Wolfram syndrome: new pathophysiological insights and therapeutic strategies.
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ABSTRACT:
SUBMITTER: Mishra R
PROVIDER: S-EPMC10032446 | biostudies-literature | 2021 Jan-Dec
REPOSITORIES: biostudies-literature
Publications
Wolfram syndrome: new pathophysiological insights and therapeutic strategies.
Mishra Ratnakar R Chen Benson S BS Richa Prachi P Yu-Wai-Man Patrick P
Therapeutic advances in rare disease 20210101
Wolfram Syndrome (WS) is an ultra-rare, progressive neurodegenerative disease characterized by early-onset diabetes mellitus and irreversible loss of vision, secondary to optic nerve degeneration. Visual loss in WS is an important cause of registrable blindness in children and young adults and the pathological hallmark is the preferential loss of retinal ganglion cells within the inner retina. In addition to optic atrophy, affected individuals frequently develop variable combinations of neurolog ...[more]
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