Clinical Trials on Birth, Preterm in New Zealand

Lynch Syndrome | Stage III Colon Cancer AJCC v8 | Colon Adenocarcinoma | DNA Repair Disorder

United States, Puerto Rico, Germany

Huntington Disease

United States, Austria, Canada, Czechia, France, Germany, Italy, Netherlands, Poland, Spain, United Kingdom

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

Autoimmune Diseases | Hodgkin Lymphoma | Hematologic Malignancies | Acute Lymphoblastic Leukemia (ALL) | Non-hodgkin Lymphoma | Severe Aplastic Anemia | Inherited Disorders of Metabolism | Inherited Abnormalities of Platelets | Histiocytic Disorders | Acute Myelogenous Leukemia (AML or ANLL) | Other Acute... and other conditions

United States

Seizures | Tuberous Sclerosis Complex

United States, Spain, Australia, Poland, United Kingdom, Netherlands

Hereditary Angioedema Types I and II

United States, Spain, Australia, Canada, Czechia, Hungary, Israel, Italy, Romania, United Kingdom

Pancreatic Adenocarcinoma | Solid Tumor | Non Small Cell Lung Cancer | Mesothelioma | Advanced Solid Tumor | Malignant Peripheral Nerve Sheath Tumors

United States

Small-cell Lung Cancer

Spain, Belgium, United States, China, Japan, Italy, France

Polycystic Kidney, Autosomal Dominant

United States, Canada, Australia, Sweden, Spain, Poland, Korea, Republic of, Switzerland, United Kingdom, Moldova, Republic of, Italy, Czech Republic, Hungary, Lithuania, Romania, Slovakia, Turkey

Hemophilia B

Belgium, Spain, Italy, Austria, United Kingdom, Netherlands, Sweden, Germany, Portugal, France

Brain and Central Nervous System Tumors | Neurofibromatosis Type 1

Canada, United States

Congenital Bleeding Disorder | Haemophilia B

Spain, United Kingdom, Australia, Taiwan, France, Malaysia, United States, Austria, Canada, Israel, Japan, Thailand, Algeria, Argentina

Gout | Chronic Kidney Disease (CKD)

United States, Hungary, Poland, Czechia

Polycystic Kidney, Autosomal Dominant | Autosomal Dominant Polycystic Kidney Disease | ADPKD

United States

Metachromatic Leukodystrophy (MLD)

United States, Canada, Belgium, Israel, Netherlands, United Kingdom, Germany, Spain, Japan, Brazil, Argentina, France, Greece, Italy

Neurofibromatosis 2 | Progressive Vestibular Schwannoma (VS)

United States

Juvenile Myelomonocytic Leukemia | Neurofibromatosis Type 1

United States

Chronic Kidney Diseases | Gout

United States

Tuberous Sclerosis | Subependymal Giant Cell Astrocytoma

United States, Belgium, Canada, Germany, Italy, Russian Federation, Netherlands, Poland, Australia, United Kingdom

Juvenile Myelomonocytic Leukemia | Secondary Acute Myeloid Leukemia | Childhood Acute Myeloid Leukemia in Remission | Accelerated Phase Chronic Myelogenous Leukemia | Childhood Acute Lymphoblastic Leukemia in Remission | Childhood Chronic Myelogenous Leukemia | Childhood Myelodysplastic Syndromes and other conditions

United States

Turner Syndrome

United States

Sphingomyelin Lipidosis

Italy, Czechia, Belgium, France, Portugal, Turkey, United States, Argentina, Brazil, Chile, Germany, Romania, Spain

Fabry Disease

United States, Italy, Spain, Turkey, Brazil, Canada, United Kingdom, France, Poland, Australia, Egypt, Argentina, Denmark

Malignant Solid Neoplasm | Neoplasms | Cancer | Neoplasm Metastasis | Solid Tumor | Metastatic Cancer | Metastasis | Advanced Solid Tumor | Metastatic Solid Tumor | Advanced Cancer | Tumor | Malignant Neoplasm | Malignant Tumor | Tumors | Cancer Metastatic | Tumor, Solid | Malignant Solid Tumor | TSC | TSC1 | TSC2 | Metastatic Neoplasm

United States, Korea, Republic of, Puerto Rico

Chronic Granulomatous Disease | Hemophagocytic Lymphohistiocytosis | Leukocyte Adhesion Deficiency | Chronic Active Epstein-Barr Virus Infection | HIGM-1 | IPEX

United States, Canada

Mucopolysaccharidosis II

United States, Spain, United Kingdom, Czechia, France, Canada, Italy, Belgium, Netherlands, Germany, Argentina, Sweden, Turkey, Brazil, Colombia

Muscular Dystrophies | Muscular Dystrophy, Facioscapulohumeral | FSHD | Facio-Scapulo-Humeral Dystrophy | FMD | Facioscapulohumeral Muscular Dystrophy 1 | FSHD2 | FSHD1 | FMD2 | Fascioscapulohumeral Muscular Dystrophy | Fascioscapulohumeral Muscular Dystrophy Type 1 | Fascioscapulohumeral Muscular Dystrophy... and other conditions

United States, United Kingdom, Canada

Sarcoma | Kidney Cancer | Neuroblastoma | Retinoblastoma | Adrenocortical Carcinoma | Gastrointestinal Carcinoid Tumor

United States

Fabry Disease

United States, United Kingdom, Canada, Hungary, Poland, Czech Republic

Chronic Kidney Disease | Autosomal Dominant Polycystic Kidney Disease

Australia, Belgium, Italy, Spain, Sweden, United States, Canada, Netherlands, Czechia, Poland, Germany, Russian Federation, Hungary, Israel, Puerto Rico, Argentina, Denmark, South Africa, United Kingdom, Norway, France, Romania

