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P5CS deficiency

MedGen UID:
977394
Concept ID:
CN294786
Disease or Syndrome
Synonym: delta1-pyrroline-5-carboxylate synthetase deficiency
 
Monarch Initiative: MONDO:0100126

Definition

An inborn error of proline/orinthine metabolism that covers a wide spectrum of phenotypes and is caused by pathogenic variants in the aldehyde dehydrogenase 18 family member A1 (ALDH18A1) gene. These variants lead to a variety of neurocutaneous and motor syndromes characterized by cutis laxa, connective tissue weakness, facial dysmorphism, growth restriction, developmental delay, cataracts, hypotonia, hypertonia, and amyotrophy. [from MONDO]

Recent clinical studies

Diagnosis

Koh K, Takaki R, Ishiura H, Tsuji S, Takiyama Y
BMC Neurol 2021 Feb 11;21(1):64. doi: 10.1186/s12883-021-02087-x. PMID: 33573605Free PMC Article
Martinelli D, Häberle J, Rubio V, Giunta C, Hausser I, Carrozzo R, Gougeard N, Marco-Marín C, Goffredo BM, Meschini MC, Bevivino E, Boenzi S, Colafati GS, Brancati F, Baumgartner MR, Dionisi-Vici C
J Inherit Metab Dis 2012 Sep;35(5):761-76. Epub 2011 Dec 15 doi: 10.1007/s10545-011-9411-8. PMID: 22170564

Therapy

Martinelli D, Häberle J, Rubio V, Giunta C, Hausser I, Carrozzo R, Gougeard N, Marco-Marín C, Goffredo BM, Meschini MC, Bevivino E, Boenzi S, Colafati GS, Brancati F, Baumgartner MR, Dionisi-Vici C
J Inherit Metab Dis 2012 Sep;35(5):761-76. Epub 2011 Dec 15 doi: 10.1007/s10545-011-9411-8. PMID: 22170564

Clinical prediction guides

Martinelli D, Häberle J, Rubio V, Giunta C, Hausser I, Carrozzo R, Gougeard N, Marco-Marín C, Goffredo BM, Meschini MC, Bevivino E, Boenzi S, Colafati GS, Brancati F, Baumgartner MR, Dionisi-Vici C
J Inherit Metab Dis 2012 Sep;35(5):761-76. Epub 2011 Dec 15 doi: 10.1007/s10545-011-9411-8. PMID: 22170564

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