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Homocystinuria patient and caregiver survey: experiences of diagnosis and patient satisfaction

BACKGROUND: The main genetic causes of homocystinuria are cystathionine beta-synthase (CBS) deficiency and the remethylation defects. Many patients present in childhood but milder forms may present later in life. Some countries have newborn screening programs for the homocystinurias but these do not...

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Библиографические подробности
Опубликовано в: :Orphanet J Rare Dis
Главные авторы: Morrison, T., Bösch, F., Landolt, M. A., Kožich, V., Huemer, M., Morris, A. A. M.
Формат: Artigo
Язык:Inglês
Опубликовано: BioMed Central 2021
Предметы:
Online-ссылка:https://ncbi.nlm.nih.gov/pmc/articles/PMC7945666/
https://ncbi.nlm.nih.gov/pubmed/33691747
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1186/s13023-021-01764-x
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