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A patient survey on the impact of alkaptonuria symptoms as perceived by the patients and their experiences of receiving diagnosis and care

BACKGROUND: Alkaptonuria (AKU) is an ultrarare and multifaceted disease characterized by the absence of functional homogentisate 1,2‐dioxygenase activity, the enzyme responsible for breakdown of homogentisic acid—a tyrosine‐degradation product. The presymptomatic phase of the disease makes diagnosis...

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Dettagli Bibliografici
Pubblicato in:JIMD Rep
Autori principali: Rudebeck, Mattias, Scott, Ciarán, Sireau, Nicolas, Ranganath, Lakshminarayan
Natura: Artigo
Lingua:Inglês
Pubblicazione: John Wiley & Sons, Inc. 2020
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Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC7203644/
https://ncbi.nlm.nih.gov/pubmed/32395411
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/jmd2.12101
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