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children with rare diseases

Showing 1 - 25 of >10,000
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Rare Diseases Trial in Tübingen (Data collection)

Recruiting
  • Rare Diseases
  • Data collection
  • Tübingen, Germany
    University Hospital Tübingen
Mar 22, 2023

Rare Diseases Trial in Washington (FAmily CEntered (FACE) pACP Intervention)

Recruiting
  • Rare Diseases
  • FAmily CEntered (FACE) pACP Intervention
  • Washington, District of Columbia
    Children's National Hospital
Nov 14, 2022

Epilepsy, Rare Diseases Trial (Cannabidiol oral solution)

Not yet recruiting
  • Epilepsy
  • Rare Diseases
  • Cannabidiol oral solution
  • (no location specified)
Mar 27, 2023

Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

Not yet recruiting
  • Sickle Cell Disease
  • Interview
  • Memphis, Tennessee
    St. Jude Children's Research Hospital
Mar 30, 2023

es to Capture the Real Life of Children With Rare Eye Diseases

Not yet recruiting
  • Eye Diseases
  • +3 more
  • Mixed Methods Research (MMR) using quantitative (QUAN) and qualitative (QUAL) tools
  • Strasbourg, France
    Centre de Référence pour les Affections Rares en Génétique Ophta
Oct 3, 2023

Rare Diseases Trial in Hamburg

Completed
  • Rare Diseases
    • Hamburg, Germany
      University Medical Center Hamburg-Eppendorf
    Nov 27, 2021

    SLC13A5 Deficiency Natural History Study - United States Only

    Recruiting
    • Citrate Transporter Deficiency
    • +9 more
      • Palo Alto, California
      • +2 more
      Nov 17, 2023

      Rare Diseases Trial in Switzerland

      Recruiting
      • Rare Diseases
        • Basel, Switzerland
        • +5 more
        Dec 16, 2021

        Genetic Disease, Genetic Syndrome, Genetic Predisposition to Disease Trial in Boston

        Recruiting
        • Genetic Disease
        • +4 more
          • Boston, Massachusetts
            Boston Children's Hospital
          Oct 17, 2022

          Rare Genetic Disorders of the Breathing Airways

          Completed
          • Kartagener Syndrome
          • +3 more
            • Palo Alto, California
            • +8 more
            Aug 9, 2022

            Hereditary Spastic Paraplegia, Neurodegenerative Diseases, Pediatric Disorder Trial in Boston

            Recruiting
            • Hereditary Spastic Paraplegia
            • +5 more
              • Boston, Massachusetts
                Boston Children's Hospital
              Aug 5, 2022

              Rare Diseases Trial in Germany (CARE-FAM, WEP-CARE)

              Active, not recruiting
              • Rare Diseases
              • CARE-FAM
              • WEP-CARE
              • Augsburg, Germany
              • +16 more
              Sep 14, 2022

              Bardet-Biedl Syndrome, POMC Deficiency Obesity, PCSK1 Deficiency Obesity Trial in Worldwide (Setmelanotide)

              Active, not recruiting
              • Bardet-Biedl Syndrome
              • +3 more
              • Aurora, Colorado
              • +5 more
              Jul 21, 2022

              Diagnosis and Treatment Management of Rare Neurological Disease

              Withdrawn
              • Xlsma
              • +3 more
                • Shanghai, China
                  Children's Hospital of Fudan University
                Aug 4, 2021

                Quality of Life and Experience of Young Utero-vaginal Aplasia

                Recruiting
                • Mrkh Syndrome
                • Questionnaires
                • Paris, France
                  Necker Children's Hospital
                Dec 30, 2022

                Gene Discovery Core, The Manton Center

                Recruiting
                • Undiagnosed Conditions
                • +2 more
                  • Boston, Massachusetts
                    Boston Children's Hospital
                  Aug 9, 2021

                  Rare and Atypical Diabetes Network

                  Recruiting
                  • Diabetes Mellitus
                  • +6 more
                    • Aurora, Colorado
                    • +13 more
                    Sep 14, 2022

                    Muscular Dystrophies, Muscular Dystrophy, Duchenne, Muscular Disorders, Atrophic Trial in Little Rock, Sacramento, Atlanta

                    Recruiting
                    • Muscular Dystrophies
                    • +7 more
                    • CAP-1002
                    • Placebo
                    • Little Rock, Arkansas
                    • +9 more
                    Jan 9, 2023

                    A Non-Interventional Post-Authorization Study of Carbaglu for

                    Enrolling by invitation
                    • Hyperammonemia
                    • +2 more
                    • carglumic acid
                    • Indianapolis, Indiana
                      Riley Children's Hospital
                    Apr 24, 2023

                    Study of Retinal Dystrophies Associated With Rare

                    Active, not recruiting
                    • Inherited Retinal Degeneration
                    • Retinitis Pigmentosa
                      • Phoenix, Arizona
                      • +23 more
                      Jan 24, 2023

                      Quality of Life of Rare Diseases: a Qualitative Approach

                      Completed
                      • Rare Diseases
                      • Semi-structured interview
                      • Paris, France
                        Hôpital Necker-Enfants Malades
                      Feb 2, 2023

                      15q Duplication Syndrome, CDKL5 Deficiency Disease Trial in United States (Soticlestat)

                      Completed
                      Has Results
                      • 15q Duplication Syndrome
                      • CDKL5 Deficiency Disease
                      • Los Angeles, California
                      • +7 more
                      May 24, 2022

                      Clinical & Anthropometric Characteristics of Achondroplasia.

                      Active, not recruiting
                      • Achondroplasia
                        • Torrance, California
                        • +32 more
                        Nov 16, 2022

                        Rare Diseases Clinical Research Network: Neurophysiological

                        Completed
                        • Rett Syndrome, Preserved Speech Variant
                        • +2 more
                        • Auditory and Visual Event-related Potentials and EEG
                        • Denver, Colorado
                        • +4 more
                        Aug 3, 2021

                        Atypical Hemolytic Uremic Syndrome, Membranoproliferative Glomerulonephritis, Autosomal Dominant Polycystic Kidney Trial in

                        Not yet recruiting
                        • Atypical Hemolytic Uremic Syndrome
                        • +3 more
                        • Skin biopsy
                        • Ranica, BG, Italy
                          Centro di Ricerche Cliniche per le Malattie Rare "Aldo e Cele Da
                        Aug 9, 2023