Search clinical trials for: congenital myasthenia

Total 9408 results

argenx

Not yet recruiting

A Phase 1b Study to Investigate Safety and Tolerability of ARGX-119 in Adult Participants With DOK7-Congenital Myasthenic Syndromes (CMS)

Congenital Myasthenic Syndrome
argenx

Recruiting

A Natural History Study in Participants With DOK7 Congenital Myasthenic Syndromes (CMS)

Congenital Myasthenic Syndrome

Spain, Italy, France, United States, Canada
Centre Hospitalier Universitaire de Liege
Sanofi; Takeda; University of Liege; Orchard Therapeutics; Centre Hospitalier Régional... and other collaborators

Recruiting

Baby Detect : Genomic Newborn Screening

Congenital Adrenal Hyperplasia | Hemophilia A | Hemophilia B | Mucopolysaccharidosis I | Mucopolysaccharidosis II | Cystic Fibrosis | Alpha 1-Antitrypsin Deficiency | Sickle Cell Disease | Fanconi Anemia | Chronic Granulomatous Disease | Wilson Disease | Severe Congenital Neutropenia | Ornithine Transcarbamylase... and other conditions

Belgium
Sanguine Biosciences

Recruiting

Tissue Repository Providing Annotated Biospecimens for Approved Investigator-directed Biomedical Research Initiatives

Heart Diseases | Stroke | Lymphoma | Multiple Sclerosis | Kidney Diseases | Diabetes | Leukemia | Age-Related Macular Degeneration | Cancer | Arthritis | Schizophrenia | Thyroid Diseases | Parkinson Disease | Hepatitis | Vasculitis | Psoriasis | Asthma | Amyloidosis | Fibromyalgia | Graves Disease | Alzheimer Disease | Celiac Disease | Cirr... and other conditions

United States
Tongji Hospital
Nanjing IASO Biotechnology Co., Ltd.

Recruiting

Safety and Efficacy of CT103A Cells for Relapsed/Refractory Antibody-associated Inflammatory Diseases of the Nervous System (CARTinNS)

Multiple Sclerosis | Autoimmune Diseases of the Nervous System | Autoimmune Diseases | Myasthenia Gravis | Neuromyelitis Optica Spectrum Disorder | Autoimmune Encephalitis | Idiopathic Inflammatory Myopathies | Chronic Inflammatory Demyelinating Polyradiculoneuropathy | Myelin Oligodendrocyte Glycoprotein... and other conditions

China
Sanford Health
National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators

Recruiting

Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)

Mitochondrial Diseases | Retinitis Pigmentosa | Myasthenia Gravis | Eosinophilic Gastroenteritis | Multiple System Atrophy | Leiomyosarcoma | Leukodystrophy | Anal Fistula | Spinocerebellar Ataxia Type 3 | Friedreich Ataxia | Kennedy Disease | Lyme Disease | Hemophagocytic Lymphohistiocytosis | Spinocerebellar Ataxia... and other conditions

United States, Australia
Cure CMD

Recruiting

Congenital Muscle Disease Study of Patient and Family Reported Medical Information (CMDPROS)

Emery-Dreifuss Muscular Dystrophy | Congenital Myasthenic Syndrome | Limb-Girdle Muscular Dystrophy | Congenital Muscular Dystrophy With ITGA7 (Integrin Alpha-7) Deficiency | Alpha-Dystroglycanopathy (Congenital Muscular Dystrophy and Abnormal Glycosylation of Dystroglycan With Severe Epilepsy) and other conditions

United States
Vern C. Juel, M.D.

No longer available

Treatment Use of 3,4-Diaminopyridine

Myasthenic Syndromes, Congenital

United States
Casa Colina Hospital and Centers for Healthcare

Completed

Exploring Accessible Beauty for Individuals With Upper Extremity Deficits

Multiple Sclerosis | Amyotrophic Lateral Sclerosis | Muscular Dystrophies | Graves Disease | Myasthenia Gravis | Traumatic Brain Injury | Stroke, Ischemic | Stroke, Acute | Stroke Hemorrhagic | SCI - Spinal Cord Injury | Guillain-Barre Syndrome | Lupus Erythematosus | Stroke (CVA) or TIA | Ehlers-Danlos Syndromes...

United States
Catalyst Pharmaceuticals, Inc.

No longer available

Expanded Access Study Amifampridine Phosphate in Lambert-Eaton Myasthenic Syndrome (LEMS),Congenital Myasthenic Syndrome (EAP-001)

Lambert-Eaton Myasthenic Syndrome | Congenital Myasthenic Syndrome | Nystagmus, Acquired

United States
Ricardo Maselli
Catalyst Pharmaceuticals, Inc.

