory Bacterial Infections or Superinfections and Colonizations in Patients With Sp NCT05712330 | Centre Hospitalier Universitaire, Amiens | N/A |
| Completed | Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administere NCT05386680 | Novartis Pharmaceuticals | Phase 3 |
| Completed | Ultrasound-assisted or Landmark-based Intrathecal Administration of Nusinersen in Adult Patients With Spinal M NCT05644899 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Completed | Mechanisms and Treatment of Exercise Intolerance and Persistent Fatigue in Spinal Muscular Atrophy NCT05518773 | Columbia University | — |
| Recruiting | Long Term Trajectories of SMA Patients Receiving or Not Disease-modifying Treatments NCT05768048 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Active Not Recruiting | Risdiplam in Patients With Spinal Muscular Atrophy Previously Treated With Nusinersen NCT05522361 | Clinic for Special Children | Phase 4 |
| Completed | Quality of Life and Participation of the Adult with Spinal Muscular Atrophy in France NCT05366465 | Hospices Civils de Lyon | — |
| Unknown | Spinal Muscular Atrophy on Wheels, Using Power Mobility NCT05589987 | University of Castilla-La Mancha | N/A |
| Unknown | Reliability and Validity of the Turkish Version of the PedsQL 3.0 Neuromuscular Module for 2-to 4- Year-old NCT05539456 | Istanbul Medipol University Hospital | — |
| Unknown | Myotonometer Intra- and Inter Examiner Reliability on Spinal Muscular Atrophy NCT05521217 | Istanbul Medipol University Hospital | — |
| Unknown | Longitudinal Data Collection in Pediatric and Adult Patients With Spinal Muscular Atrophy in Latin America NCT05475691 | Hospital Israelita Albert Einstein | — |
| Active Not Recruiting | A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy NCT05337553 | Biohaven Pharmaceuticals, Inc. | Phase 3 |
| Unknown | Schulze Muscular Dystrophy Ability Clinical Study NCT05409079 | AbiliTech Medical Inc. | N/A |
| Completed | Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective NCT06178393 | Novartis Pharmaceuticals | — |
| Active Not Recruiting | COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis NCT05035823 | Synchron Medical, Inc. | N/A |
| Recruiting | Hippotherapy in Children With Spinal Muscular Atrophy NCT05341453 | Charles University, Czech Republic | N/A |
| Withdrawn | SWITCH II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis NCT05041114 | Synchron Medical, Inc. | N/A |
| Completed | Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinerse NCT05156320 | Scholar Rock, Inc. | Phase 3 |
| Active Not Recruiting | Long-term Follow-up Study of Risdiplam in Participants With Spinal Muscular Atrophy (SMA) NCT05232929 | Genentech, Inc. | Phase 4 |
| Completed | A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders NCT05109637 | Biogen | N/A |
| Completed | Implementation of FEES in Spinal Muscle Atrophy NCT04773470 | University of Giessen | N/A |
| Completed | Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) NCT04576494 | CHU de Reims | N/A |
| Active Not Recruiting | A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants NCT05067790 | Biogen | Phase 3 |
| Unknown | Antisense Oligonucleotide for Spinal Muscular Atrophy NCT05187260 | First Affiliated Hospital of Fujian Medical University | — |
| Recruiting | Outpatient Rehabilitation Intervention for Young Children With SMA NCT05638750 | Holland Bloorview Kids Rehabilitation Hospital | N/A |
| Active Not Recruiting | Pediatric Powered Wheelchair Standing Devices: An Exploratory Study NCT05117827 | Grand Valley State University | N/A |
| Unknown | Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices NCT05110885 | Bahçeşehir University | — |
| Completed | Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration NCT05219487 | Bakri Elsheikh | — |
| Completed | Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Rec NCT04335942 | Assistance Publique - Hôpitaux de Paris | N/A |
| Completed | Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) NCT04851873 | Novartis Pharmaceuticals | Phase 3 |
| Unknown | Personalized Medicine for SMA: a Translational Project NCT05779956 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Unknown | SMN Circular RNAs as Potential New Targets and Biomarkers for SMA NCT05760209 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Active Not Recruiting | Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients NCT04690998 | Assistance Publique Hopitaux De Marseille | N/A |
| Active Not Recruiting | Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies NCT04833348 | Assistance Publique - Hôpitaux de Paris | N/A |
| Unknown | Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant Women NCT04859179 | Mahidol University | — |
| Unknown | Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA). NCT05632666 | UMC Utrecht | N/A |
| Completed | Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen NCT04602195 | Hospices Civils de Lyon | — |
| Unknown | Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen NCT04644393 | Hospices Civils de Lyon | — |
| Completed | Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observationa NCT04404764 | Hospital Israelita Albert Einstein | — |
| Recruiting | CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain NCT07231549 | Fundació Sant Joan de Déu | — |
