| Recruiting | Phase 2 Study of SAT-3247 in Pediatric Ambulatory Patients NCT07287189 | Satellos Bioscience, Inc. | Phase 2 |
| Unknown | Validation of the "ExSpiron©" in Patients With ALS NCT04089696 | University Medical Center Groningen | N/A |
| Not Yet Recruiting | Nebulised Hypertonic Saline to Decrease Respiratory Exacerbations in Neuromuscular Disease or Neurodisability NCT06134401 | Imperial College London | N/A |
| Recruiting | Elaboration of a Pronostic Score of Changes on Wheelchair's Seating System NCT05785546 | Assistance Publique - Hôpitaux de Paris | — |
| Recruiting | MSOT as Non-invasive Biomarker for Diagnosis and Monitoring of Neuromuscular Diseases NCT06438965 | Children's Hospital of Eastern Ontario | — |
| Not Yet Recruiting | Efficacy and Safety of Efgartigimod Sequential Therapy with Telitacicept in Generalized Myasthenia Gravis NCT06827587 | First Affiliated Hospital of Wenzhou Medical University | N/A |
| Recruiting | Detecting Systemic Carbon Dioxide Levels With a Novel Biosensor NCT06367686 | The Hospital of Vestfold | — |
| Recruiting | A Phase 3 Study of NTLA-2001 in ATTRv-PN NCT06672237 | Intellia Therapeutics | Phase 3 |
| Active Not Recruiting | Mechanical Insufflation in the Philadelphia Amyotrophic Lateral Sclerosis Cohort (MI-PALS) Study NCT06119087 | University of Pennsylvania | N/A |
| Completed | Pain and Muscle Biopsy NCT06379932 | Assistance Publique - Hôpitaux de Paris | — |
| Recruiting | A Prospective Natural History and Outcome Measure Discovery Study of Charcot-Marie-Tooth Disease, Type 4J NCT06151600 | Elpida Therapeutics SPC | — |
| Recruiting | Spastic Paraplegia - Centers of Excellence Research Network NCT06553976 | Boston Children's Hospital | — |
| Recruiting | Assessment of Volume-targeted Ventilation in Patients With Neuromuscular Disease NCT06339580 | Guy's and St Thomas' NHS Foundation Trust | Phase 2 |
| Recruiting | Gait Analysis Parameter and Upper Limb Evaluation in Adult Patients With Neurological or Metabolic Pathology NCT07136844 | Centre Hospitalier Universitaire de Liege | N/A |
| Completed | Expediting Myasthenia Gravis (MG) Diagnostic Evaluation: A Novel, Proof-of-Concept for Undiagnosed, Symptomati NCT06381284 | ZS Associates | — |
| Recruiting | Polygraphy at Home for Neuromuscular Patients Under Non-invasive Mechanical Ventilation NCT05785637 | Assistance Publique - Hôpitaux de Paris | — |
| Completed | Mollii Suit and Fibromyalgia (EXOFIB 2) NCT06226376 | Sheikh Shakhbout Medical City | N/A |
| Recruiting | Emotion Processing Among Patients With ALS NCT06566651 | University of Aarhus | — |
| Recruiting | Investigation on the Cortical Communication System NCT06207591 | UMC Utrecht | N/A |
| Unknown | Does the Coaching Function Improve the Learning of Mechanical In-exsufflation (MI-E) in Paediatric Subjects? NCT06190431 | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | N/A |
| Completed | Posterior Chain EMG Patterns in Athletes With and Without Hamstring Injury NCT07171385 | Ankara Yildirim Beyazıt University | — |
| Unknown | Effects of Power Wheelchair Soccer for Individuals Presenting With Physical Impairments NCT06259643 | Université Catholique de Louvain | N/A |
| Completed | Effect of the Use of a 3D Scanner Application on a Smartphone to Mold Garchois Orthotic Device in Neuromuscula NCT06428864 | Hospices Civils de Lyon | — |
| Unknown | Developing a Mobile Application for the Care of Children With Gastrostomy and The Effect of the Application on NCT05831514 | Akdeniz University | N/A |
| Unknown | Feasibility, Validation and Application of Digital Tools for the Follow-up of Neuromuscular Patient Mobility i NCT05798325 | Institut de Myologie, France | N/A |
