Search clinical trials for: Congenital Myasthenic Syndrome

Total 44153 results

argenx

Recruiting

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

Congenital Myasthenic Syndrome

Spain, 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
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
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
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
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
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
Enyo Pharma

Not yet recruiting

Vonafexor ALPort Syndrome Efficacy & Safety TRIAl-1 (ALPESTRIA-1) (ALPESTRIA-1)

Alport Syndrome

Spain, United States, France, Germany
Necmi Kadıoğlu Hospital

Recruiting

Caudal Anesthesia Versus Local Anesthesia in Hypospadias

Children | Hypospadias | Hypospadias, Coronal | Caudal Anesthesia

North Macedonia, Russian Federation, Turkey
Ultragenyx Pharmaceutical Inc

Not yet recruiting

Long-term Extension of GTX-102 in Angelman Syndrome

Angelman Syndrome

United States, Australia, Canada, Germany, United Kingdom, Israel, France, Spain
Spinogenix
Avance Clinical Pty Ltd.

Not yet recruiting

Study of SPG601 in Adult Men With Fragile X Syndrome

Fragile X Syndrome

United States
IRCCS Ospedale San Raffaele

Not yet recruiting

Validation of Human Drugs Target of Repurposed Drugs and Novel Therapies

Rare Diseases | Corneal Neovascularization | Limbal Stem Cell Deficiency | Aniridia | Neurotrophic Keratopathy | Ocular Cicatricial Pemphigoid | Ocular Graft-versus-host Disease | EEC Syndrome
Rigshospitalet, Denmark

Not yet recruiting

Enhancing Lung Health in Kids With Structural Lung Damage and Malformations: Azithromycin (AZI) for Airway Infection Prevention (TRALULALA-AZI)

Bronchiectasis | Bronchopulmonary Dysplasia (BPD) | Tracheomalacia | Cystic Lung Disease | Vascular Ring | Chronic Atelectasis
Riphah International University

Not yet recruiting

Effects of Lower Limb Fitness Program in Children With DS

Down Syndrome

Pakistan
Hanoi Medical University

Recruiting

Treatment Result of Zirconia Laminate Veneer

Tooth Abnormalities | Tooth Injuries | Teeth, Endodontically-Treated | Tooth Fracture | Tooth Crowding | Dislocation, Tooth | Cracked Tooth Syndrome

Vietnam
Riphah International University

Recruiting

Intrinsic Foot Muscle Training With and Without Hip Abductor Muscle Strengthening in Pronation Distortion Syndrome

Pronation Deformity of the Foot

Pakistan
Istanbul University - Cerrahpasa (IUC)

Not yet recruiting

Comparing Efficacies of Median Nerve Hydrodissection With Dexamethasone and Dextrose in Carpal Tunnel Syndrome

Carpal Tunnel Syndrome | Entrapment Neuropathy | Median Nerve Entrapment

Turkey
Peking University First Hospital

Recruiting

Establishment of a Multidimensional Prediction Model for the Natural Course of VHL Disease-related Renal Cell Carcinoma

Renal Cell Carcinoma | VHL Syndrome

China
GRIN Therapeutics, Inc.

Recruiting

A Study of Radiprodil on Safety, Tolerability, Pharmacokinetics, and Effect on Seizures and Behavioral Symptoms in Patients With TSC or FCD Type II

Tuberous Sclerosis Complex | Focal Cortical Dysplasia

Spain, United Kingdom, Australia, Belgium, Canada, Italy, Poland
Lawson Health Research Institute
Greenstone Biosciences, Inc.

Not yet recruiting

iPSC Biobank of Biomarkers Diversity in Cardiovascular Disease (INFERENCE)

Cardiovascular Diseases | Heart Failure | Cardiomyopathies | Congenital Heart Disease | Cerebrovascular Accident | Arrythmia | Cardiometabolic Syndrome
Harmony Biosciences, LLC

Recruiting

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome

United States
Octapharma

Recruiting

Study of IV Human Plasma-derived C1 Esterase Inhibitor Concentrate in Patients With Congenital C1-INH Deficiency for Treatment and Pre-procedure Preventing of Acute Hereditary Angioedema Attacks

Acute Hereditary Angio Edema

Romania
IRCCS Eugenio Medea

Recruiting

Structural-functional Connectome in Drug-resistant Epilepsies and Neurodevelopmental Syndromes With Epilepsy

Epilepsy | Angelman Syndrome | Dup15q Syndrome

Italy
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Prevalence and Characterization of Pain in RASopathies (3717)

Noonan Syndrome | Costello Syndrome | RASopathy | Cardio-Facio-Cutaneous Syndrome

Italy
Hasan Kalyoncu University

Recruiting

Effects of Orofacial Therapy and Therapeutic Yoga in Children With Down Syndrome

Down Syndrome

Turkey
Karamanoğlu Mehmetbey University

Recruiting

Physical Activity in Children With Wolff-Parkinson-White Syndrome

Wolff-Parkinson-White Syndrome

Turkey
Fondazione IRCCS Ca' Granda, Ospedale Maggiore...
Federico II University; Istituto Auxologico Italiano; Università degli Studi...

