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Molecular Genetics and Metabolism|August 8, 2012
An exploratory study of brain function and structure in mucopolysaccharidosis type I: long term observations following hematopoietic cell transplantation (HCT)Elsa Shapiro, O Evren Guler, Kyle Rudser, et al.Journal of Patient-Reported Outcomes|April 25, 2022
Caregivers' assessment of meaningful and relevant clinical outcome assessments for Sanfilippo syndromeKatherine Ackerman Porter, Cara O'Neill, Elise Drake, et al.Advanced Science (Weinheim, Baden-Wurttemberg, Germany)|November 11, 2020
A Pumpless Microfluidic Neonatal Lung Assist Device for Support of Preterm Neonates in Respiratory DistressMohammadhossein Dabaghi, Niels Rochow, Neda Saraei, et al.Orphanet Journal of Rare Diseases|June 28, 2017
Recommendations on clinical trial design for treatment of Mucopolysaccharidosis Type IIIArunabha Ghosh, Elsa Shapiro, Stewart Rust, et al.Molecular Genetics and Metabolism Reports|November 24, 2021
A non-interventional observational study to identify and validate clinical outcome assessments for adults with phenylketonuria for use in clinical trialsBarbara K Burton, Anne Skalicky, Christoph Baerwald, et al.Molecular Genetics and Metabolism|December 27, 2014
Cognitive, medical, and neuroimaging characteristics of attenuated mucopolysaccharidosis type IIBrianna Yund, Kyle Rudser, Alia Ahmed, et al.Molecular Genetics and Metabolism|June 23, 2015
Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatmentElsa G Shapiro, Igor Nestrasil, Kyle Rudser, et al.The Lancet. Psychiatry|December 20, 2020
International consensus on a standard set of outcome measures for child and youth anxiety, depression, obsessive-compulsive disorder, and post-traumatic stress disorderKarolin R Krause, Sophie Chung, Abiodun O Adewuya, et al.Pageof 5