| Recruiting | Research Biobank From Neuromuscular Biopsy Residues (in the Context of Care) NCT06074822 | University Hospital, Bordeaux | — |
| Unknown | Evaluation of the Feasibility of Remote Monitoring of Mechanical In-exsufflation Devices in Paralytic and Neur NCT05723289 | KerNel Biomedical | — |
| Recruiting | Precision Diagnosis and Therapy for Rare Diseases by Interpreting Non-coding Genomes NCT06775561 | IRCCS Azienda Ospedaliero-Universitaria di Bologna | — |
| 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 |
| Active Not Recruiting | Development and Test of a Headset for BCI Until Obtaining an Efficient and Comfortable System That Can be Used NCT04380649 | Centre Hospitalier Universitaire de Nice | N/A |
| Withdrawn | Experimentation of a Personalized Physical Self-education Program at Home: Feasibility Study (PROSOL TELEKINEC NCT05597592 | Centre Hospitalier Universitaire de Nice | N/A |
| Completed | Absorption, Metabolism, Excretion and Absolute Bioavailability of EDG-5506 in Healthy Volunteers NCT05730842 | Edgewise Therapeutics, Inc. | Phase 1 |
| Completed | Reliability Study of a Motor Function Measure Digitalized Playful Completion Modules NCT05227274 | Hospices Civils de Lyon | — |
| Unknown | Soft-robotic Glove Support of High-demand Tasks NCT05318638 | Roessingh Research and Development | N/A |
| Completed | The Peer Support Study NCT05070624 | The Hospital for Sick Children | 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 | — |
| Recruiting | Controlled Study of Upper and Lower Limb Movements in Patients Patients with Pathologies That Have an Impact o NCT06439823 | Laurent Servais | N/A |
| Completed | Extension of AOC 1001-CS1 (MARINA) Study in Adult Myotonic Dystrophy Type 1 (DM1) Patients NCT05479981 | Avidity Biosciences, Inc. | Phase 2 |
| Unknown | The Effect of Aerobic Exercise Training in Patients With Type III Spinal Muscular Atrophy NCT05544994 | Istanbul Medipol University Hospital | N/A |
| 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 |
| Active Not Recruiting | A Study of Deramiocel (CAP-1002) in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy NCT05126758 | Capricor Inc. | Phase 3 |
| Enrolling By Invitation | MYO-SHARE: MYO-MRI in Neuromuscular Diseases NCT04866459 | Ottawa Hospital Research Institute | — |
| Completed | Efficacy and Safety of Apitegromab in Patients With Later-Onset Spinal Muscular Atrophy Treated With Nusinerse NCT05156320 | Scholar Rock, Inc. | Phase 3 |
| Completed | Understanding Control and Mechanisms of Shoulder Instability in FSHD NCT05239520 | University of Liverpool | — |
| Unknown | Volume Mode Non-invasive Ventilation in Amyotrophic Lateral Sclerosis NCT05328492 | University Hospitals Coventry and Warwickshire NHS Trust | N/A |
| Unknown | Efficacy of Intermittent Abdominal Pressure Ventilation in Neuromuscular Patients NCT05346263 | Fondazione Don Carlo Gnocchi Onlus | N/A |
| Completed | The Bridges Self-management Programme for People With Neuromuscular Diseases NCT05460156 | University College London Hospitals | N/A |
| Recruiting | Respiratory Muscles and Work of Breathing in Children NCT05051254 | Assistance Publique - Hôpitaux de Paris | N/A |
| Recruiting | Can Waveform and Flow Traces From Mechanical Insuflattion:Exsufflation (MI:E) be Used to Identify Laryngeal Re NCT05189600 | Royal Free Hospital NHS Foundation Trust | — |
| Unknown | Assessment of the Effectiveness of Muscle Activating Training With the Use of EMG Biofeedback in Patients With NCT05122273 | EGZOTech | — |
| Terminated | Oscillation and Lung Expansion (OLE) Therapy for Treatment of Neuromuscular Disease (NMD) Patients NCT05366010 | Baxter Healthcare Corporation | N/A |