Recruiting

Maternal Genes and Epimutations: Beckwith-Wiedemann Syndrome & Reproductive Risks

Beckwith-Wiedemann Syndrome

Italy
Mauro H. Schenone

Not yet recruiting

Fetal Aortic Valvuloplasty for Evolving Hypoplastic Left Heart Syndrome

Hypoplastic Left Heart Syndrome

United States
Assistance Publique - Hôpitaux de Paris

Recruiting

Relationship Between Breathing and Attention in Children With Ondine Syndrome (OndineCo)

Ondine Syndrome | Congenital Central Hypoventilation Syndrome (CCHS)

France
IRCCS Eugenio Medea

Recruiting

Study of the Prevalence of Autistic Traits in Angelman Syndrome

Angelman Syndrome

Italy
GCS Ramsay Santé pour l'Enseignement et la Recherche

Not yet recruiting

Evaluation of the Effects of Cross Linking on the Biomechanical Characteristics of Skin Samples From Patients With Classic or Hypermobile Ehlers-Danlos Syndrome (SEDSKIN)

Ehlers-Danlos Syndrome

France
Centre Hospitalier Sud Francilien

Not yet recruiting

Infectious and Non-infectious Lower Respiratory Diseases in Children With Down Syndrome Followed in Pediatric Pulmonology Consultations in Ile-de-France (ARBT21)

Down Syndrome

France
Craig Erickson
Healx Limited

Recruiting

Placebo-Controlled, Single-Dose Challenge Study of Gaboxadol in Adult Males With Fragile X Syndrome (FXS)

Fragile X Syndrome

United States
Meyer Children's Hospital IRCCS

Recruiting

Immunodeficiency and Cancer: Identification of Congenital Immune System Defects Underlying Paediatric Lymphomas

Immune Deficiency

Italy
Maastricht University Medical Center

Recruiting

Qualitative Study in Patients With Genodermatoses and Healthcare Professionals on Reproductive Counselling

Quality of Life | Tuberous Sclerosis | Basal Cell Nevus Syndrome | Cutis Laxa | Epidermolysis Bullosa | Ichthyosis | Ectodermal Dysplasia | Albinism | Birt-Hogg-Dube Syndrome | Palmoplantar Keratoses

Netherlands
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Effect of RAS/MAPK Pathway Hyperactivation on Growth' and Bone' Profile of the RASopathies (3776)

Noonan Syndrome | Costello Syndrome | RASopathy | Cardio-Facio-Cutaneous Syndrome

Italy
ENCell

Recruiting

Evaluate the Safety and Efficacy of EN001 in Patients With Charcot-Marie-Tooth Disease Type 1A

Charcot-Marie-Tooth Disease Type 1A

Korea, Republic of
Copka Sonpashan

Not yet recruiting

Effect of Oral Feeding in Infants With Pierre Robin Syndrome

Pierre Robin Syndrome
University Health Network, Toronto

Not yet recruiting

RCT of Gut-directed Hypnotherapy in Ehlers-Danlos Syndromes and Generalized Hypermobility Spectrum Disorders

Irritable Bowel Syndrome | Gastrointestinal Dysfunction | Ehlers-Danlos Syndrome | Hypermobility Syndrome

Canada
University of Aarhus
Aarhus University Hospital

Recruiting

Lymphedema, Low-grade Inflammation and the Vasculature in Turner Syndrome (TSCOR_V)

Cardiovascular Diseases | Inflammation | Lymphedema | Turner Syndrome

Denmark
Qilu Hospital of Shandong University

Recruiting

Clemastine Fumarate in the Treatment of Neurodevelopmental Delays in Williams Syndrome

Child | Williams Syndrome | Neurodevelopmental Delay

China
Peking Union Medical College Hospital

Recruiting

Anesthesia Neurodevelopmental Impact in Congenital Scoliosis Children

General Anesthetics Toxicity

China
University of California, Los Angeles

Recruiting

Remote Assessment and Intervention for Behavior Problems in Kids With TSC (RAINBOW)

Tuberous Sclerosis

United States
John Elfar

Not yet recruiting

4AP to Delay Carpal Tunnel Release (CTR) (CTR)

Carpal Tunnel Syndrome | Compression Neuropathy

Search clinical trials for: Congenital Myasthenic Syndrome

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