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Novel CHM mutations in Polish patients with choroideremia – an orphan disease with close perspective of treatment

BACKGROUND: Choroideremia (CHM) is a rare X-linked recessive retinal dystrophy characterized by progressive chorioretinal degeneration in the males affected. The symptoms include night blindness in childhood, progressive peripheral vision loss and total blindness in the late stages. The disease is c...

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Detalhes bibliográficos
Publicado no:Orphanet J Rare Dis
Main Authors: Skorczyk-Werner, Anna, Wawrocka, Anna, Kochalska, Natalia, Krawczynski, Maciej Robert
Formato: Artigo
Idioma:Inglês
Publicado em: BioMed Central 2018
Assuntos:
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6291982/
https://ncbi.nlm.nih.gov/pubmed/30541579
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-018-0965-5
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