| A Study to Evaluate the Efficacy and Safety of Taldefgrobep Alfa in Participants With Spinal Muscular Atrophy (NCT05337553) | Biohaven Pharmaceuticals, Inc. | 2022-07-06 |
| Schulze Muscular Dystrophy Ability Clinical Study (NCT05409079) | AbiliTech Medical Inc. | 2022-05-26 |
| Real-World Use of Novel Treatments in Patients With Spinal Muscular Atrophy (SMA): A Multi-Site Retrospective Chart Review of Pediatric SMA Patients Outside of the United States (NCT06178393) | Novartis Pharmaceuticals | 2022-05-05 |
| COMMAND Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis (NCT05035823) | Synchron Medical, Inc. | 2022-04-27 |
| Equine-Assisted Physiotherapy for Spinal Muscular Atrophy (NCT05341453) | Charles University, Czech Republic | 2022-04-22 |
| SWITCH II Early Feasibility Study: Implantable BCI to Control a Digital Device for People With Paralysis (NCT05041114) | Synchron Medical, Inc. | 2022-04-21 |
| Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinersen or Risdiplam (NCT05156320) | Scholar Rock, Inc. | 2022-04-14 |
| A Study to Assess the Clinical Validity of Konectom™ in Adults Living With Neuromuscular Disorders (NCT05109637) | Biogen | 2022-02-10 |
| Implementation of FEES in Spinal Muscle Atrophy (NCT04773470) | University of Giessen | 2022-02-01 |
| Study of the Functional Effects of Nusinersen in 5q-spinal Muscular Amyotrophy Adults (SMA Type 2 or 3 Forms) (NCT04576494) | CHU de Reims | 2022-01-24 |
| A Study to Learn About the Effect of Higher Doses of Nusinersen (BIIB058) Given as Injections to Participants With Spinal Muscular Atrophy (SMA) Who Were Previously Treated With Risdiplam (ASCEND) (NCT05067790) | Biogen | 2022-01-21 |
| Outpatient Rehabilitation Intervention for Young Children With SMA (NCT05638750) | Holland Bloorview Kids Rehabilitation Hospital | 2022-01-01 |
| Antisense Oligonucleotide for Spinal Muscular Atrophy (NCT05187260) | First Affiliated Hospital of Fujian Medical University | 2022-01-01 |
| Public AttitudesTowards SMA and DMD Awareness, Newborn and Carrier Screening and Physiotherapy Practices (NCT05110885) | Bahçeşehir University | 2021-11-01 |
| Investigating NMJ Defects in SMA Following Central and Peripheral SMN Restoration (NCT05219487) | Bakri Elsheikh | 2021-10-13 |
| Characterization of the Postural Habits of Wheelchair Users Analysis of the Acceptability of International Recommendations in the Prevention of Pressure Sores Risk by Using a Connected Textile Sensor (NCT04335942) | Assistance Publique - Hôpitaux de Paris | 2021-09-30 |
| Safety and Efficacy of Intravenous OAV101 (AVXS-101) in Pediatric Patients With Spinal Muscular Atrophy (SMA) (NCT04851873) | Novartis Pharmaceuticals | 2021-09-08 |
| Personalized Medicine for SMA: a Translational Project (NCT05779956) | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 2021-09-01 |
| SMN Circular RNAs as Potential New Targets and Biomarkers for SMA (NCT05760209) | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 2021-07-22 |
| Outcome Measures and Biomarkers in a Cohort of Spinal Muscular Atrophy Type III/ IV Patients (NCT04690998) | Assistance Publique Hopitaux De Marseille | 2021-07-13 |
| Quantification of Motor Function in Infants With Spinal Muscular Atrophy Treated With Innovative Therapies (NCT04833348) | Assistance Publique - Hôpitaux de Paris | 2021-03-20 |
| Prenatal Carrier Screening for Spinal Muscular Atrophy Among Thai Pregnant Women (NCT04859179) | Mahidol University | 2021-03-10 |
| Respiratory Muscle Training in Patients With Spinal Muscular Atrophy (SMA). (NCT05632666) | UMC Utrecht | 2021-02-02 |
| Responsiveness and Validation Study of MFM-32 in SMA Patients Treated With Nusinersen (NCT04602195) | Hospices Civils de Lyon | 2021-01-19 |
