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Trial Match

Clinical trials test new treatments — sometimes the newest options available for a disease. Find trials that are recruiting now, near you. Open one to see its eligibility rules as simple questions to go through with your doctor.

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30 recruiting trials

for Neuromuscular; Disorder, Hereditary

Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 NCT07070999Phase 1, Phase 21 site📍 Porto Alegre Gemma Biotherapeutics · Tests: GB221 Multiparametric Ultrafast Ultrasound Biomarkers for Duchenne and Becker Muscular Dystrophies NCT07378553n/a1 site📍 Nantes Nantes University Hospital Safety and Preliminary Efficacy of ULSC in Facioscapulohumeral Muscular Dystrophy (FSHD) NCT07086521Phase 11 site📍 Palo Alto Restem, LLC. · Tests: ULSC, Placebo Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting NCT07542314Phase 41 site📍 Houston Sarepta Therapeutics, Inc. · Tests: ELEVIDYS, Sirolimus, Glucocorticoids, Antibiotics A First in Human Study to Assess the Safety, Tolerability, and Pharmacokinetics of EDK060 in Adults With CMT1A. NCT07140614Phase 14 sites📍 Ottawa📍 Montreal📍 Montreal Novartis Pharmaceuticals · Tests: EDK060, dose A, EDK060, dose B, EDK060, dose C, EDK060, dose D, Placebo Neurodegenerative Diseases Progression Markers (MARKERS-NDD) NCT06596746n/a1 site📍 Cassino Casa di Cura San Raffaele Cassino · Tests: Observation Cystinosis and Mitochondrial Metabolism NCT07319091NA9 sites📍 Bron📍 Lille📍 Lyon Hospices Civils de Lyon · Tests: Mitochondrial metabolism Amino Acids and Exercise in FSHD NCT07543016NA1 site📍 Voghera University of Pavia · Tests: Essential Amino Acids Supplementation intervention, Exercise, Diet, Placebo Control The NF Registry Sponsored by the Children's Tumor Foundation NCT01885767n/a1 site📍 New York The Children's Tumor Foundation A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1 NCT06541847Phase 214 sites📍 Birmingham📍 Los Angeles📍 Palo Alto Healx Limited · Tests: HLX-1502 Neurosurgical Outcome Network NCT06724029n/a27 sites📍 Bergamo📍 Brescia📍 Brescia Fondazione I.R.C.C.S. Istituto Neurologico Carlo Besta Motor Outcomes to Validate Evaluations in Pediatric FSHD (MOVE Peds) NCT06847282n/a7 sites📍 Palo Alto📍 Iowa City📍 Kansas City University of Kansas Medical Center Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST) NCT06222203n/a1 site📍 Bethesda National Cancer Institute (NCI) Cardiac Amyloidosis Registry of University Hospital Leipzig NCT06129656n/a1 site📍 Leipzig University of Leipzig · Tests: Routine diagnostics, Routine treatment Patisiran-Lipid Nanoparticle (LNP) Pregnancy Surveillance Program NCT05040373n/a7 sites📍 Iowa City📍 Nantes📍 Münster Alnylam Pharmaceuticals Registry for Duchenne and Becker Muscular Dystrophy NCT07402122n/a30 sites📍 Graz📍 Innsbruck📍 Linz Dr. Andreas Ziegler Natural History Evaluation of Charcot Marie Tooth Disease (CMT) Types CMT1B, CMT2A, CMT4A, CMT4C, and Others NCT01193075n/a24 sites📍 Los Angeles📍 Palo Alto📍 Aurora Michael Shy Registry and Natural History Study for Early Onset Hereditary Spastic Paraplegia NCT04712812n/a1 site📍 Boston Boston Children's Hospital Cardiac Magnetic Resonance and Echocardiography Study With Acoramidis NCT07695701Phase 41 site📍 St Louis Eidos Therapeutics, a BridgeBio company · Tests: Acoramidis (AG10) oral tablet A Study of SGT-003 Gene Therapy in Duchenne Muscular Dystrophy (INSPIRE DUCHENNE) NCT06138639Phase 1, Phase 215 sites📍 Little Rock📍 Los Angeles📍 Sacramento Solid Biosciences Inc. · Tests: SGT-003 PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas NCT06961565Phase 15 sites📍 Birmingham📍 St Leonards📍 Melbourne Pasithea Therapeutics Corp. · Tests: PAS-004 Tablets Evaluating Long-term Use of a Pediatric Robotic Exoskeleton (P.REX/Agilik) to Improve Gait in Children With Movement Disorders NCT05726591NA1 site📍 Bethesda National Institutes of Health Clinical Center (CC) · Tests: EA-KAFO Multimodal Analysis of Endomyocardial Biopsies NCT07359690n/a1 site📍 Essen University Hospital, Essen A Gene Transfer Therapy Study to Evaluate the Safety of and Expression From Delandistrogene Moxeparvovec (SRP-9001) in Participants With Duchenne Muscular Dystrophy (DMD) - Non-Ambulatory Cohort NCT04626674Phase 112 sites📍 Little Rock📍 La Jolla📍 Los Angeles Sarepta Therapeutics, Inc. · Tests: delandistrogene moxeparvovec Plantar Stimulation, Whole-Body Vibration, and Baduanjin for Muscle Activation NCT07811674NA1 site📍 Guangzhou Nanfang Hospital, Southern Medical University · Tests: Whole-body Vibration Training,Whole-Body Vibration With Regional Plantar Stimulation,Baduanjin A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia NCT05849662Phase 1, Phase 221 sites📍 Phoenix📍 Los Angeles📍 San Francisco Therapeutic Advances in Childhood Leukemia Consortium · Tests: Trametinib, Azacitidine, Fludarabine, Cytarabine Self-Supporting Nasopharyngeal Airway (ssNPA) Treating Upper Airway Obstruction in Hypotonia NCT05527652NA1 site📍 Ann Arbor University of Michigan · Tests: Self-Supporting Nasopharyngeal Airway (ssNPA) A Study to Evaluate ARV-6723 Alone and With Pembrolizumab in Participants With Advanced Solid Tumors NCT07749586Phase 1, Phase 24 sites📍 New York📍 Huntersville📍 San Antonio Arvinas Inc. · Tests: ARV-6723, Pembrolizumab, SOC Clenbuterol to Target DUX4 in FSHD NCT06721299Phase 13 sites📍 Kansas City📍 Rochester📍 Seattle Jeffrey Statland · Tests: Clenbuterol A Study to Investigate the Safety, Tolerability, and Efficacy of SAR446268, an Adeno-associated Viral Vector-mediated Gene Therapy in Participants Aged 10 to 55 Years of Age With Non-congenital Myotonic Dystrophy Type 1 NCT06844214Phase 1, Phase 210 sites📍 Gainesville📍 Tampa📍 New York Sanofi · Tests: SAR446268
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