Qualitative interviews with patients with mucopolysaccharidosis type I (MPS I) and caregivers to evaluate the Mucopolysaccharidosis Health Assessment Questionnaire (MPS-HAQ)
Pabla, Anureet K., Litcher-Kelly, Leighann, Pollissard, Laurence, Yaworsky, Andrew, Ollis, Sarah, Kelly, Masami, Padilla, Brad, Hamed, Alaa
Published in Molecular genetics and metabolism (01.02.2019)
Published in Molecular genetics and metabolism (01.02.2019)
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Journal Article
Functional performance in late-onset GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases), longitudinal data over 3 consecutive years
Puga, Ana C., Hamed, Alaa, Kissell, Julie, Minini, Pascal, Pabla, Anureet K., Kahn, Susan, Eichler, Florian, Stephen, Christopher D., Tifft, Cynthia J., Toro, Camilo, Johnston, Jean, Lau, Heather A., Haxton, Elizabeth, Gray-Edwards, Heather, Fischer, Tanya
Published in Molecular genetics and metabolism (01.02.2018)
Published in Molecular genetics and metabolism (01.02.2018)
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Journal Article