Search clinical trials for: Congenital myotonia

Total 9258 results

AMO Pharma Limited

Recruiting

Safety and Efficacy of Tideglusib in Congenital or Childhood Onset Myotonic Dystrophy (REACH CDM X)

Congenital Myotonic Dystrophy

United States, Canada, Australia, New Zealand
Myotonic Dystrophy Foundation

Recruiting

Myotonic Dystrophy Family Registry (MDFR)

Myotonic Dystrophy | Myotonic Dystrophy 1 | Steinert's Disease | Congenital Myotonic Dystrophy | PROMM (Proximal Myotonic Myopathy) | Steinert Disease | Dystrophia Myotonica 1 | Myotonic Dystrophy 2 | Dystrophia Myotonica | Dystrophia Myotonica 2 | Myotonia Dystrophica | Myotonic Dystrophy, Congenital | Myotonic... and other conditions

United States
AMO Pharma Limited

Completed

Efficacy and Safety of Tideglusib in Congenital Myotonic Dystrophy

Congenital Myotonic Dystrophy

United States, Canada, New Zealand, Australia, United Kingdom
Grete Andersen, MD

Completed

Relations Between Myotonia and Fitness

Congenital Myotonia

Denmark
NMD Pharma A/S

Not yet recruiting

Safety and Efficacy of NMD670 in Adult Patients With Type 1 and Type 2 Charcot-Marie-Tooth Disease (SYNAPSE-CMT)

Charcot-Marie-Tooth Disease
Bristol-Myers Squibb

Not yet recruiting

A Study to Evaluate BMS-986470 in Healthy Volunteers and Participants With Sickle Cell Disease

Healthy Volunteers | Anemia, Sickle Cell

United States
Connecta Therapeutics, S.L.
Adknoma Health Research; Ministerio de Ciencia e Innovación, Spain; Hospital...

Recruiting

CTH120 First-in-Human Study: Single and Multiple Ascending Doses and Potential Food Interaction (FIH-CTH120)

Fragile X Syndrome

Spain
Children's Hospital of Fudan University

Recruiting

A Long-term Follow-up Study in Participants Who Received CS-101

Beta-Thalassemia

China
Zhuhai Tonbridge Medical Tech. Co., Ltd.

Not yet recruiting

Efficacy and Safety of the Liquid Embolic System (Tonbridge) for Endovascular Treatment of Cerebrovascular Malformations

Intracranial Arteriovenous Malformations

China
University of Malaya

Recruiting

Radiation Free Fistuloplasty is the Way Forward

Fistulas Arteriovenous

Malaysia
Sohag University

Not yet recruiting

Velopharyngeal Insufficiency Evaluation Post Cleft Palate Repair. Furlow With Buccinator Myomucosal Flap Versus Two Flap Palatoplasty

Cleft Palate

Egypt
Kafrelsheikh University

Not yet recruiting

PLR and NLR as Predictor Factors for Semen Abnormality

Abnormal Semen

Egypt
Shanghai 6th People's Hospital

Recruiting

Calcium Folinate Treatment of Spastic Paraplegia 56 (CFT-SPG56)

Hereditary Spastic Paraplegia

China
St. Jude Children's Research Hospital

Recruiting

Peripheral Arterial Tonometry and Neurocognition in Sickle Cell Disease

Sickle Cell Disease

United States
College of Staten Island, the City University of...
Duquesne University

Not yet recruiting

Using Technology for Prediction and Prevention of Infant Torticollis and Plagiocephaly

Torticollis
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Characterization and Contribution of Genome-wide DNA Methylation (DNA Methylation Episignatures) in Rare Diseases With Prenatal Onset (FOETEPISIGN)

Congenital Malformation | Rare Fetal Genetic Diseases

France
HeartWorks, Inc.

Recruiting

The Co-Op @ HeartWorks

Congenital Heart Disease

United States
Xuanwu Hospital, Beijing

Not yet recruiting

A Novel Classification of Sagittal Spinal Alignment to Aid Surgical Planning for Adult Spinal Deformity (CSSAASD)

Adult Spinal Deformity | Sagittal Deformity | Sagittal Imbalance | Sagittal Alignment

China
Oslo University Hospital

Recruiting

Intestinal Ischemia Biomarker in Patients With Chronic Mesenteric Ischemia

Chronic Mesenteric Ischemia | Median Arcuate Ligament Syndrome

Norway
Ahram Canadian University

Recruiting

Inspiratory Muscle Training in Children With Beta Thalasemia

Beta Thalassemia Major

Egypt
Radboud University Medical Center

Not yet recruiting

Methylphenidate in KBG Syndrome: N-of-1 Series (KBGS_N_of_1)

ADHD - Combined Type | Kbg Syndrome
Shanghai BDgene Co., Ltd.

