104-week efficacy and safety of cipaglucosidase alfa plus miglustat in adults with late-onset Pompe disease: a phase III open-label extension study (ATB200-07)
2024 · Journal of Neurology · 20 citations
https://doi.org/10.1007/s00415-024-12236-0
H
Heather Lau
Yale University(US)
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Heather Lau is an academic researcher from Yale University. The author has contributed to research in topics: Lysosomal Storage Disorders Research & Glycogen Storage Diseases and Myoclonus & Trypanosoma species research and implications. The author has an h-index of 23, co-authored 94 publications.
ORCID: 0000-0003-1690-9457Papers by this author
worksCommunity consensus for Heparan sulfate as a biomarker to support accelerated approval in Neuronopathic Mucopolysaccharidoses
2024 · Molecular Genetics and Metabolism · 16 citations
https://doi.org/10.1016/j.ymgme.2024.108535
Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey
2023 · Molecular Genetics and Metabolism Reports · 8 citations
https://doi.org/10.1016/j.ymgmr.2023.101005
Clinical outcome assessments of disease burden and progression in late-onset GM2 gangliosidoses
2024 · Molecular Genetics and Metabolism · 7 citations
https://doi.org/10.1016/j.ymgme.2024.108512
Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII
2022 · Molecular Genetics and Metabolism · 6 citations
https://doi.org/10.1016/j.ymgme.2022.03.002
A systematic review of ergonomic and muscular strain in surgeons comparing robotic to laparoscopic approaches
2025 · Journal of Robotic Surgery · 6 citations
https://doi.org/10.1007/s11701-025-02401-6
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