No longer available

Treatment Use of 3,4 Diaminopyridine in Congenital Myasthenia

Congenital Myasthenic Syndrome

United States
Institut de Myologie, France
Association Française contre les Myopathies (AFM), Paris

Completed

Exercise in Autoimmune Myasthenia Gravis and Myasthenic Syndromes (MYaEX)

Myasthenia Gravis | Lambert-Eaton Myasthenic Syndrome | Congenital Myasthenic Syndrome | Autoimmune Myasthenia Gravis

France
Xperiome
The Touro College and University System

Withdrawn

Medication Adherence and Non-adherence in Adults With Rare Disease

Hemophilia A | Hemophilia B | Idiopathic Pulmonary Fibrosis | Myasthenia Gravis | Cystic Fibrosis | Sickle Cell Disease
Lahey Clinic

Approved for marketing

3,4-Diaminopyridine Use in Lambert-Eaton Myasthenic Syndrome(LEMS) and Congenital Myasthenic Syndromes (CMS) (LEMS)

Lambert-Eaton Myasthenic Syndrome | Congenital Myasthenic Syndrome

United States
Catalyst Pharmaceuticals, Inc.

Completed

Amifampridine Phosphate for the Treatment of Congenital Myasthenic Syndromes

Myasthenic Syndromes, Congenital

United States
Mayo Clinic

Completed

Spontaneous Coronary Artery Dissection (SCAD) and Autoimmunity

Multiple Sclerosis | Rheumatoid Arthritis | Systemic Lupus Erythematosus | Uveitis | Vasculitis | Psoriasis | Graves Disease | Systemic Sclerosis | Celiac Disease | Crohn Disease | Type 1 Diabetes Mellitus | Myasthenia Gravis | Ulcerative Colitis | Dermatomyositis | Polymyositis | Vitiligo | Hepatitis, Autoimmune | Polymyalgia... and other conditions

United States
Oregon Health and Science University
Jacobus Pharmaceutical

No longer available

3,4-Diaminopyridine for Lambert-Eaton Myasthenic Syndrome (LEMS) and Congenital Myasthenia (CM)

Lambert-Eaton Myasthenic Syndrome (LEMS) | Congenital Myasthenia (CM)

United States
Mayo Clinic

Completed

Efficacy of Albuterol in the Treatment of Congenital Myasthenic Syndromes

Congenital Myasthenic Syndrome

United States
Brigham and Women's Hospital

Unknown

Study of an Intervention to Improve Problem List Accuracy and Use (MAPLE)

Coronary Artery Disease | Stroke | Glaucoma | Hypertension | Diabetes | Breast Cancer | Renal Insufficiency | Hemophilia | Hypothyroidism | Asthma | Renal Failure | Hyperthyroidism | Osteoporosis | Myasthenia Gravis | Sickle Cell Disease | Osteopenia | COPD | Congestive Heart Failure | Attention Deficit Disorder With Hyperactivity

United States
Institut de Myologie, France
Assistance Publique - Hôpitaux de Paris

Completed

Pregnancy Outcomes in Congenital Myasthenie Syndrome (POCoMS)

Congenital Myasthenic Syndrome
Hadassah Medical Organization

Unknown

Ephedrine for the Treatment of Congenital Myasthenia

Myasthenic Syndromes, Congenital

Israel
University Health Network, Toronto

Completed

Intravenous Immune Globulin Treatment Compared to Placebo in Patients With Myasthenia Gravis

Myasthenia Gravis

Canada
University of Seville

Not yet recruiting

Relationship of Muscular Agenesis: Thin Plantar and Palmar Long as a Biomarker of Injury and Performance Marker in the Lower Limb and Upper Limb Respectively (MA-TP-PL)

Abnormality, Congenital

Spain
Shanghai Jiaolian Drug Research and Development...
Shanghai Pharmaceuticals Holding Co., Ltd

Not yet recruiting

A Clinical Study to Evaluate Safety, Tolerability and Pharmacokinetics of SV001 in Chinese Healthy Adult Volunteers.

Generalized Myasthenia Gravis

China
Taipei Tzu Chi Hospital, Buddhist Tzu Chi Medical...

Recruiting

Ultrasound to Assess Sarcopenia in Prader Willi Syndrome (PWS)

Metabolic Syndrome | Sarcopenia | Prader-Willi Syndrome

Taiwan
Semmelweis University
Új Nemzeti Kiválóság Program

Recruiting

Assessing Virtual Reality for Perioperative Anxiolysis and Postoperative Pain Modulation in Adolescents Undergoing MIRPE Surgery

Pectus Excavatum | Pectus Deformity

Hungary
Institute of Liver and Biliary Sciences, India

Not yet recruiting

Efficacy of New Post Kasai ILBS Protocol in Biliary Atresia.

Biliary Atresia

India
Xuanwu Hospital, Beijing

Recruiting

National, Multicentric Registry Study on Neuroimmunological Diseases in China (NIDBase)

Multiple Sclerosis | Myasthenia Gravis | Autoimmune Encephalitis | Acute Disseminated Encephalomyelitis | NMO Spectrum Disorder | Myelin Oligodendrocyte Glycoprotein Antibody-associated Disease

China
Nantes University Hospital
University of Angers - Pays de la Loire psychology laboratory

Not yet recruiting

Characterization and Support for Neurodevelopmental Disorders Associated With Congenital Heart Defects (CATAMARAN Ped)

Congenital Heart Defects | Neurodevelopmental Disorder

France
Rofaida Hassan Ahmed

Not yet recruiting

EKLF Gene Expression in β-thalassemia

β-thalassemia
Teva takeda

Not yet recruiting

A Survey of Recombinant ADAMTS13 in Participants With Congenital Thrombotic Thrombocytopenic Purpura

Thrombotic Thrombocytopenic Purpura (TTP)
St. Jude Children's Research Hospital
BioLineRx, Ltd.

