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Focal lesions following intracerebral gene therapy for mucopolysaccharidosis IIIA.
Ann Clin Transl Neurol. 2023 Jun;10(6):904-917. doi: 10.1002/acn3.51772. Epub 2023 May 11.
Ann Clin Transl Neurol. 2023.
PMID: 37165777
Free PMC article.
An observational, prospective, multicenter, natural history study of patients with mucopolysaccharidosis type IIIA.
Wijburg FA, Aiach K, Chakrapani A, Eisengart JB, Giugliani R, Héron B, Muschol N, O'Neill C, Olivier S, Parker S.
Wijburg FA, et al. Among authors: aiach k.
Mol Genet Metab. 2022 Feb;135(2):133-142. doi: 10.1016/j.ymgme.2021.12.002. Epub 2021 Dec 10.
Mol Genet Metab. 2022.
PMID: 34991944
Free article.
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AAVrh10 vector corrects pathology in animal models of GM1 gangliosidosis and achieves widespread distribution in the CNS of nonhuman primates.
Hocquemiller M, Giersch L, Mei X, Gross AL, Randle AN, Gray-Edwards HL, Hudson JA, Todeasa S, Stoica L, Martin DR, Sena-Esteves M, Aiach K, Laufer R.
Hocquemiller M, et al. Among authors: aiach k.
Mol Ther Methods Clin Dev. 2022 Oct 7;27:281-292. doi: 10.1016/j.omtm.2022.10.004. eCollection 2022 Dec 8.
Mol Ther Methods Clin Dev. 2022.
PMID: 36320411
Free PMC article.
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Understanding disease symptoms and impacts and producing qualitatively-derived severity stages for MPS IIIA: a mixed methods approach.
Lanar S, Parker S, O'Neill C, Marrel A, Arnould B, Héron B, Muschol N, Wijburg FA, Chakrapani A, Olivier S, Aiach K.
Lanar S, et al. Among authors: aiach k.
Orphanet J Rare Dis. 2022 Feb 22;17(1):75. doi: 10.1186/s13023-022-02208-w.
Orphanet J Rare Dis. 2022.
PMID: 35193633
Free PMC article.
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[Place of the expert patient in the R&D process in the field of rare diseases].
Aïach K.
Aïach K.
Med Sci (Paris). 2018 May;34 Hors série n°1:28-29. doi: 10.1051/medsci/201834s114. Epub 2018 Jun 18.
Med Sci (Paris). 2018.
PMID: 29911552
Free article.
French.
No abstract available.
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