| Completed | Neuro-psychosocial Teleassistance for Neuromuscular Diseases NCT06031701 | University of Deusto | N/A |
| Completed | Changes of Motor Function Tests in Congenital Myopathy Subjects Treated With Oral Salbutamol as Compared to no NCT05099107 | Vastra Gotaland Region | N/A |
| Completed | NDT Effectiveness Study NCT05070910 | MultiCare Health System Research Institute | N/A |
| Unknown | Effects of Pre-event Massage on Muscle Activity NCT04986059 | Universitat Internacional de Catalunya | N/A |
| Completed | Efficacy of Vaccination Against COVID-19 in Patients Presenting a Neuromuscular Disease With Severe Amyotrophy NCT05353738 | University Hospital, Bordeaux | — |
| Recruiting | The Neuroimage Study of the Neuromuscular Disorders. NCT05048862 | National Taiwan University Hospital | — |
| Completed | Impact of Covid-19 Pandemic on Children With Neuromuscular Disease and Their Parents in Turkey: Parent Perspec NCT05771727 | Hacettepe University | — |
| Recruiting | Wheelchair Positioning and Neuromuscular Diseases NCT05006859 | Groupement Hospitalier Intercommunal du Vexin | — |
| Unknown | Study of Efficacy and Safety of the Plasmapheresis Method With Albumin Compensation Compared With the Plasmaph NCT04897113 | National Medical Research Center for Rehabilitation and Balneology | N/A |
| Completed | Validation Study of Posturology Platforms for Evaluating Postural Control of Hemiparetic and Neuro-muscular Pa NCT04283175 | Assistance Publique - Hôpitaux de Paris | — |
| Withdrawn | Tracheostomy With Bedside Simultaneous Gastrostomy Vs Usual Care Tracheostomy And Delayed Gastrostomy Placemen NCT04758052 | University of Mississippi Medical Center | N/A |
| Recruiting | CIMR Neuromuscular Research Biobank NCT05434572 | Virginia Commonwealth University | — |
| Completed | Efficacy and Safety of Vaccination Against COVID-19 in Neuromuscular Patients NCT05311904 | University Hospital, Bordeaux | — |
| Withdrawn | Validation of the Efficacy and Usability of the BELK Orthosis Knee Module in a Neurological Setting (BELK@CCP) NCT04173975 | Casa di Cura Privata del Policlinico SpA | N/A |
| Completed | Accuracy of Peak Cough Flow Measurements Using a Mechanical Insufflator-exsufflator NCT04935905 | Guy's and St Thomas' NHS Foundation Trust | — |
| Completed | Quality of Life in Ventilator-dependant Neuromuscular Patients NCT04652401 | Centre d'Investigation Clinique et Technologique 805 | — |
| Recruiting | Measuring Physical Activity With ActivPal NCT06084026 | Columbia University | — |
| Completed | Evaluation of the Exoskeleton ATLAS 2030 as Robot-assisted Physical Therapy to Children With Neuromuscular Dis NCT04837157 | MarsiBionics | N/A |
| Completed | Digitalization Playful Module of Motor Function Measure Assessment for Children With Neuromuscular Disorders, NCT04435093 | Hospices Civils de Lyon | — |
| Completed | Perceptions of the Clinical and Psychosocial Impact of Covid-19 in Patients With NMD NCT04581577 | Royal Brompton & Harefield NHS Foundation Trust | — |
| Terminated | Detection of Neuromuscular Complications in Critically Ill Patients NCT04541602 | University of Rostock | — |
| Completed | Physical Status and Upper Airway Function in Neuromuscular Patients NCT04473612 | Universidad de Granada | — |
| Completed | Rehabilitative Effect of the Use of a Gait Exoskeleton in Patients With Neuromuscular Disease or Cerebral Pals NCT04813601 | MarsiBionics | N/A |
| Terminated | Breath Stacking Technique Associated With Expiratory Muscle Training in Amyotrophic Lateral Sclerosis Patients NCT04226144 | Escola Superior de Ciencias da Saude | N/A |
