Clinical Trials on Cerebellar Ataxia - Areflexia - Pes Cavus - Optic Atrophy - Sensorineural Hearing Loss

Total 5 results

  • Sanford Health
    National Ataxia Foundation; Beyond Batten Disease Foundation; Pitt Hopkins Research... and other collaborators
    Recruiting
    Rare Disease Patient Registry & Natural History Study - Coordination of Rare Diseases at Sanford (CoRDS)
    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
  • Hadassah Medical Organization
    Unknown
    Treatment of Wolfram Syndrome Type 2 With the Chelator Deferiprone and Incretin Based Therapy
    Diabetes Mellitus | Iron Metabolism Disorders | Optic Atrophy | Sensorineural Hearing Loss | Platelet Dysfunction | Gastroduodenal Ulcer
    Israel
  • Washington University School of Medicine
    National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National... and other collaborators
    Recruiting
    Wolfram Syndrome and WFS1-related Disorders International Registry and Clinical Study
    Diabetes Mellitus | Deafness | Ataxia | Diabetes Insipidus | Wolfram Syndrome | Optic Nerve Atrophy
    United States
  • Washington University School of Medicine
    National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK); National...
    Completed
    A Clinical Trial of Dantrolene Sodium in Pediatric and Adult Patients With Wolfram Syndrome
    Diabetes Mellitus | Ataxia | Wolfram Syndrome | Optic Nerve Atrophy
    United States
  • RTI International
    Eunice Kennedy Shriver National Institute of Child Health and Human Development... and other collaborators
    Enrolling by invitation
    Early Check: Expanded Screening in Newborns
    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

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