| Responsiveness and Validation Study of MFM-20 in SMA Patients Treated With Nusinersen (NCT04644393) | Hospices Civils de Lyon | 2020-11-01 |
| Characterization of the Clinical-epidemiological Profile of Patients With SMA5q Types II and III: Observational Study (NCT04404764) | Hospital Israelita Albert Einstein | 2020-05-27 |
| CuidAME: Longitudinal Data Collection From Patients With Spinal Muscular Atrophy in Spain (NCT07231549) | Fundació Sant Joan de Déu | 2020-05-05 |
| NuroSleeve Powered Brace & Stimulation System to Restore Arm Function (NCT04798378) | Thomas Jefferson University | 2020-04-16 |
| Evaluation of Therapeutic Response in Spinal Muscular Atrophy Using Multispectral Optoacoustic Tomography (MSOT) and Magnetic Resonance Imaging (MRI) (NCT04262570) | University of Erlangen-Nürnberg Medical School | 2020-02-25 |
| French Register of Patients With Spinal Muscular Atrophy (NCT04177134) | Assistance Publique - Hôpitaux de Paris | 2020-01-31 |
| SMN Circular RNAs as Potential Biomarkers for the Therapeutic Response to Nusinersen in Spinal Muscular Atrophy Patients (NCT05761262) | Fondazione Policlinico Universitario Agostino Gemelli IRCCS | 2019-12-13 |
| 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 | 2019-11-11 |
| New Imaging Biomarkers for Muscular Diseases - Multispectral Optoacoustic Imaging in Spinal Muscular Atrophy (NCT04115475) | University of Erlangen-Nürnberg Medical School | 2019-11-07 |
| Pulmonary Function Test Changes and Respiratory Muscle Strength Trends in Spinal Muscular Atrophy Patients Receiving Nusinersen Treatments (NCT04050852) | NYU Langone Health | 2019-07-17 |
| A Registered Cohort Study on SMA (NCT04010604) | Wan-Jin Chen | 2019-07-01 |
| The Burden of Primary Caregivers of Spinal Muscular Atrophy Patients and Their Needs (NCT04228718) | Marmara University | 2019-07-01 |
| A Drug-drug Interaction Study With Risdiplam Multiple Dose and Midazolam in Healthy Participants (NCT03988907) | Hoffmann-La Roche | 2019-06-18 |
| An Active Treatment Study of SRK-015 in Patients With Type 2 or Type 3 Spinal Muscular Atrophy (NCT03921528) | Scholar Rock, Inc. | 2019-04-22 |
| Feasibility of Virtual Reality in Children With Neuromuscular Disease, Effectiveness of Virtual Reality and Biofeedback (NCT03689660) | Merve Kurt | 2019-02-01 |
| Early Check: Expanded Screening in Newborns (NCT03655223) | RTI International | 2018-10-15 |
| Adults With SMA Treated With Nusinersen (NCT04591678) | Ohio State University | 2018-10-01 |
| Spinraza in Adult Spinal Muscular Atrophy (NCT03709784) | Washington University School of Medicine | 2018-08-16 |
| Motor Unit Number Estimation (MUNE) in Adults With Spinal Muscular Atrophy (SMA) (NCT04139343) | Ohio State University | 2018-08-10 |
| Holter of Movement in Patients with SMA Undergoing Treatment. (NCT04888702) | Centre Hospitalier Universitaire de Liege | 2018-08-08 |
| Pre-Symptomatic Study of Intravenous Onasemnogene Abeparvovec-xioi in Spinal Muscular Atrophy (SMA) for Patients With Multiple Copies of SMN2 (NCT03505099) | Novartis Gene Therapies | 2018-04-02 |
| Sun May Arise on SMA : Newborn Screening of Spinal Muscular Atrophy in Belgium (NCT03554343) | Centre Hospitalier Régional de la Citadelle | 2018-03-05 |
| Genetic Newborn Screening for Cystinosis and Spinal Muscular Atrophy (NCT06027385) | Cystinose Stiftung | 2018-01-15 |
| Study of Intrathecal Administration of Onasemnogene Abeparvovec-xioi for Spinal Muscular Atrophy (NCT03381729) | Novartis Gene Therapies | 2017-12-14 |
| European Registry of Patients With Infantile-onset Spinal Muscular Atrophy (NCT03339830) | Institut de Myologie, France | 2017-10-10 |