| Unknown | Optimisation of Mechanical Insufflation:Exsufflation NCT04163198 | Guy's and St Thomas' NHS Foundation Trust | N/A |
| Unknown | Investigating Health Related Quality of Life in Patients With Chronic Respiratory Failure NCT04236726 | Guy's and St Thomas' NHS Foundation Trust | — |
| Unknown | Evaluation of the Effect of a Speech Detection Tool on the Improvement of Phonation in Neuromuscular Patients NCT03504514 | Centre d'Investigation Clinique et Technologique 805 | N/A |
| Completed | Impacting Children's Physical and Mental Health Through Kinesiology Support in Clinical Care NCT04106154 | Children's Hospital of Eastern Ontario | N/A |
| Completed | Multisystem and Autonomic Complications of NMD on Long-term NIV NCT04100993 | Royal Brompton & Harefield NHS Foundation Trust | — |
| Unknown | Outcomes of RV Dysfunction in Acute Exacerbation of Chronic Respiratory Diseases NCT04098094 | Association pour le Développement et l'Organisation de la Recherche en Pneumologie et sur le Sommeil | — |
| Completed | Home Use of Mechanical Insufflation/Exsufflation Devices in Neuromuscular Diseases NCT04729920 | Centre Hospitalier Universitaire Vaudois | — |
| Recruiting | The Role of Concomitant Diseases in Postoperative Complications Risk Stratification. NCT03945968 | Russian Federation of Anesthesiologists and Reanimatologists | — |
| Recruiting | Genesis Electrical Impedance Tomography (EIT): A Preliminary Study NCT03797183 | University of Colorado, Denver | — |
| Unknown | Effects of Pre-event Massage Over the Neuromuscular Response NCT03941067 | Universitat Internacional de Catalunya | N/A |
| 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 |
| Active Not Recruiting | Comparison of Two Methods of Adjusting the Mechanical In-Exsuflation in Neuromuscular Adult Patients NCT03355105 | Assistance Publique - Hôpitaux de Paris | N/A |
| Unknown | Effect of Functional Massage in Neuromuscular Response. NCT03964961 | Universitat Internacional de Catalunya | N/A |
| Completed | 'Fitness To Fly' Tests In Children With Type II Respiratory Failure NCT03824223 | Great Ormond Street Hospital for Children NHS Foundation Trust | N/A |
| Terminated | Comparative Trial of Home-Based Palliative Care NCT03694431 | Kaiser Permanente | N/A |
| Withdrawn | Identity Development in Youth With Neuromuscular Diseases NCT03775135 | Universitaire Ziekenhuizen KU Leuven | — |
| Completed | The Debated Role of Sleep Studies in Patients Under Established Home Mechanical Ventilation NCT03506906 | Wissenschaftliches Institut Bethanien e.V | N/A |
| Active Not Recruiting | Transcutaneous CO2 Monitoring at Home for Children With Neuromuscular Disease NCT03478566 | The Hospital for Sick Children | N/A |
| Completed | Acute Intermittent Hypoxia and Breathing in Neuromuscular Disease NCT03645031 | University of Florida | N/A |
| Completed | North Carolina Genomic Evaluation by Next-generation Exome Sequencing, 2 NCT03548779 | University of North Carolina, Chapel Hill | N/A |
| Completed | Nebulised Hypertonic Saline in Children and Young People With Neuromuscular Disease and Cerebral Palsy NCT03623698 | Imperial College London | — |
| Completed | Impact of the Type of Interface in Neuromuscular Patients Treated With Nocturnal Noninvasive Ventilation NCT03458507 | University Hospital, Grenoble | N/A |
| Completed | Study of Pioglitazone in Sporadic Inclusion Body Myositis NCT03440034 | Johns Hopkins University | Phase 1 |
| Completed | A Study of CAP-1002 in Ambulatory and Non-Ambulatory Patients With Duchenne Muscular Dystrophy NCT03406780 | Capricor Inc. | Phase 2 |