Recruiting

A Phase 1 Study of Gene-modified Autologous Hematopoietic Stem Cell (BD211) Treating β-thalassemia Major

β-thalassemia

China
Stefano Vicari
Italian Institute of Technology (IIT)

Recruiting

Efficacy of Bumetanide to Improve Cognitive Functions in Down Syndrome

Down Syndrome

Italy
Oslo University Hospital
University of Oslo; Lovisenberg Diakonale Hospital

Recruiting

Laryngomalacia, Examinations and Quality of Life in Children Before and After Treatment With Follow-up After 1 Year

Quality of Life | Sleep Apnea, Obstructive | Sleep Apnea | Surgery | Pediatric Disorder | Laryngomalacia | Ear, Nose and Throat Disorder

Norway
University Hospital, Strasbourg, France

Recruiting

Optimizing the Management of Sickle Cell Patients on Hydroxyurea: The Value of Therapeutic Pharmacological Monitoring (OPTIMDREP)

Sickle Cell Disease

France
Inozyme Pharma

Not yet recruiting

ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency

Pseudoxanthoma Elasticum | Arterial Calcification | Gene Mutations | Ectonucleotide Pyrophosphatase/phosphodiesterase1 Deficiency | Autosomal Recessive Hypophosphatemic Rickets Type 2
Emory University
University of California, San Diego; Ventrix, Inc.

Not yet recruiting

Study of Intramyocardial Injection of Ventrix Bio Extracellular Matrix (VentriGel) to Assess the Safety and Feasibility in Pediatric Patients With Hypoplastic Left Heart Syndrome (HLHS)

Hypoplastic Left Heart Syndrome

United States
Acibadem University
Boston Children's Hospital; Ege University

Recruiting

PTEN Hamartoma Tumor Syndrome Pediatric Patient Registry

PTEN Hamartoma Tumor Syndrome | Macrocephaly Autism Syndrome

Turkey
Montreal Heart Institute

Not yet recruiting

Harnessing ECG Artificial Intelligence for Rapid Treatment and Accurate Interpretation, an Open Label Randomized Controlled Trial (HEART-AI)

Structural Heart Abnormality
Riphah International University

Not yet recruiting

Effects of Cuevas Medek Exercises on Trunk Control and Balance in Children With Down Syndrome

Down Syndrome

Pakistan
Riphah International University

Not yet recruiting

Effect of Group-Task-Oriented Training on GMFM and ADL in Children With DS

Downs Syndrome

Pakistan
Istanbul University

Not yet recruiting

Intraosseous Local Anesthesia System in Children With Molar Incisor Hypomineralization

Hypersensitivity | Molar Incisor Hypomineralization | Composite Restoration

Turkey
PYC Therapeutics

Not yet recruiting

SAD of IVT PYC-001 in OPA1 Mutation-Associated Autosomal Dominant Optic Atrophy (Sundew)

Autosomal Dominant Optic Atrophy | Kjer Optic Atrophy | OPA1 Gene Mutation | Hereditary Optic Atrophies
Azienda Ospedaliera di Perugia

Recruiting

Effects of the Application of a Hyper-oxygenated Gel After Oral Surgery

Pain | Trismus | Edema Face | Extracting Own Teeth | Molar, Fourth

Italy
Al-Azhar University

Not yet recruiting

The Effectiveness of Adhesive Versus Conventional Nasoalveolar Molding on Children With Unilateral Cleft Lip and Palate

Cleft Lip and Palate

Egypt
Hôpital Fribourgeois

Not yet recruiting

Cardiovascular and Endocrine Response to Muscular Training Program of Young Soccer Players Aged 14-18 Years (YoungSoccer)

Cardiovascular Abnormalities | Sport Injury | Hormone Disturbance
Arizona State University
American Speech-Language-Hearing Foundation

Recruiting

Proactive Speech and Language Intervention for Infants With Down Syndrome (BBC-DS)

Down Syndrome

United States
Hacettepe University

Recruiting

Exercise Capacity, Physical Fitness, and Physical Activity Levels in Children With CAH

Quality of Life | Congenital Adrenal Hyperplasia | Physical Activity | Exercise Capacity

Turkey
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
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

Recruiting

Efficacy of New Post Kasai ILBS Protocol in Biliary Atresia.

Biliary Atresia

India
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
St. Jude Children's Research Hospital
BioLineRx, Ltd.

Not yet recruiting

SCD Stem Cell Mobilization and Apheresis Using Motixafortide

Sickle Cell Disease

United States
Takeda

Recruiting

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

Thrombotic Thrombocytopenic Purpura (TTP)

Japan
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

Search clinical trials for: Congenital myotonia

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