Search clinical trials for: myotonia congenita

Total 9230 results

University of Campania "Luigi Vanvitelli"
Fondazione IRCCS Ca' Granda, Ospedale Maggiore Policlinico; University of Milan and other collaborators

Recruiting

The PRO-FUTURE Project (PRO-FUTURE)

Chronic Kidney Diseases | Ureteropelvic Junction Obstruction

Italy
University Ghent
Research Foundation Flanders

Not yet recruiting

Effect and Cost-utility of of High Intensity vs. Low Intensity Speech Intervention in Children With Cleft Palate

Cleft Lip and Palate | Cleft Palate Children | Speech Therapy | Speech Disorders in Children
Chongqing Medical University

Recruiting

Effect of Bilirubin on Prognosis in Heart Failure With Preserved Ejection Fraction

Heart Failure With Preserved Ejection Fraction | Prognathism

China
Regeneron Pharmaceuticals
Intellia Therapeutics

Not yet recruiting

A Trial to Learn if REGV131-LNP1265 is Safe and Works to Help the Body Make Clotting Factor in Pediatric, Adolescent and Adult Patients With Hemophilia B (BEYOND-9)

Hemophilia B
IRCCS Eugenio Medea

Recruiting

Myotonic Dystrophy Type 1 Congenital and Juvenile Form: From Diagnosis to Rehabilitation [MDCJ-NeuBeRe] (MDCJ-NeuBeRe)

Myotonic Dystrophy 1

Italy
Assiut University

Not yet recruiting

Thyroid Function , HbA1c in Relation to Ferritin Level in Adullt Patient e B Thalassemia

B Thalassemia
Didier TCHETCHE
Abbott

Not yet recruiting

NavIIcusp: Bicuspid Aortic Valve Stenosis With Navitor Platform International Experience

Bicuspid Aortic Valve

France
Clinic for Special Children

Recruiting

WiTNNess - TNNT1 Myopathy Natural History Study (WiTNNess)

Myopathy | Myopathy; Hereditary | Myopathies, Nemaline | TNNT1-associated Myopathy | Infantile-onset Nemaline Rod Myopathy | Myopathy, Rod | Amish Nemaline Myopathy | Nemaline Myopathy 5 | NEM5 | Genetic Muscle Disease | Recessive Hereditary Disorder (Autosomal) | ANM

United States
University College, London
Washington University School of Medicine; University of Washington; Alzheimer... and other collaborators

Not yet recruiting

A Trial Evaluating the Effect of NIO752 on Tau Synthesis Measured by a Process Known as SILK (NIO-SILK)

Alzheimer Disease | Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 1 (Disorder) | Autosomal Dominant Alzheimer Disease Due to Mutation of Presenilin 2 (Disorder) | Autosomal Dominant Alzheimer Disease Due to Mutation of Amyloid Precursor Protein (Disorder)
Kocaeli University

Recruiting

Maternal Consequences of Membrane Stripping

Maternal Care for Other Abnormalities of Cervix

Turkey
Fondation Hôpital Saint-Joseph

Not yet recruiting

Procedural Pain Management by Multimodal Sedation Analgesia Combining Hypnosis in Children With Congenital Heart Disease (PEACE-HYPNO)

Congenital Heart Disease | Hypnosis | Procedural Pain | Heart Surgery
University of Pittsburgh
National Heart, Lung, and Blood Institute (NHLBI)

Not yet recruiting

Peer Support for Adolescents and Emerging Adults With Sickle Cell Pain (PRESENCE)

Pain | Sickle Cell Disease
Stanford University
National Institutes of Health (NIH)

Not yet recruiting

Cardiac Simulator for Surgical Planning

Congenital Heart Disease in Children
Sensorion

Not yet recruiting

A Phase I/II Clinical Trial With SENS-501 in Children Suffering From Severe to Profound Hearing Loss Due to Otoferlin (OTOF) Mutations (AUDIOGENE)

Otorhinolaryngologic Diseases | Ear Diseases | Hearing Disorders | Deafness | Hearing Loss, Sensorineural | Congenital Deafness | DFNB9 | OTOF Gene Mutation

France
Lawson Health Research Institute
Greenstone Biosciences, Inc.

Recruiting

iPSC Biobank of Biomarkers Diversity in Cardiovascular Disease (INFERENCE)

Cardiovascular Diseases | Heart Failure | Cardiomyopathies | Congenital Heart Disease | Cerebrovascular Accident | Arrythmia | Cardiometabolic Syndrome

Canada
Cairo University

Not yet recruiting

En-mass Retraction by Lingual Retractor Versus Clear Aligner Appliances

Maxillary Prognathism

Egypt
Harmony Biosciences, LLC

Recruiting

A Study of Pitolisant in Patients With Prader-Willi Syndrome

Prader-Willi Syndrome

United States
St. Jude Children's Research Hospital
National Heart, Lung, and Blood Institute (NHLBI)

Recruiting

School Readiness Intervention for Preschool Children With Sickle Cell Disease

Sickle Cell Disease

United States
Children's Hospital of Philadelphia

Not yet recruiting

ALS20-101 Lentiviral Gene Therapy for Beta Thalassemia

Beta-Thalassemia

United States
Dan Bing

Not yet recruiting

Genetic Feature of Congenital Hearing Loss in Chinese Population

Congenital Hearing Loss | Congenital Deafness
Centre Hospitalier Universitaire de Besancon

Not yet recruiting

EPBONF : Evaluation of the Safety and Effectiveness of a Medical Device Aimed at Guiding Orbito-naso-frontal Band Surgery, for the Treatment of Craniostenoses (EPBONF)

Craniosynostoses
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Perinatal Thoraco-abdomino-pelvic Tumors Study (CONTRAST)

Perinatal Solid Tumors of the Thoraco-abdomino-pelvic Region

France
Editas Medicine, Inc.

