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Blood|February 28, 2002
Retrovirally mediated correction of bone marrow-derived mesenchymal stem cells from patients with mucopolysaccharidosis type IMelissa A Baxter, Robert F Wynn, Jonathan A Deakin, et al.
Orphanet Journal of Rare Diseases|April 19, 2015
Sleep disordered breathing in mucopolysaccharidosis I: a multivariate analysis of patient, therapeutic and metabolic correlators modifying long term clinical outcomeAbhijit Ricky Pal, Eveline J Langereis, Muhammad A Saif, et al.
JIMD Reports|January 31, 2016
Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell TransplantationA S Kunin-Batson, E G Shapiro, K D Rudser, et al.
International Journal of Neonatal Screening|March 26, 2025
Does Early Diagnosis and Treatment Alter the Clinical Course of Wolman Disease? Divergent Trajectories in Two Siblings and a Consideration for Newborn ScreeningMaria Jose de Castro Lopez, Fiona J White, Victoria Holmes, et al.
Plos One|December 15, 2010
Genistein improves neuropathology and corrects behaviour in a mouse model of neurodegenerative metabolic diseaseMarcelina Malinowska, Fiona L Wilkinson, Kia J Langford-Smith, et al.
Cytotherapy|May 14, 2018
Multicenter phase 1/2 application of adenovirus-specific T cells in high-risk pediatric patients after allogeneic stem cell transplantationWinnie Ip, Juliana M F Silva, Hubert Gaspar, et al.
Therapeutic Drug Monitoring|September 14, 2012
Predictive performance of a busulfan pharmacokinetic model in children and young adultsImke H Bartelink, Charlotte van Kesteren, Jaap J Boelens, et al.
JAMA|April 20, 2001
Effectiveness of St John's wort in major depression: a randomized controlled trialR C Shelton, M B Keller, A Gelenberg, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|May 2, 2012
Hematopoietic stem cell and gene therapy corrects primary neuropathology and behavior in mucopolysaccharidosis IIIA miceAlexander Langford-Smith, Fiona L Wilkinson, Kia J Langford-Smith, et al.
Cell Reports|October 26, 2017
Absence of Carbohydrate Response Element Binding Protein in Adipocytes Causes Systemic Insulin Resistance and Impairs Glucose TransportArchana Vijayakumar, Pratik Aryal, Jennifer Wen, et al.
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