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Hip Morphology in Mucolipidosis Type II.
Ammer LS, Oussoren E, Muschol NM, Pohl S, Rubio-Gozalbo ME, Santer R, Stuecker R, Vettorazzi E, Breyer SR. Ammer LS, et al. Among authors: oussoren e. J Clin Med. 2020 Mar 8;9(3):728. doi: 10.3390/jcm9030728. J Clin Med. 2020. PMID: 32182687 Free PMC article.
Intrafamilial oocyte donation in classic galactosemia: ethical and societal aspects.
Haskovic M, Poot WJ, van Golde RJT, Benneheij SH, Oussoren E, de Wert GMWR, Krumeich A, Rubio-Gozalbo ME. Haskovic M, et al. Among authors: oussoren e. J Inherit Metab Dis. 2018 Sep;41(5):791-797. doi: 10.1007/s10545-018-0179-y. Epub 2018 Apr 18. J Inherit Metab Dis. 2018. PMID: 29671189 Free PMC article.
Tagged IDS causes efficient and engraftment-independent prevention of brain pathology during lentiviral gene therapy for Mucopolysaccharidosis type II.
Catalano F, Vlaar EC, Katsavelis D, Dammou Z, Huizer TF, van den Bosch JC, Hoogeveen-Westerveld M, van den Hout HJMP, Oussoren E, Ruijter GJG, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP. Catalano F, et al. Among authors: oussoren e. Mol Ther Methods Clin Dev. 2023 Nov 2;31:101149. doi: 10.1016/j.omtm.2023.101149. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38033460 Free PMC article.
Isolated neurological presentations of mevalonate kinase deficiency.
Hoytema van Konijnenburg EMM, Oussoren E, Frenkel J, van Hasselt PM. Hoytema van Konijnenburg EMM, et al. Among authors: oussoren e. JIMD Rep. 2022 Nov 18;64(1):53-56. doi: 10.1002/jmd2.12348. eCollection 2023 Jan. JIMD Rep. 2022. PMID: 36636591 Free PMC article.
Lentiviral Gene Therapy for Mucopolysaccharidosis II with Tagged Iduronate 2-Sulfatase Prevents Life-Threatening Pathology in Peripheral Tissues But Fails to Correct Cartilage.
Catalano F, Vlaar EC, Dammou Z, Katsavelis D, Huizer TF, Zundo G, Hoogeveen-Westerveld M, Oussoren E, van den Hout HJMP, Schaaf G, Pike-Overzet K, Staal FJT, van der Ploeg AT, Pijnappel WWMP. Catalano F, et al. Among authors: oussoren e. Hum Gene Ther. 2024 Apr;35(7-8):256-268. doi: 10.1089/hum.2023.177. Epub 2024 Feb 2. Hum Gene Ther. 2024. PMID: 38085235 Free PMC article.
Disease characteristics, effectiveness, and safety of vestronidase alfa for the treatment of patients with mucopolysaccharidosis VII in a novel, longitudinal, multicenter disease monitoring program.
Giugliani R, Gonzalez-Meneses A, Scarpa M, Burton B, Wang R, Martins E, Oussoren E, Hennermann JB, Chabrol B, Grant CL, Sun A, Durand C, Hetzer J, Malkus B, Marsden D, Merritt Ii JL. Giugliani R, et al. Among authors: oussoren e. Orphanet J Rare Dis. 2024 May 7;19(1):189. doi: 10.1186/s13023-024-03176-z. Orphanet J Rare Dis. 2024. PMID: 38715031 Free PMC article.
Modeling cartilage pathology in mucopolysaccharidosis VI using iPSCs reveals early dysregulation of chondrogenic and metabolic gene expression.
Broeders M, van Rooij J, Oussoren E, van Gestel T, Smith C, Kimber S, Verdijk R, Wagenmakers M, van den Hout J, van der Ploeg A, Narcisi R, Pijnappel W. Broeders M, et al. Among authors: oussoren e. Front Bioeng Biotechnol. 2022 Dec 6;10:949063. doi: 10.3389/fbioe.2022.949063. eCollection 2022. Front Bioeng Biotechnol. 2022. PMID: 36561048 Free PMC article.
44 results