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Found 270 clinical trials
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Retrospective study examining the differences in vocalizations of babies and toddlers with Rett compared to same age peers

Retrospective study examining the differences in vocalizations of babies and toddlers with Rett compared to same age peers: We are looking to recruit children and their parent(s) to participate in a study examining the early speech development of infants and toddlers later diagnosed with Rett syndrome, compared to girls their …

  • 0 views
  • 02 Oct, 2020
  • 1 location
featured
Eltrombopag for People With Fanconi Anemia

Fanconi anemia is a genetic disease. Some people with it have reduced blood cell counts. This means their bone marrow no longer works properly. These people may need blood transfusions for anemia

anemia
fanconi's syndrome
fanconi's anemia
anemia studies
  • 91 views
  • 22 Dec, 2020
  • 1 location
featured
Cardiovascular Disease Discovery Protocol

Background: Researchers are interested in studying individuals who have known or suspected metabolic or genetic diseases that put them at a high risk for heart

angiography
  • 327 views
  • 08 Apr, 2019
  • 1 location
featured
Inflammatory Bowel Disease - Minors 17 and younger

Inflammatory Bowel Disease - Minors 17 and younger

  • 44 views
  • 08 Nov, 2020
  • 1 location
featured
HAE - Patients diagnosed with Acute Hereditary Angioedema

HAE - Patients diagnosed with Acute Hereditary Angioedema

  • 598 views
  • 08 Nov, 2020
  • 1 location
featured
CASCADE FH Registry: Cascade Screening for Awareness and Detection of Familial Hypercholesterolemia

CASCADE FH Registry: Cascade Screening for Awareness and Detection of Familial Hypercholesterolemia

  • 434 views
  • 23 Nov, 2020
  • 1 location
Rady Children's Institute Genomic Biorepository

provide a mechanism for making a diagnosis of a genetic disease. That is, once genomic sequences have been derived from biological samples, they will be immediately analyzed. If a genetic disease is

  • 0 views
  • 03 Jun, 2019
  • 1 location
South-seq: Deoxyribonucleic Acid (DNA) Sequencing for Newborn Nurseries in the South

2,000 infants with signs suggestive of a genetic disorder being treated at a neonatal intensive care unit (NICU) in which African-American and rural populations are highly represented will be

  • 0 views
  • 19 Jan, 2021
  • 3 locations
Study of the Anti-FGF23 Antibody Burosumab in Adults With XLH

This is phase 3b open-label, international, multicenter study to continue to monitor the long-term safety and efficacy of burosumab in adult patients with XLH that participated in previous clinical trials with burosumab (UX023-CL303 / UX023-CL304).

  • 32 views
  • 01 Dec, 2019
  • 11 locations
Non Invasive Prenatal Test of Rare Genetic Diseases: Application to Rare Intellectual Disabilities

The aim of this study is to evaluate performances of a NIPT test based onto the study of the maternal blood to search known genetic mutations already detected in the family and potentially inherited by the fetus. This test will avoid an invasive prenatal diagnosis in those families with a …

  • 0 views
  • 08 Nov, 2020
  • 1 location