Congenital Bleeding Disorder | Haemophilia A

United States, Spain, Germany, Switzerland, Serbia, Taiwan, United Kingdom, Croatia, Italy, Malaysia, Brazil, Japan, Denmark, Hungary, Israel, Turkey, Russian Federation, Former Serbia and Montenegro

Acute Hepatic Porphyria | Acute Intermittent Porphyria | Porphyria, Acute Intermittent | Acute Porphyria | Hereditary Coproporphyria (HCP) | Variegate Porphyria (VP) | ALA Dehydratase Deficient Porphyria (ADP)

United States, Spain, United Kingdom, Korea, Republic of, Australia, Bulgaria, Canada, Denmark, Finland, France, Germany, Italy, Japan, Mexico, Netherlands, Poland, Sweden, Taiwan

Polycystic Kidney Disease, Autosomal Dominant

United States, Belgium, United Kingdom, Russian Federation, France, Australia, Netherlands, Italy, Japan, Denmark, Romania, Poland, Canada, Argentina, Germany

Congenital Bleeding Disorder | Haemophilia A | Haemophilia B

United States, Spain, Taiwan, Turkey, Poland, Croatia, Italy, Malaysia, Brazil, United Kingdom, Hungary, Israel, Thailand, Japan, South Africa, Canada, France, Argentina

Tuberous Sclerosis Complex

United States, Spain, United Kingdom, China, Italy, France, Israel, Germany, Canada, Australia

Congenital Bleeding Disorder | Haemophilia A With Inhibitors | Haemophilia B With Inhibitors

Taiwan, United Kingdom, Thailand, Serbia, Croatia, Italy, Poland, Romania, Hungary, Malaysia, United States, Austria, Brazil, Greece, Japan, Puerto Rico, Russian Federation, South Africa, Turkey

Melanoma | P53 Mutation | Uveal Melanoma | Cutaneous Melanoma | Mucosal Melanoma | Malignant Peripheral Nerve Sheath Tumors | MPNST | Unresectable or Metastatic Melanoma or Advanced Solid Tumors | MDM2 Gene Mutation

United States, Australia

Soft Tissue Sarcoma | Lymphoma | Acute Myeloid Leukemia | Leukemia | Osteosarcoma | Acute Lymphoblastic Leukemia | Ewing Sarcoma | Rhabdoid Tumor | Neuroblastoma | Retinoblastoma | Rhabdomyosarcoma | Malignant Neoplasm | Germ Cell Tumor | Central Nervous System Neoplasm

United States, Canada

Lynch Syndrome | Malignant Glioma | Recurrent Childhood Ependymoma | Recurrent Medulloblastoma | Refractory Ependymoma | Refractory Medulloblastoma | Recurrent Diffuse Intrinsic Pontine Glioma | Refractory Diffuse Intrinsic Pontine Glioma | Recurrent Brain Neoplasm | Refractory Brain Neoplasm | Constitutional...

United States, Canada

Amyloid Neuropathies | Amyloid Neuropathies, Familial | TTR-mediated Amyloidosis | Amyloidosis, Hereditary | Amyloidosis, Hereditary, Transthyretin-Related | Familial Amyloid Polyneuropathies

United States, Canada, Argentina, Australia, Bulgaria, Cyprus, Italy, Japan, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Germany, France, Brazil, Turkey, Korea, Republic of, Taiwan, United Kingdom

Recurrent Neuroblastoma | Recurrent Rhabdomyosarcoma | Wilms Tumor | Recurrent Malignant Peripheral Nerve Sheath Tumor | Recurrent Synovial Sarcoma | Pleuropulmonary Blastoma

United States

Low Grade Glioma | Recurrent Visual Pathway Glioma | Refractory Visual Pathway Glioma | Recurrent Childhood Pilocytic Astrocytoma | Recurrent Neurofibromatosis Type 1 | Refractory Neurofibromatosis Type 1

United States

Hypercortisolism

United States, Austria, Bulgaria, Germany, Israel, Italy, Poland, Romania, Spain

Sarcoma

United States

Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

United States, Poland, Argentina, Australia, Belgium, France, Germany, Japan, Netherlands, Portugal, Spain, Sweden, United Kingdom, Canada, Czechia, Denmark, Hungary, Ireland, Norway, Korea, Republic of, Austria, Croatia, Israel, Latvia, Lithuan... and more

Basal Cell Carcinoma

United States, Spain, France, Germany, Netherlands, Russian Federation, Italy, Mexico, Austria, Canada

Anatomic Stage IV Breast Cancer AJCC v8 | Metastatic HER2-Negative Breast Carcinoma | Metastatic Hormone Receptor-Positive Breast Carcinoma

United States, Puerto Rico

Mitochondrial Diseases | Drug Resistant Epilepsy | Leigh Disease | Leigh Syndrome | Mitochondrial Encephalopathy (MELAS) | Pontocerebellar Hypoplasia Type 6 (PCH6) | Alpers Disease | Alpers Syndrome

United States, Spain, United Kingdom, Canada, France, Italy, Poland, Sweden, Japan

Hereditary Angioedema (HAE)

France, Austria, Australia, Spain, Czechia, Italy, United Kingdom, Germany, Israel, Ireland, Brazil, Denmark, Greece, Sweden

Down Syndrome | Recurrent Acute Myeloid Leukemia | Recurrent Myelodysplastic Syndrome | Refractory Acute Myeloid Leukemia | Refractory Myelodysplastic Syndrome | Recurrent Mixed Phenotype Acute Leukemia | Refractory Mixed Phenotype Acute Leukemia | Acute Myeloid Leukemia Post Cytotoxic Therapy | Myelodysplastic...

United States