Lau Heather - Department of Neurology, New York University

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论文共 71 篇作者统计合作学者相似作者

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Clinical outcome assessments of disease burden and progression in late-onset GM2 gangliosidoses

Julie Kissell,Camille Rochmann,Pascal Minini,Florian Eichler, Christopher D Stephen,Heather Lau,Camilo Toro,Jean M Johnston, Robert Krupnick, Alaa Hamed,Gerald F Cox

Molecular genetics and metabolismno. 3 (2024): 108512-108512

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Long-Term Treatment of Gaucher Disease with Velaglucerase Alfa in ERT-Naïve Patients from the Gaucher Outcome Survey (GOS) Registry

Patrick Deegan,Heather Lau,Deborah Elstein,Diego Fernandez-Sasso,Pilar Giraldo,Derralynn Hughes,Ari Zimran,Majdolen Istaiti,Noga Gadir,Jaco Botha,Shoshana Revel-Vilk, GOS Study Group

Journal of clinical medicineno. 10 (2024): 2782

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Clinical characteristics and somatic burden of patients with mucopolysaccharidosis II with or without neurological involvement: An analysis from the Hunter Outcome Survey

Heather Lau,Paul Harmatz,Jaco Botha,Jennifer Audi,Bianca Link

Molecular Genetics and Metabolism Reports（2023）

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Reply to Mistry et al. The Two Substrate Reduction Therapies for Type 1 Gaucher Disease Are Not Equivalent. Comment on "Hughes et al. Switching between Enzyme Replacement Therapies and Substrate Reduction Therapies in Patients with Gaucher Disease: Data from the Gaucher Outcome Survey (GOS). J. Clin. Med. 2022, 11 , 5158"

Derralynn A Hughes,Patrick Deegan,Pilar Giraldo,Özlem Göker-Alpan,Heather Lau,Elena Lukina,Shoshana Revel-Vilk,Maurizio Scarpa,Jaco Botha,Noga Gadir,Ari Zimran

Journal of clinical medicineno. 12 (2023): 4017-4017

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The risk of hepatocellular carcinoma is markedly increased in Gaucher disease

Mohsen Basiri,Jiapeng Ruan,Shiny Nair,Heather A. Lau,Pramod K. Mistry,Tamar Taddei

HEPATOLOGY（2023）

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Screening, patient identification, evaluation, and treatment in patients with Gaucher disease: Results from a Delphi consensus

Priya S Kishnani,Walla Al-Hertani,Manisha Balwani,Özlem Göker-Alpan,Heather A Lau,Melissa Wasserstein,Neal J Weinreb,Gregory Grabowski

MOLECULAR GENETICS AND METABOLISM（2022）

引用7浏览0WOS引用

First-in-human in vivo genome editing via AAV-zinc finger nucleases for mucopolysaccharidosis I/II and hemophilia B

Paul Harmatz,Carlos E Prada,Barbara K Burton,Heather Lau,Craig M Kessler,Liching Cao,Marina Falaleeva, Andres G Villegas, Jennifer Zeitler,Kathleen Meyer,Weston Miller,Cheryl Wong Po Foo,

Molecular therapy : the journal of the American Society of Gene Therapy（2022）

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Efficacy and Safety of N-Acetyl-l-Leucine in Children and Adults With GM2 Gangliosidoses

Kyriakos Martakis,Jens Claassen, Jordi Gascon-Bayari,Nicolina Goldschagg,Andreas Hahn,Anhar Hassan,Anita Hennig,Simon Jones,Richard Kay,Heather Lau,Susan Perlman,Reena Sharma,

Neurology（2022）

引用4浏览0WOS引用

First-in-human in vivo genome editing via AAV-zinc-finger nucleases for mucopolysaccharidosis I/II and hemophilia B

Paul Harmatz,Carlos E Prada,Barbara K Burton,Heather Lau,Craig M Kessler,Liching Cao,Marina Falaleeva, Andres G Villegas, Jennifer Zeitler,Kathleen Meyer,Weston Miller,Cheryl Wong Po Foo,

Molecular therapy : the journal of the American Society of Gene Therapyno. 12 (2022): 3587-3600

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Long-term efficacy and safety of vestronidase alfa enzyme replacement therapy in pediatric subjects < 5 years with mucopolysaccharidosis VII

Heather A Lau,David Viskochil,Pranoot Tanpaiboon,Antonio Gonzalez-Meneses Lopez,Esmeralda Martins,Julie Taylor, Betsy Malkus,Lin Zhang,Agnieszka Jurecka,Deborah Marsden

MOLECULAR GENETICS AND METABOLISM（2022）

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