Not yet recruiting

A Long-Term Follow-Up Study of Participants With Sickle Cell Disease or Transfusion Dependent β-Thalassemia Who Received EDIT-301

Sickle Cell Disease | Hemoglobinopathies | Transfusion-dependent Beta-Thalassemia
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
University Hospital, Toulouse

Not yet recruiting

Efficacy of Injectable Gentamicin in Hereditary Ichthyosis (GENTIC)

Ichthyosis

France
Universidad Nacional Autonoma de Mexico

Recruiting

Effects of Different Remineralization Agents on Molar Incisor Hypomineralization Defects: a Randomized Clinical Study

Molar Incisor Hypomineralization

Mexico
Children's National Research Institute
The Hospital for Sick Children; Ann & Robert H Lurie Children's Hospital of... and other collaborators

Recruiting

Sickle Cell Disease Transplant Using a Nonmyeloablative Approach for Patients With Anti-donor Red Cell AntibodY (SUN-RAY)

Sickle Cell Disease

United States
Children's Hospital of Fudan University

Not yet recruiting

Phenotype and Multi-omics Analysis of Children With Congenital Diarrhea and Enteropathy in China

Diarrhea Infantile
Karamanoğlu Mehmetbey University

Not yet recruiting

Knowledge, Attitudes and Practices of Paediatric Cardiologists About Cardiac Rehabilitation in Children With Congenital Heart Disease

Physician's Role
IRCCS Eugenio Medea

Recruiting

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

Epilepsy | Angelman Syndrome | Dup15q Syndrome

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

Not yet recruiting

Natural History Study of Children With LAMA2-related Dystrophies (LAMA2)

Merosin Deficient Congenital Muscular Dystrophy

France
Fondazione Policlinico Universitario Agostino Gemelli...

Recruiting

Prevalence and Characterization of Pain in RASopathies (3717)

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

Italy
Assistance Publique - Hôpitaux de Paris

Not yet recruiting

Comparative Health Status and Quality of Life of Patients With Sickle Cell Disease (SCD) Who Underwent Matched-sibling Hematopoietic Stem Cell Transplantation Versus Non Transplanted SCD Case-control Patients (TRANSPLANTORN2)

Sickle Cell Disease
Hasan Kalyoncu University

Recruiting

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

Down Syndrome

Turkey
Hans Bahlmann
Linkoeping University

Recruiting

Spinal Morphine or Intravenous Lidocaine in Robot-assisted Upper Urologic Surgery (SMILe)

Renal Cancer | Ureter Cancer | Other Specified Disorders of Kidney and Ureter | Benign Neoplasm of Ureter | Calculus of Kidney and Ureter | Ureteric Reflux | Congenital Ureteric Anomaly | Benign Renal Neoplasm

Sweden
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
Vertex Pharmaceuticals Incorporated

Recruiting

A Phase 1 Study to Evaluate Safety, Tolerability, and Pharmacokinetics (PK) of VX-407 in Healthy Participants

Autosomal Dominant Polycystic Kidney Disease (ADPKD)

United States
China National Center for Cardiovascular Diseases
Peking University First Hospital; Beijing Children's Hospital

Recruiting

Etablishment of Follow-up System and End-Stage Heart Registration Platform for Pediatric Heart Failure

Heart Failure | End-stage Heart Failure | Congenital

China
ICE Intelligent Healthcare Co., Ltd

Not yet recruiting

Cardiac Interventional ICE Imaging Trial (INTELICE)

Atrial Fibrillation | Arrhythmias, Cardiac | Atrial Septal Defect | Patent Foramen Ovale | Valve Disease, Heart | Supraventricular Tachycardia | Structural Heart Diseases

China
Mauro H. Schenone

Not yet recruiting

Fetal Aortic Valvuloplasty for Evolving Hypoplastic Left Heart Syndrome

Hypoplastic Left Heart Syndrome

United States
Hacettepe University

Not yet recruiting

Investigation of the Effects of Exercise and Kinesiological Taping on Spinal Deformities and Serum Markers in Individuals With Scoliosis

Scoliosis | Scoliosis Idiopathic | Musculoskeletal Deformity
Cairo University

Not yet recruiting

Association Between Molar Incisor Hypomineralization And Type I Diabetes Milletus. (MIH and TIDM)

Molar Incisor Hypomineralization
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
Istituto Auxologico Italiano

Recruiting

Early Detection of Renal Abnormalities in Metabolically Healthy and Unhealthy Weight Excess" (OB-KID)

Obesity

Italy
IRCCS Eugenio Medea

Recruiting

Study of the Prevalence of Autistic Traits in Angelman Syndrome

Angelman Syndrome

Italy
Seattle Children's Hospital
University of Washington; Ohio State University; Nationwide Children's Hospital; Children's Hospital Los Angeles

Not yet recruiting

Book Sharing for Toddlers With Clefts (BOOST)

Cleft Lip and Palate | Cleft Palate
UMC Utrecht
Karolinska University Hospital; Erasmus Medical Center; Great Ormond Street Hospital... and other collaborators

Not yet recruiting

Primary Posterior Tracheopexy Prevents Tracheal Collapse (PORTRAIT)

Oesophageal Atresia | Tracheomalacia | Oesophageal Atresia With Tracheo-Oesophageal Fistula
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
Assiut University

Not yet recruiting

Brachio Basilic Arterio Venous Fistula One Stage vs Two Stage

Arterio-venous Fistula

Search clinical trials for: myotonia congenita

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