| Active Not Recruiting | NuroSleeve Powered Brace & Stimulation System to Restore Arm Function NCT04798378 | Thomas Jefferson University | N/A |
| Unknown | Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSO NCT04262570 | University of Erlangen-Nürnberg Medical School | N/A |
| Active Not Recruiting | Monitoring to the Evolution of Motor Function in SMA Type II Adults Patients Treated With SPINRAZA® NCT04159987 | Centre Hospitalier Universitaire de Nice | N/A |
| Recruiting | French Register of Patients With Spinal Muscular Atrophy NCT04177134 | Assistance Publique - Hôpitaux de Paris | — |
| Unknown | SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atroph NCT05761262 | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | — |
| Completed | Newborn Screening for Spinal Muscular Atrophy (SMA) - a Proof of Principle Study Using Anonymised Blood Spots. NCT05237544 | Great Ormond Street Hospital for Children NHS Foundation Trust | — |
| Completed | New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy NCT04115475 | University of Erlangen-Nürnberg Medical School | N/A |
| Withdrawn | Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Rec NCT04050852 | NYU Langone Health | EARLY_Phase 1 |
| Recruiting | A Registered Cohort Study on SMA NCT04010604 | Wan-Jin Chen | — |
| Completed | The Burden of Primary Caregivers of Spinal Muscular Atrophy Patients and Their Needs NCT04228718 | Marmara University | — |
| Completed | A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants NCT03988907 | Hoffmann-La Roche | Phase 1 |
| Completed | An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy NCT03921528 | Scholar Rock, Inc. | Phase 2 |
| Unknown | Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Bi NCT03689660 | Merve Kurt | N/A |
| Enrolling By Invitation | Early Check: Expanded Screening in Newborns NCT03655223 | RTI International | — |
| Completed | Adults With SMA Treated With Nusinersen NCT04591678 | Ohio State University | — |
| Active Not Recruiting | Spinraza in Adult Spinal Muscular Atrophy NCT03709784 | Washington University School of Medicine | — |
| Completed | Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA) NCT04139343 | Ohio State University | — |
| Active Not Recruiting | Holter of Movement in Patients with SMA Undergoing Treatment. NCT04888702 | Centre Hospitalier Universitaire de Liege | N/A |
| Completed | Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patien NCT03505099 | Novartis Gene Therapies | Phase 3 |
| Completed | Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium NCT03554343 | Centre Hospitalier Régional de la Citadelle | — |
| Completed | Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy NCT06027385 | Cystinose Stiftung | N/A |
| Terminated | Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy NCT03381729 | Novartis Gene Therapies | Phase 1 |
| Completed | European Registry of Patients With Infantile-onset Spinal Muscular Atrophy NCT03339830 | Institut de Myologie, France | — |
| Completed | Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy NCT03223051 | Centre d'Investigation Clinique et Technologique 805 | N/A |
| Recruiting | Neonatal Spinal Muscular Atrophy (SMA) Screening NCT03217578 | Kaohsiung Medical University Chung-Ho Memorial Hospital | — |
| Unknown | Natural History of Types 2 and 3 SMA in Taiwan NCT03300869 | Kaohsiung Medical University Chung-Ho Memorial Hospital | — |
| Completed | Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass NCT03970135 | Rigshospitalet, Denmark | N/A |
| Completed | A Study of Risdiplam (RO7034067) in Adult and Pediatric Participants With Spinal Muscular Atrophy NCT03032172 | Hoffmann-La Roche | Phase 2 |
| Completed | Metabolomics of Children With SMA NCT04587492 | University Medical Centre Ljubljana | — |
| Completed | Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy NCT03808233 | University of Texas Southwestern Medical Center | — |
| Withdrawn | Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients W NCT02235090 | Charitable Foundation Children with Spinal Muscular Atrophy | N/A |
| Unknown | Prospective Evaluation of Infants With Spinal Muscular Atrophy: NCT02831296 | Massachusetts General Hospital | — |
| Completed | A Study of CK-2127107 in Patients With Spinal Muscular Atrophy NCT02644668 | Cytokinetics | Phase 2 |
| Completed | A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067 NCT02633709 | Hoffmann-La Roche | Phase 1 |
| Terminated | Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene NCT02550691 | University Hospital, Rouen | N/A |
| Completed | Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions NCT02428673 | Gillette Children's Specialty Healthcare | N/A |
| Completed | SPACE Trial: Pyridostigmine vs Placebo in SMA Types 2, 3 and 4 NCT02941328 | UMC Utrecht | Phase 2 |
| Completed | A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 39 NCT02594124 | Biogen | Phase 3 |
| Recruiting | Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised D NCT05663008 | University of Dublin, Trinity College | — |
| Terminated | A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular NCT02462759 | Biogen | Phase 2 |
| Completed | Genetics of Pediatric-Onset Motor Neuron and Neuromuscular Diseases NCT02532244 | Nemours Children's Clinic | — |