| Development of a Space Exploration Assessment for Children With Spinal Muscular Atrophy (NCT03223051) | Centre d'Investigation Clinique et Technologique 805 | 2017-09-07 |
| Neonatal Spinal Muscular Atrophy (SMA) Screening (NCT03217578) | Kaohsiung Medical University Chung-Ho Memorial Hospital | 2017-09-01 |
| Natural History of Types 2 and 3 SMA in Taiwan (NCT03300869) | Kaohsiung Medical University Chung-Ho Memorial Hospital | 2017-09-01 |
| Fat and Glucose Metabolism in Fed and Fasted State in Patients With Low Skeletal Muscle Mass (NCT03970135) | Rigshospitalet, Denmark | 2017-05-29 |
| Metabolomics of Children With SMA (NCT04587492) | University Medical Centre Ljubljana | 2017-01-01 |
| Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy (NCT03808233) | University of Texas Southwestern Medical Center | 2016-11-05 |
| Study of Feasibility to Reliably Measure Functional Abilities' Changes in Nonambulant Neuromuscular Patients Without Trial Site Visiting (NCT02235090) | Charitable Foundation Children with Spinal Muscular Atrophy | 2016-10-01 |
| Prospective Evaluation of Infants With Spinal Muscular Atrophy: (NCT02831296) | Massachusetts General Hospital | 2016-02-01 |
| A Study of CK-2127107 in Patients With Spinal Muscular Atrophy (NCT02644668) | Cytokinetics | 2016-01-14 |
| A Study to Investigate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Risdiplam (RO7034067) Given by Mouth in Healthy Volunteers (NCT02633709) | Hoffmann-La Roche | 2016-01-07 |
| Identification of a Biomarker Associated With Cis-duplication of the SMN1 Gene (NCT02550691) | University Hospital, Rouen | 2015-12-15 |
| Effects of Standing on Non-Ambulatory Children With Neuromuscular Conditions (NCT02428673) | Gillette Children's Specialty Healthcare | 2015-12-09 |
| A Study for Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in Nusinersen (ISIS 396443) Investigational Studies (NCT02594124) | Biogen | 2015-11-04 |
| Impairments of Neuro-muscular Communication in Motor-Neuron Disease: A Bio-Marker for Early and Personalised Diagnosis (NCT05663008) | University of Dublin, Trinity College | 2015-10-01 |
| A Study to Assess the Safety and Tolerability of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA). (NCT02462759) | Biogen | 2015-08-19 |
| A Study of Multiple Doses of Nusinersen (ISIS 396443) Delivered to Infants With Genetically Diagnosed and Presymptomatic Spinal Muscular Atrophy (NCT02386553) | Biogen | 2015-05-18 |
| An Open Label Study of LMI070 (Branaplam) in Type 1 Spinal Muscular Atrophy (SMA) (NCT02268552) | Novartis Pharmaceuticals | 2015-04-02 |
| A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Participants With Later-onset Spinal Muscular Atrophy (SMA) (NCT02292537) | Biogen | 2014-11-24 |
| A Study to Assess the Efficacy and Safety of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (NCT02193074) | Biogen | 2014-08-19 |
| WREX Outcome Study (NCT02218593) | Nemours Children's Clinic | 2014-07-01 |
| National Registry for Egyptian Pediatric Neuromuscular Diseases (NCT02124616) | Ain Shams University | 2014-04-01 |
| An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) Who Previously Participated in ISIS 396443-CS2 (NCT01703988) or ISIS 396443-CS10 (NCT01780246) (NCT02052791) | Biogen | 2014-01-31 |
| Improving Standards of Care and Translational Research in Spinal Muscular Atrophy (SMA) (NCT03520179) | Great Ormond Street Hospital for Children NHS Foundation Trust | 2013-12-17 |
| Newborn Screening for Spinal Muscular Atrophy (NCT02123186) | National Taiwan University Hospital | 2013-10-01 |