| Unknown | Effects of Fear of Falling on Physical Performance and Quality of Life in Children With Duchenne Muscular Dyst NCT03507530 | Canan İpek | — |
| Completed | Validation of Completion of Items 18, 19 and 22 of the Motor Function Measure (MFM) on a Digital Tablet NCT03465358 | Hospices Civils de Lyon | — |
| Unknown | Impact of the Pre-phonatory Inspiratory Volume on the Speech Quality of Neuromuscular Patients Dependent on No NCT03381937 | Centre d'Investigation Clinique et Technologique 805 | N/A |
| Completed | Efficacy of Surgical Treatment by Different Pedicle Screw Systems in Pediatric Neuromuscular Spinal Deformity NCT03863496 | Ilizarov Center | N/A |
| Completed | Evaluation of an On-Demand Humidifier on Neuromuscular Patients Requiring Mechanical Ventilation NCT03289715 | Philips Electronics Nederland B.V. acting through Philips CTO organization | N/A |
| Completed | Pulmonary Function and Neuromuscular Disease NCT03445832 | Centre Hospitalier Universitaire de Besancon | — |
| Completed | Mechanical Cough Augmentation in Children With NMD NCT03831568 | Oslo University Hospital | — |
| Active Not Recruiting | 3 Year Follow up on ANO5 Patients NCT05206617 | Rigshospitalet, Denmark | — |
| Terminated | Telemedicine for Improving Outcome in Inner City Patient Population With Hypercapneic Respiratory Failure NCT03353064 | Albert Einstein Healthcare Network | N/A |
| Completed | Long-Term Outcomes of Ataluren in Duchenne Muscular Dystrophy NCT03179631 | PTC Therapeutics | Phase 3 |
| Completed | AMX0035 in Patients With Amyotrophic Lateral Sclerosis (ALS) NCT03127514 | Amylyx Pharmaceuticals Inc. | Phase 2 |
| Unknown | Treatment Effect of Edaravone in Patients With Amyotrophic Lateral Sclerosis (ALS) NCT03272802 | Isfahan University of Medical Sciences | Phase 2 / Phase 3 |
| Unknown | Solution Focused Coaching Strategy Within a Paediatric Multidisciplinary Neuromuscular Clinic NCT02997514 | Holland Bloorview Kids Rehabilitation Hospital | N/A |
| Completed | Oculomotor Training and Chinese Characters Recognition in Children With Neuromuscular Disease NCT03627962 | The Hong Kong Polytechnic University | N/A |
| Completed | Effectiveness Monitoring of Home Non-invasive Mechanical Ventilation by Digital Diagnosis Platform NCT03085537 | Corporacion Parc Tauli | — |
| Completed | Validation of the French Version of the Sydney Swallow Questionnaire in Patients With Neuromuscular Diseases NCT02845362 | Cliniques universitaires Saint-Luc- Université Catholique de Louvain | N/A |
| Completed | Changes in Regional Lung Ventilation Following Mechanical Insufflation-Exsufflation NCT02753959 | Guy's and St Thomas' NHS Foundation Trust | N/A |
| Terminated | Repeat of: A Study to Evaluate Efficacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patient NCT02829814 | Tonix Pharmaceuticals, Inc. | Phase 3 |
| Completed | Assessing Air Travel Safety in Neuromuscular Dystrophy: Standard Versus Prolonged High Altitude Simulation Tes NCT03312361 | The Hospital for Sick Children | N/A |
| Completed | Electronic Medical Records and Genomics (eMERGE) Phase III NCT03394859 | Vanderbilt University Medical Center | — |
| Completed | Muscle Oxygenation in Effort in Neuromuscular Diseases NCT02789059 | University Hospital, Lille | N/A |
| Completed | Initiation of Home Mechanical Ventilation at Home in Patients With Chronic Hypercapnic Respiratory Failure NCT03203577 | University Medical Center Groningen | N/A |
| Completed | Monitoring Mechanical Assisted Cough NCT03218215 | Corporacion Parc Tauli | — |
| Completed | A Study to Evaluate eFFIcacy and Safety of Sublingual TNX-102 SL Tablet Taken at Bedtime in Patients With fibR NCT02436096 | Tonix Pharmaceuticals, Inc. | Phase 3 |
