Clinical Trials on Consent in United States

Cryopyrin-associated Periodic Syndromes (CAPS) | Familial Cold Autoinflam Syn (FCAS) | Muckle-wells Syn (MWS) | Neonatal Onset Multisystem Inflam Disease (NOMID)

Switzerland, United States, Germany, Norway, Austria

Mucopolysaccharidosis I | Mucopolysaccharidosis II | Mucopolysaccharidosis VI | Wolman Disease | Pompe Disease Infantile-Onset | Mucopolysaccharidosis IV A | Mucopolysaccharidosis VII | Neuronopathic Gaucher Disease

United States

Late-onset Pompe Disease

United States, United Kingdom, Germany, France, Italy, Portugal, Belgium, Netherlands

Lysosomal Storage Diseases | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

United States, Italy, United Kingdom, Netherlands, France, Germany

Cardiovascular Diseases | Obstructive Sleep Apnea | Arrhythmia | Atrial Fibrillation New Onset

United Kingdom

Premature Birth | Necrotizing Enterocolitis | Late-onset Invasive Infection

United Kingdom, Ireland

Glycogenesis 2 Acid Maltase Deficiency | Pompe Disease (Late-Onset) | Glycogen Storage Disease Type II (GSD II)

United States, Netherlands, United Kingdom, Germany

Pompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2

United States, France, Netherlands

Late-onset Pompe Disease

United States, Canada, Australia, Belgium

Pompe Disease (Late-onset)

United States, Taiwan, Japan, Australia, Canada

Lysosomal Storage Diseases | Glycogen Storage Disease Type II | Glycogen Storage Disease Type 2 | Pompe Disease (Late-onset) | Pompe Disease | LOPD | Acid Maltase Deficiency

United States, Canada, Netherlands, France, Denmark, Germany, Italy, United Kingdom

Pompe Disease (Late-onset) | Glycogen Storage Disease Type II (GSD-II) | Glycogenesis 2 Acid Maltase Deficiency

Croatia, United States, Italy, Belgium, Czechia

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

Glycogenosis 2 | Glycogen Storage Disease Type II (GSD II) | Acid Maltase Deficiency | Pompe Disease (Infantile-Onset)

United States, Taiwan, Germany

Familial Cold Autoinflammatory Syndrome | Cryopyrin-Associated Periodic Syndromes | Neonatal Onset Multisystem Inflammatory Disease | Muckle Wells Syndrome

United States, Germany, Italy, Spain, United Kingdom, Turkey, France, Belgium, India

Pompe Disease (Late-onset)

United States, Germany, Taiwan, Belgium, Australia, Slovenia, Spain, Netherlands, Hungary, Japan, Austria, United Kingdom, France, Argentina, Canada, Sweden, Denmark, New Zealand, Greece, Korea, Republic of, Bosnia and Herzegovina, Ita... and more

Pompe Disease (Late-onset)

United States, Australia, Korea, Republic of, Japan, Taiwan, Belgium, Slovenia, New Zealand, United Kingdom, France, Argentina, Canada, Spain, Bosnia and Herzegovina, Germany, Sweden, Hungary, Austria, Bulgaria, Denmark, Greece, I... and more

Glycogen Storage Disease Type II (GSD-II) | Pompe Disease (Late-Onset) | Glycogenesis Type II | Acid Maltase Deficiency (AMD)

United States, France, Canada, Netherlands, Australia

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

Familial Cold Autoinflammatory Syndrome | Muckle-Wells Syndrome | Neonatal Onset Multisystem Inflammatory Disease | Cryopyrin-associated Periodic Syndromes

Germany, France, Belgium, Canada, Spain, Switzerland, United Kingdom

Familial Cold Autoinflammatory Syndrome | Muckle-Wells Syndrome | Neonatal Onset Multisystem Inflammatory Disease | Cryopyrin-associated Periodic Syndromes

Germany, France, Belgium, Canada, Spain, Switzerland, United Kingdom

Glycogen Storage Disease Type II (GSD-II) | Acid Maltase Deficiency Disease | Glycogenosis 2 | Pompe Disease (Late-Onset)

United States

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

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

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