Dysferlinopathy

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Information
Disease name
Dysferlinopathy
Disease ID
Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
Genes Mutation Description Source Links
NCT ID Status Phase Summary Start date Completion date
NCT01676077 Active, not recruiting Clinical Outcome Study for Dysferlinopathy September 2012 March 2024
NCT00527228 Completed Phase 2/Phase 3 Deflazacort in Dysferlinopathies September 2003 September 2008
NCT02710500 Completed Phase 1 rAAVrh74.MHCK7.DYSF.DV for Treatment of Dysferlinopathies March 2016 July 2019
NCT04824040 Enrolling by invitation Clinical, Immunological, Morphological and Genetic Characteristics of Patients With Dysferlinopathy in the RF January 15, 2020 July 2024
NCT01863004 Terminated Phase 1 Proteasomal Inhibition for Patients With Mis-sense Mutated Dysferlin December 2012 September 15, 2017