| Completed | Patient Preference for Mouthpiece Ventilation NCT03867721 | Revalidatieziekenhuis InkendaaI | N/A |
| Unknown | Installation of Non-invasive Ventilation at Home Using Telemedicine : a Pilot Study on Feasibility and Impact NCT02103790 | Centre d'Investigation Clinique et Technologique 805 | N/A |
| Completed | Do Handgrip Strength and Dexterity Predict of Respiratory Function ? NCT05021744 | Bezmialem Vakif University | — |
| Recruiting | Neuropsychological and Psychosocial Follow up of Children and Adolescents With Neuromuscular Disease NCT02253290 | Universitaire Ziekenhuizen KU Leuven | — |
| Completed | Methods to Improve Expiratory Cough Flow and Lung Volume in Children With NMDs NCT02342132 | University of British Columbia | N/A |
| Completed | Effectiveness and Cost-effectiveness of the Energetic Program NCT02208687 | Radboud University Medical Center | N/A |
| Recruiting | Non-Invasive Respiratory Monitor NCT02851043 | Nemours Children's Clinic | N/A |
| Unknown | Bulbar Function in Neuromuscular Patients NCT02097368 | Centre d'Investigation Clinique et Technologique 805 | — |
| Terminated | An Extension Study of Ataluren (PTC124) in Participants With Nonsense Mutation Dystrophinopathy NCT02090959 | PTC Therapeutics | Phase 3 |
| Terminated | Volontary Inspiratory Muscles Measures Compared With Phrenic Nerves Magnetic Stimulations Measures NCT02078050 | Centre d'Investigation Clinique et Technologique 805 | N/A |
| Completed | Air Stacking vs Glossopharyngeal Breathing in Neuromuscular Diseases NCT02500030 | University of Chile | N/A |
| Completed | Phase 3 Study of Ataluren in Participants With Nonsense Mutation Duchenne Muscular Dystrophy (nmDMD) NCT01826487 | PTC Therapeutics | Phase 3 |
| Terminated | Non-invasive Ventilation and Physical Activity NCT02951572 | Universitaire Ziekenhuizen KU Leuven | N/A |
| Completed | Effectiveness of Respiratory Physiotherapy in Children With Neuromuscular Disease NCT02743702 | University of Seville | N/A |
| Completed | A Longitudinal Study of Amyotrophic Lateral Sclerosis (ALS) Biomarkers NCT01495390 | Massachusetts General Hospital | — |
| Completed | Epidemiology and Clinical Presentation of H1N1 Infection in Children in Geneva NCT01022931 | University Hospital, Geneva | — |
| Completed | The Value of Traction in the Treatment of Cervical Radiculopathy NCT00979108 | Intermountain Health Care, Inc. | Phase 3 |
| Completed | The Value of Traction in Treatment of Lumbar Radiculopathy NCT00942227 | Intermountain Health Care, Inc. | Phase 3 |
| Terminated | Evaluation of a Mechanical Device During Acute Respiratory Failure in Patients With Neuromuscular Disorders NCT00839033 | Assistance Publique - Hôpitaux de Paris | Phase 3 |
| Completed | Establishment of a Non-invasive and Indirect Measure of Volitional Pimax NCT02242162 | Centre d'Investigation Clinique et Technologique 805 | — |
| Unknown | Comparative Study of Non-Invasive Mask Ventilation vs Cuirass Ventilation in Patients With Acute Respiratory F NCT00331656 | Hadassah Medical Organization | Phase 4 |
| Completed | Metformin in Children With Motor Deficit NCT00720161 | Universitaire Ziekenhuizen KU Leuven | N/A |
| Completed | Hippotherapy to Improve the Balance of Children With Movement Disorders NCT01313325 | Central Michigan University | N/A |
| Available | Managed Access Program for Del-zota in Participants With DMD Mutations Amenable to Exon 44 Skipping NCT07250737 | Avidity Biosciences, Inc. | — |
| Completed | A Pilot Study To Evaluate the Effect of Retrovir (Zidovudine: AZT) in the Treatment of Human Immunodeficiency NCT00002044 | Glaxo Wellcome | N/A |