Not yet recruiting

SCD Stem Cell Mobilization and Apheresis Using Motiixafortide

Sickle Cell Disease

United States
Heinrich-Heine University, Duesseldorf

Recruiting

Patient Observation With Environmental and Wearable Sensors in Myasthenia Gravis (POWER-MG)

Myasthenia Gravis | Lambert-Eaton Myasthenic Syndrome | Myasthenic Syndrome

Germany
Sohag University

Not yet recruiting

Surgical Treatment of DDH With and Without Capsulorrhaphy

Developmental Dysplasia of the Hip

Egypt
Assiut University

Not yet recruiting

Screening of Congenital Anomalies of Kidney

Kidney Congenital Anomalies
Novartis Pharmaceuticals

Not yet recruiting

A Study to Investigate the Efficacy and Safety of Crizanlizumab (5 mg/kg) Compared With Placebo in Adolescent and Adult Sickle Cell Disease Patients Who Experience Frequent Vaso-Occlusive Crises (SPARKLE) (SPARKLE)

Sickle Cell Disease
Başakşehir Çam & Sakura City Hospital

Not yet recruiting

Serratus Anterior Plane Block and Transthoracic Plane Block in Pectus Surgery

Pectus Excavatum

Turkey
Xuanwu Hospital, Beijing

Not yet recruiting

Subiculum Electrical Stimulation for Temporal Lobe Epilepsy With Biliteral Hippocampus Sclerosis(SESTB)

Epilepsy, Drug Resistant

China
Beijing Tongren Hospital
The First Affiliated Hospital with Nanjing Medical University

Not yet recruiting

Efficacy and Safety of Sugammadex in Thoracoscopy Thymectomy for Chinese Adults With Myasthenia Gravis

Reversal of Neuromuscular Blockade

China
West China Hospital

Recruiting

Use of Bleomycin in the Sclerotherapy of Lymphatic Malformations for Pediatric Patients

Lymphatic Malformation

China
UCB Biopharma SRL

Not yet recruiting

An Open-label Extension Study to Evaluate Subcutaneous Zilucoplan in Pediatric Participants With Generalized Myasthenia Gravis (ziMyG+)

Generalized Myasthenia Gravis
Boston Children's Hospital

Not yet recruiting

Extracellular Vesicle Micro RNA Profiling in Congenital Heart Disease: Fetal-Maternal Regulation in Neonatal Thrombosis (EVmiRNA)

Thrombosis | Congenital Heart Disease | Single-ventricle

United States
Cantonal Hospital Graubuenden
University of Zurich

Not yet recruiting

Short-term Effects of an SGLT2 Inhibitor on Divalent Ions in Autosomal Dominant Polycystic Kidney Disease (SIDIA)

Autosomal Dominant Polycystic Kidney Disease

Switzerland
Novo Nordisk A/S

Not yet recruiting

A Research Study of the Effect of Food on Etavopivat in Healthy Participants

Healthy Volunteers Sickle Cell Disease, Thalassemia

United States
Assiut University

Not yet recruiting

Detection of Krupple Like Factor -1(KLF1/ EKLF) DNA Mutations in Beta Thalassemia Patients

EKLF Mutations in Beta Thalassemia Patients
Ankara Yildirim Beyazıt University

Not yet recruiting

The Effect of Structured Transition Care Model Applied to Adolescents With Congenital Heart Disease

Congenital Heart Disease in Adolescence

Turkey
University Hospital, Montpellier

Not yet recruiting

Feasibility and Efficiency of Screening for Neurodevelopmental Disorders by an Advanced Practice Nurse in Children With Congenital Heart Disease (NEURODEV-IPA)

Neurodevelopmental Disorders | Congenital Heart Disease in Children

France
Tanta University

Recruiting

General Anesthesia Versus Sedation By Dexmedetomidine and Ketamine With Local Infiltration for Percutaneous Transcatheter Closure of Atrial Septal Defect in Pediatric Patients

Atrial Septal Defect | Ketamine | General Anesthesia | Dexmedetomidine | Local Infiltration

Egypt
University Hospital, Caen

Recruiting

CORRECTION OF EAR DEFORMITIES IN NEWBORNS BY MODELING, COMPARISON OF TWO PROTOCOLS (MOLDEAR)

Ear Malformation

France
University of Sao Paulo

Not yet recruiting

Interventions to Control Hypersensitivity Pain in Teeth With Insisive Molar Hypomineralization (LBIARHMI)

Molar Incisor Hypomineralization

Search clinical trials for: congenital myasthenia

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