| Completed | A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Pres NCT02386553 | Biogen | Phase 2 |
| Completed | An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) NCT02268552 | Novartis Pharmaceuticals | Phase 1 / Phase 2 |
| Completed | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal NCT02292537 | Biogen | Phase 3 |
| Terminated | A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy NCT02193074 | Biogen | Phase 3 |
| Completed | WREX Outcome Study NCT02218593 | Nemours Children's Clinic | — |
| Unknown | National Registry for Egyptian Pediatric Neuromuscular Diseases NCT02124616 | Ain Shams University | — |
| Completed | An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular A NCT02052791 | Biogen | Phase 1 |
| Unknown | Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA) NCT03520179 | Great Ormond Street Hospital for Children NHS Foundation Trust | — |
| Completed | Newborn Screening for Spinal Muscular Atrophy NCT02123186 | National Taiwan University Hospital | — |
| Unknown | Valproate and Levocarnitine in Children With Spinal Muscular Atrophy NCT01671384 | All India Institute of Medical Sciences | Phase 3 |
| Unknown | Reciprocating Gait Orthoses for Paraplegia Patients NCT02227407 | National Taiwan University Hospital | N/A |
| Completed | At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurolo NCT01931644 | Sanguine Biosciences | — |
| Completed | A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal NCT01839656 | Biogen | Phase 2 |
| Completed | An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular A NCT01780246 | Biogen | Phase 1 |
| Completed | An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) NCT01703988 | Biogen | Phase 1 / Phase 2 |
| Completed | Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients NCT01645787 | Columbia University | Phase 2 / Phase 3 |
| Terminated | Infants With Spinal Muscular Atrophy Type I NCT01547871 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| Completed | An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants W NCT01494701 | Biogen | Phase 1 |
| Completed | Flu Vaccine Study in Neuromuscular Patients 2011 NCT01422200 | Children's Hospital Medical Center, Cincinnati | Phase 4 |
| Completed | Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe) NCT01611610 | Institut de Myologie, France | N/A |
| Completed | Spinal Muscular Atrophy and Cardiac Autonomic Function NCT01522079 | Centro Universitário Augusto Motta | N/A |
| Completed | Optimum Insufflation Capacity in NMD NCT01981915 | Universität Duisburg-Essen | N/A |
| Completed | Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA) NCT01166022 | Columbia University | N/A |
| Completed | SMN Copy Number Distribution in Mali, West Africa NCT01059240 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| Completed | Motor Development and Orthoses in Spinal Muscular Atrophy (SMA) NCT00961103 | Azienda USL Reggio Emilia - IRCCS | — |
| Completed | Aerobic Training in Patients With Spinal Muscular Atrophy Type III NCT02003937 | Rigshospitalet, Denmark | N/A |
| Completed | Effects of Power Mobility on Young Children With Severe Motor Impairments NCT01028833 | University of Oklahoma | Phase 2 |
| Completed | A Pilot Study of Biomarkers for Spinal Muscular Atrophy NCT00756821 | Carelon Research | — |
| Recruiting | UK SMA Patient Registry NCT04292574 | Newcastle University | — |
| Completed | Mechanisms of Cell Death in Spinal Muscular Atrophy NCT01754441 | Nemours Children's Clinic | — |
| Completed | Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy NCT00481013 | University of Utah | Phase 2 |
| Completed | Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy NCT00528268 | University of Utah | Phase 1 / Phase 2 |
| Completed | A Trial of Hydroxyurea in Spinal Muscular Atrophy NCT00485511 | Kaohsiung Medical University Chung-Ho Memorial Hospital | Phase 2 / Phase 3 |
| Completed | Evaluation of the Muscle Strength and Motor Ability in Children With Spinal Muscle Atrophy(SMA) Treated With V NCT01033331 | University of Sao Paulo General Hospital | — |
| Completed | Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy NCT00227266 | University of Utah | Phase 2 |
| Completed | Clinical Study of Spinal Muscular Atrophy NCT00443066 | Columbia University | — |
| Unknown | Establishing Novel Detection Techniques for Various Genetic-Related Diseases by Applying DHPLC Platform. NCT00154960 | National Taiwan University Hospital | — |
| Unknown | Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System NCT00155168 | National Taiwan University Hospital | — |
| Completed | Study of Safety and Dosing Effect on SMN Levels of Valproic Acid (VPA) in Patients With Spinal Muscular Atroph NCT00374075 | University of Utah | Phase 1 |
| Completed | Measuring Levels of SMN in Blood Samples of SMA Patients NCT00061607 | National Institute of Neurological Disorders and Stroke (NINDS) | — |
| Completed | Phase II Study of Leuprolide and Testosterone for Men With Kennedy's Disease or Other Motor Neuron Disease NCT00004771 | National Center for Research Resources (NCRR) | Phase 2 |
| Approved For Marketing | AveXis Managed Access Program Cohort for Access to AVXS-101 NCT03955679 | United BioSource, LLC | — |