Clinical Trials on Pompe Disease (Late-onset)

Chronic Pain | Inguinal Hernia | Ventral Hernia | Umbilical Hernia | Femoral Hernia | Acute Post Operative Pain | Recrrence Rate

Russian Federation

Chronic Atypical Neutrophilic Dermatosis With Lipodystrophy and Elevated Temperature (CANDLE) | Juvenile Dermatomyositis (JDM) | Stimulator of Interferon Genes (STING)-Associated Vasculopathy With Onset During Infancy (SAVI) | Aicardi-Goutières Syndrome (AGS)

United States, United Kingdom

Enterocolitis, Necrotizing | Retinopathy of Prematurity | Bronchopulmonary Dysplasia | Early-Onset Sepses, Neonatal | Small for Gestational Age Infant | Periventricular Haemorrhage Neonatal

Objective Response Rate

China

Lung Disease | Cerebral Aneurysm | Macroglossia | Cardiac Diseases | Obstructive Sleep Apnoea | Muscular Weakness | Hepato-splenomegaly

Germany, India, Sri Lanka

Dysphagia | Aspiration | Brief Resolved Unexplained Event (BRUE) | Apparent Life Threatening Event (ALTE)

United States

Cervical Cancer Screening | Breast Cancer Screening | Cholesterol Screening | Asthma Admission Rate | Diabetes Management

United States

Aspirin as an Adjuvant Therapy, to Observe Its Effect on the Disease Free Survival Rate of Patients With Esophageal Squamous Cell Carcinoma

China

Dysphagia | Xerostomia | Sjögren's Syndrome | Salivary Flow Rate

Turkey

Type 1 Diabetes With a Subcutaneous Insulin Pump | Adjustment of the Recommended Basal Insulin Flow Rate in the Event of Physical Activity | Adjustment of the Recommended Prandial Insulin in the Event of Physical Activity

France

Respiratory Depression | Neonatal Resuscitation | Birth Asphyxia | Fetal Heart Rate Abnormalities

Tanzania

Acute Myeloid Leukemia | Hodgkin Disease | Osteosarcoma | Non Hodgkin Lymphoma | Late Effects | Ewing's Sarcoma | Testicular Germ Cell Tumor Mixed | Non-Metastatic Breast Carcinoma | Soft Tissue Sarcoma, Adult, Stage IIC

France

Depression | Exercise Addiction | Perfectionism | Binge Eating | Body Image | Eating Disorder | Resting Metabolic Rate | Dietary Deficiency | Bone Mineral Density, Low, Susceptibility to | Exercise-Related Amenorrhea | Dietary Amenorrhea

Norway

Fabry Disease | Niemann-Pick Disease | Gaucher Disease | Pompe Disease | Lysosomal Storage Disorders

United States

Low Back Pain | Lumbar Disc Herniation | Radiculopathy | Lumbar Spinal Stenosis | Radicular Pain | 3D Printing Guide Plate | Lumbosacral Facet Joint Syndrome | Ischemic Pain of the Lower Extremity | Spinal Minimally Invasive and Interventional Surgeries

China

Autoimmune Diseases | Pregnancy Related | Sjogren's Syndrome | SLE | Neonatal Lupus | Fetal Heart Rate or Rhythm Abnormality Affecting Fetus

Italy

Chemotherapy | Recurrent Cervical Carcinoma | Adverse Drug Event | Cervical Neuroendocrine Carcinoma | Objective Response Rate | Advanced Cervical Carcinoma | Anti-pd-1 Antibody

China

Depression | Quality of Life | Pain | Generalized Anxiety Disorder | End Stage Renal Disease | Anxiety | Blood Pressure | Major Depressive Disorder | Compliance, Patient | Compliance, Medication | Mood | Heart Rate Fast | Energy Supply; Deficiency

United States

Acute Myeloid Leukemia | Recurrent Adult Acute Myeloid Leukemia | Secondary Acute Myeloid Leukemia | Untreated Adult Acute Myeloid Leukemia | Adult Acute Myeloid Leukemia in Remission | Refractory Acute Myeloid Leukemia | Blastic Plasmacytoid Dendritic Cell Neoplasm | Minimal Residual Disease | Acute... and other conditions

