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Cockayne Syndrome
Clinical trial pipeline · Data from ClinicalTrials.gov
See which Cockayne Syndrome trials you may qualify forClinical trial pipeline · Data from ClinicalTrials.gov
See which Cockayne Syndrome trials you may qualify forThis prospective observational study aims to investigate the impact of transcatheter aortic valve implantation (TAVI) and transcatheter mitral valve edge-to-edg…
The Myelin Disorders Biorepository Project (MDBP) seeks to collect and analyze clinical data and biological samples from leukodystrophy patients worldwide to su…
This will be a single-center, single-arm, non-interventional natural history study to evaluate the longitudinal clinical course, functional outcome measures, an…
CoRDS, or the Coordination of Rare Diseases at Sanford, is based at Sanford Research in Sioux Falls, South Dakota. It provides researchers with a centralized, i…
Four rare genetic diseases, xeroderma pigmentosum (XP), Cockayne syndrome (CS), the XP/CS complex and trichothiodystrophy (TTD) have defective DNA excision repa…
The palliative care needs of family caregivers of children with rare diseases and their children are largely unmet, including the need for support to prepare fo…
Leukodystrophies, and other heritable disorders of the white matter of the brain, were previously resistant to genetic characterization, largely due to the extr…