Trial Readiness and Endpoint Assessment in Congenital and Childhood Myotonic Dystrophy
Conditions: CDM; ChDM Sponsors: Fondazione Serena Onlus - Centro Clinico NeMO Milano; Bambino Ges ù Hospital and Research Institute; Fondazione Policlinico Universitario Agostino Gemelli IRCCS; IRCCS Istituto delle Scienze Neurologiche di Bologna; IRCCS Fondazione Stella Maris; IRCCS Istituto Neurologico Mondino, Pavia, Italy; Azienda Ospedaliera Città della Salute e della Scienza di Torino; F ondazione I.R.C.C.S. Istituto Neurologico Carlo Besta; Istituto Giannina Gaslini Active, not recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 23, 2024 Category: Research Source Type: clinical trials

An Observational Study Comparing Delandistrogene Moxeparvovec With Standard of Care in Participants With Duchenne Muscular Dystrophy
Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: delandistrogene moxeparvovec; Drug: Standard of Care Sponsors: Sarepta Therapeutics, Inc. Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 21, 2024 Category: Research Source Type: clinical trials

Evaluation of Cognitive Functions in 20 Patients With Type 1 Myotonic Dystrophy With Virtual Reality Approach
Conditions: Myotonic Dystrophy 1 Interventions: Diagnostic Test: Type 1 myotonic dystrophy Sponsors: Centre Hospitalier Universitaire de Nice Withdrawn (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 21, 2024 Category: Research Source Type: clinical trials

Autonomic Dysfunction in Temporal Lobe Epilepsy and SUDEP
Conditions: Temporal Lobe Epilepsy Interventions: Diagnostic Test: Sympathetic skin response, heart rate variability test, quantitative EEG Sponsors: Cairo University Completed (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 21, 2024 Category: Research Source Type: clinical trials

Plasma Rich in Growth Factors in Corneal Endothelial Transplantation
Conditions: Fuchs ' Endothelial Dystrophy; Corneal Edema Interventions: Procedure: PRGF Sponsors: Price Vision Group Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 15, 2024 Category: Research Source Type: clinical trials

Effects of a Supervised Rehabilitation Program on Disease Severity in Spastic Ataxias
Conditions: ARSACS; SPG7 Interventions: Procedure: IMPACT - rehabIlitation prograM for sPAstiC aTaxias Sponsors: Universit é du Québec à Chicoutimi; McGill University; Université de Sherbrooke; Université du Québec a Montréal; Laval University; University of Calgary; Corporation de recherche et d ' action sur les maladies h éréditaires (CORAMH); Cégep de Jonquière; Centre intégré universitaire de santé et de services sociaux du Saguenay-Lac-St-Jean; Muscular Dystrophy Canada; University of Alberta; Integrated University Health and Social Services Center of the Capitale-Nationale Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 15, 2024 Category: Research Source Type: clinical trials

Edgewise Receives U.S. FDA Fast Track Designation for EDG-5506 for the Treatment of Duchenne Muscular Dystrophy
BOULDER, Colo.--(BUSINESS WIRE) February 13, 2024 -- Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track... (Source: Drugs.com - Clinical Trials)
Source: Drugs.com - Clinical Trials - February 13, 2024 Category: Pharmaceuticals Source Type: clinical trials

A Trial to Learn More About an Experimental Gene Therapy Called Bidridistrogene Xeboparvovec (SRP-9003) as a Possible Treatment for Limb Girdle Muscular Dystrophy 2E/R4 (EMERGENE)
Conditions: Limb-girdle Muscular Dystrophy Interventions: Biological: SRP-9003; Drug: Glucocorticoid Sponsors: Sarepta Therapeutics, Inc. Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 7, 2024 Category: Research Source Type: clinical trials

Ph2 Open-label Study of AOC 1044 in Duchenne Muscular Dystrophy Participants With Mutations Amenable to Exon44 Skipping
Conditions: DMD; Duchenne Muscular Dystrophy; Duchenne; Exon 44 Interventions: Drug: AOC 1044 Sponsors: Avidity Biosciences, Inc. Enrolling by invitation (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 6, 2024 Category: Research Source Type: clinical trials

The Success of Lumbar Sympathetic Ganglion Block Using Lower Extremity Perfusion Index
Conditions: Chronic Pain; Complex Regional Pain Syndromes; Peripheral Neuropathy; Post Herpetic Neuralgia Interventions: Procedure: Lumbar Sympathetic Ganglion Block Sponsors: Jeongsoo Kim Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 5, 2024 Category: Research Source Type: clinical trials

A Gene Transfer Therapy Study to Evaluate the Safety and Efficacy of Delandistrogene Moxeparvovec (SRP-9001) Following Imlifidase Infusion in Participants With Duchenne Muscular Dystrophy (DMD) Determined to Have Pre-existing Antibodies to Recombinant Adeno-Associated Virus Serotype (rAAVrh74)
Conditions: Duchenne Muscular Dystrophy Interventions: Genetic: delandistrogene moxeparvovec; Biological: imlifidase Sponsors: Sarepta Therapeutics, Inc.; Hansa Biopharma AB Enrolling by invitation (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - February 5, 2024 Category: Research Source Type: clinical trials

18F-mFBG Expression in Neural Crest Tumors and Organs Innervated by the Sympathetic Nervous System
Conditions: Neuroblastoma; Pheochromocytoma; Paraganglioma Interventions: Drug: 18F-mFBG Sponsors: Innervate Radiopharmaceuticals LLC (Formerly: Illumina Radiopharmaceuticals LLC); Albert Einstein College of Medicine Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - January 31, 2024 Category: Research Source Type: clinical trials

Magnetic Resonance Imaging and Ultrasound Comparison With Load Evaluation
Conditions: Muscular Dystrophy, Facioscapulohumeral Interventions: Diagnostic Test: Muscle ultrasound with surface electromyography and dynamometry; Diagnostic Test: MRI scan Sponsors: Radboud University Medical Center; Solve FSHD Recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - January 26, 2024 Category: Research Source Type: clinical trials

A Bicentric, Randomized, Double Blind, Placebo-controlled Pilot Study to Evaluate the Efficacy and Safety of Satralizumab in FSHD1
Conditions: Facioscapulohumeral Muscular Dystrophy 1 Interventions: Drug: Satralizumab Prefilled Syringe; Drug: Placebo Comparator Sponsors: Centre Hospitalier Universitaire de Nice Not yet recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - January 25, 2024 Category: Research Source Type: clinical trials

Non Motoric Reactions in Vojta Reflex Locomotion
Conditions: Locomotion Disorder, Neurologic Interventions: Other: Reflex locomotion according to Vojta Sponsors: Charles University, Czech Republic Active, not recruiting (Source: ClinicalTrials.gov)
Source: ClinicalTrials.gov - January 25, 2024 Category: Research Source Type: clinical trials