United States

Epilepsy | Neurodevelopmental Disorders | Autism Spectrum Disorder | Down Syndrome | Mitochondrial Encephalomyopathies | Pediatric Acute-Onset Neuropsychiatric Syndrome | Cerebral Folate Deficiency | Pediatric Autoimmune Neuropsychiatric Disorder Associated With Streptococcal Infection

United States

Adrenoleukodystrophy | Adrenomyeloneuropathy | Metachromatic Leukodystrophy | Adult-Onset Leukoencephalopathy With Axonal Spheroids and Pigmented Glia

France

Chronic Hepatitis B | Roadmap Concept in Chronic Hepatitis B Treatment | 24-week PCR Negativity of Telbivudine | PCR Negativity at 52 and 104 Week | HBeAg Seroconversion Rate at 52 and 104 Week

Taiwan

Change in Albumin to Creatinine Ratio and Glomerular Filtration Rate | Progression of Kidney Failure and or All-cause Mortality

France

Chronic Kidney Diseases | Secondary Hyperparathyroidism | Renal Osteodystrophy | Bone Turnover Rate Disorder | CKD-MBD

United States

Mantle Cell Lymphoma | Chronic Lymphocytic Leukemia | Adverse Event | Chronic Graft-versus-host-disease | Response Rate

Poland

Systemic Lupus Erythematosus (SLE) | Multiple Sclerosis (MS) | Rheumatoid Arthritis (RA) | Juvenile Dermatomyositis (JDM) | Juvenile Idiopathic Arthritis (JIA) | Pemphigus Vulgaris | Systemic Sclerosis (SSc) | Pediatric SLE | Pediatric-Onset Multiple Sclerosis (POMS)

United States

Anger | PTSD | TBI (Traumatic Brain Injury) | Sleep Disturbance | Headache | Posttraumatic Stress Disorder | Combat Stress Disorders | Eating Disorders | Nightmare | Post-Traumatic Headache | TBI | Military Activity | Posttraumatic Stress Disorder, Delayed Onset | Irritable Mood | Combat and Operational Stress Reaction | Military Family and other conditions

United States

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

The Primary Evaluation is the Change From Baseline at End of Study for CDSS Total Scores. | The Secondary Efficacy Evaluations Include: MADRS, PANSS Total Scores and Subscales and Responder Rate; CGI-S and CGI-I Scales. | Safety Evaluations Include: Laboratory Test Abnormalities; BARS... and other conditions

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

Alzheimer Disease | Traumatic Brain Injury | Autism Spectrum Disorder | Lewy Body Disease | Vascular Dementia | Autism | Dementia, Mixed | Alzheimer Dementia | Dementia, Multi-Infarct | Cadasil | Parkinson-Dementia Syndrome | Chronic Traumatic Encephalopathy | Autistic Behavior | Lewy Body Dementia With Behavioral... and other conditions

United States, United Arab Emirates

Efficacy Endpoint: no New Cerebrovascular Adverse Events Within 1 Year | Safety Endpoint: no New Moderate or Serious Adverse Events Within 1 Year | All-cause Mortality Events (Within 1 Year) | Incident Ischemic Stroke Events (Within 1 Year) | New Hemorrhagic Stroke Events (Within 1 Year) | New... and other conditions

Heart Failure | Epilepsy | Pain | Pneumonia | Sepsis | Inflammation | Hypertension | Chronic Kidney Diseases | Hemophilia | Schizophrenia | Central Nervous System Infections | Nosocomial Pneumonia | Insomnia | Anxiety | Bipolar Disorder | Neutropenia | Staphylococcal Infections | Bradycardia | Seizures | Urinary Tract Infections | Pulmonary... and other conditions

United States, Canada, United Kingdom, Israel, Singapore

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

Primary Hyperoxaluria Type 3 | Diabetes Mellitus | Hemophilia A | Hemophilia B | Hereditary Fructose Intolerance | Cystic Fibrosis | Factor VII Deficiency | Phenylketonurias | Sickle Cell Disease | Dravet Syndrome | Duchenne Muscular Dystrophy | Prader-Willi Syndrome | Fragile X Syndrome | Chronic Granulomatous Disease and other conditions

United States