| Valproate and Levocarnitine in Children With Spinal Muscular Atrophy (NCT01671384) | All India Institute of Medical Sciences | 2013-08-01 |
| Reciprocating Gait Orthoses for Paraplegia Patients (NCT02227407) | National Taiwan University Hospital | 2013-08-01 |
| At-Home Research Study for Patients With Autoimmune, Inflammatory, Genetic, Hematological, Infectious, Neurological, CNS, Oncological, Respiratory, Metabolic Conditions (NCT01931644) | Sanguine Biosciences | 2013-07-01 |
| A Study to Assess the Efficacy, Safety and Pharmacokinetics of Nusinersen (ISIS 396443) in Infants With Spinal Muscular Atrophy (SMA) (NCT01839656) | Biogen | 2013-05-08 |
| An Open-label Safety and Tolerability Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy Who Previously Participated in ISIS 396443-CS1 (NCT01494701) (NCT01780246) | Biogen | 2013-01-31 |
| An Open-label Safety, Tolerability and Dose-Range Finding Study of Multiple Doses of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (NCT01703988) | Biogen | 2012-10-31 |
| Short and Long Term Treatment With 4-AP in Ambulatory SMA Patients (NCT01645787) | Columbia University | 2012-06-01 |
| Infants With Spinal Muscular Atrophy Type I (NCT01547871) | National Institute of Neurological Disorders and Stroke (NINDS) | 2012-02-07 |
| An Open-label Safety, Tolerability, and Dose-range Finding Study of Nusinersen (ISIS 396443) in Participants With Spinal Muscular Atrophy (SMA) (NCT01494701) | Biogen | 2011-11-30 |
| Flu Vaccine Study in Neuromuscular Patients 2011 (NCT01422200) | Children's Hospital Medical Center, Cincinnati | 2011-08-01 |
| Clinical Assessment of Spinal Muscular Atrophy Type II and III (SMA Europe) (NCT01611610) | Institut de Myologie, France | 2011-05-01 |
| Spinal Muscular Atrophy and Cardiac Autonomic Function (NCT01522079) | Centro Universitário Augusto Motta | 2011-01-01 |
| Optimum Insufflation Capacity in NMD (NCT01981915) | Universität Duisburg-Essen | 2011-01-01 |
| Clinical Trial of Exercise in Patients With Spinal Muscular Atrophy (SMA) (NCT01166022) | Columbia University | 2010-12-01 |
| SMN Copy Number Distribution in Mali, West Africa (NCT01059240) | National Institute of Neurological Disorders and Stroke (NINDS) | 2010-01-21 |
| Aerobic Training in Patients With Spinal Muscular Atrophy Type III (NCT02003937) | Rigshospitalet, Denmark | 2009-09-01 |
| Effects of Power Mobility on Young Children With Severe Motor Impairments (NCT01028833) | University of Oklahoma | 2008-11-01 |
| A Pilot Study of Biomarkers for Spinal Muscular Atrophy (NCT00756821) | Carelon Research | 2008-10-01 |
| UK SMA Patient Registry (NCT04292574) | Newcastle University | 2008-07-13 |
| Mechanisms of Cell Death in Spinal Muscular Atrophy (NCT01754441) | Nemours Children's Clinic | 2008-05-01 |
| Valproic Acid in Ambulant Adults With Spinal Muscular Atrophy (NCT00481013) | University of Utah | 2007-07-01 |
| Study to Evaluate Sodium Phenylbutyrate in Pre-symptomatic Infants With Spinal Muscular Atrophy (NCT00528268) | University of Utah | 2007-07-01 |
| A Trial of Hydroxyurea in Spinal Muscular Atrophy (NCT00485511) | Kaohsiung Medical University Chung-Ho Memorial Hospital | 2007-06-01 |
| Valproic Acid and Carnitine in Patients With Spinal Muscular Atrophy (NCT00227266) | University of Utah | 2005-09-01 |
| Clinical Study of Spinal Muscular Atrophy (NCT00443066) | Columbia University | 2005-05-01 |
| Establishing Novel Detection Techniques for Various Genetic-Related Diseases by Applying DHPLC Platform. (NCT00154960) | National Taiwan University Hospital | 2004-06-01 |
| Quantitative Analysis of SMN1 and SMN2 Gene Based on DHPLC System (NCT00155168) | National Taiwan University Hospital | 2004-04-01 |