mucopolysaccharidosis type iiia
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An observational, prospective, multicenter, natural history study of patients with mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2021.12.002 ◽

2021 ◽

Author(s):

Frits A. Wijburg ◽

Karen Aiach ◽

Anupam Chakrapani ◽

Julie B. Eisengart ◽

Roberto Giugliani ◽

...

Keyword(s):

Natural History ◽

Mucopolysaccharidosis Type ◽

Natural History Study ◽

Type Iiia ◽

Mucopolysaccharidosis Type Iiia

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Ex-vivo autologous stem cell gene therapy clinical trial for mucopolysaccharidosis type IIIA: Update on phase I/II clinical trial

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.126 ◽

2021 ◽

Vol 132 (2) ◽

pp. S56-S57

Author(s):

Jane Louise Kinsella ◽

Simon Jones ◽

Adrian J. Thrasher ◽

Claire Booth ◽

Karen F. Buckland ◽

...

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Stem Cell ◽

Ex Vivo ◽

Mucopolysaccharidosis Type ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Therapy Clinical Trial ◽

Stem Cell Gene Therapy

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A disease progression model for trials in mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.157 ◽

2021 ◽

Vol 132 (2) ◽

pp. S69

Author(s):

Joe Marion ◽

Ben Saville ◽

Juan Ruiz

Keyword(s):

Disease Progression ◽

Mucopolysaccharidosis Type ◽

Progression Model ◽

Type Iiia ◽

Disease Progression Model ◽

Mucopolysaccharidosis Type Iiia

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Non-clinical evaluation of a blood-brain barrier-penetrable N-sulfoglucosamine sulfohydrolase in a mouse model of mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2020.12.258 ◽

2021 ◽

Vol 132 (2) ◽

pp. S105-S106

Author(s):

Satowa Tanaka ◽

Sachiho Kida ◽

Yuri Koshimura ◽

Shinji Kakimoto ◽

Atsushi Imakiire ◽

...

Keyword(s):

Mouse Model ◽

Blood Brain Barrier ◽

Clinical Evaluation ◽

Brain Barrier ◽

Mucopolysaccharidosis Type ◽

Type Iiia ◽

Blood Brain ◽

Mucopolysaccharidosis Type Iiia

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Systemic scAAV9.U1a.hSGSH delivery corrects brain biochemistry in mucopolysaccharidosis type IIIA at early and later stages of disease

Human Gene Therapy ◽

10.1089/hum.2020.253 ◽

2020 ◽

Author(s):

Jennifer Saville ◽

Ainslie Derrick-Roberts ◽

Chantelle McIntyre ◽

Maria Fuller

Keyword(s):

Mucopolysaccharidosis Type ◽

Type Iiia ◽

Mucopolysaccharidosis Type Iiia

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Hematopoietic stem cell transplantation in mucopolysaccharidosis type IIIA: A case description and comparison with a genotype-matched control group

Molecular Genetics and Metabolism Reports ◽

10.1016/j.ymgmr.2020.100578 ◽

2020 ◽

Vol 23 ◽

pp. 100578 ◽

Author(s):

Anja F. Köhn ◽

Lorenz Grigull ◽

Marcel du Moulin ◽

Sarah Kabisch ◽

Luise Ammer ◽

...

Keyword(s):

Stem Cell ◽

Hematopoietic Stem Cell Transplantation ◽

Stem Cell Transplantation ◽

Matched Control ◽

Control Group ◽

Mucopolysaccharidosis Type ◽

Case Description ◽

Hematopoietic Stem ◽

Matched Control Group ◽

Mucopolysaccharidosis Type Iiia

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Mucopolysaccharidosis Type IIIA

10.32388/wd98g0 ◽

2020 ◽

Author(s):

Keyword(s):

Mucopolysaccharidosis Type ◽

Type Iiia ◽

Mucopolysaccharidosis Type Iiia

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Interim results of Transpher A, a multicenter, single-dose, phase 1/2 clinical trial of ABO-102 gene therapy for Sanfilippo syndrome type A (mucopolysaccharidosis type IIIA)

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.11.129 ◽

2020 ◽

Vol 129 (2) ◽

pp. S56-S57 ◽

Author(s):

Kevin M. Flanigan ◽

Nicholas J.C. Smith ◽

Maria Luz Couce ◽

Kristen V. Truxal ◽

Kim L. McBride ◽

...

Keyword(s):

Clinical Trial ◽

Gene Therapy ◽

Single Dose ◽

Phase 1 ◽

Type A ◽

Syndrome Type ◽

Mucopolysaccharidosis Type ◽

Sanfilippo Syndrome ◽

Type Iiia ◽

Mucopolysaccharidosis Type Iiia

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Case report of the first patient treated with ex-vivo autologous haematopoietic stem cell gene therapy transplant in mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.11.225 ◽

2020 ◽

Vol 129 (2) ◽

pp. S90 ◽

Author(s):

Jane Louise Kinsella ◽

Heather Church ◽

Wendy Ogden ◽

Ceri Jones ◽

Karen Buckland ◽

...

Keyword(s):

Gene Therapy ◽

Stem Cell ◽

Case Report ◽

Ex Vivo ◽

Haematopoietic Stem Cell ◽

Mucopolysaccharidosis Type ◽

Cell Gene ◽

Mucopolysaccharidosis Type Iiia ◽

Stem Cell Gene ◽

Stem Cell Gene Therapy

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Sphingolipid dyshomeostasis in the brain of the mouse model of mucopolysaccharidosis type IIIA

Molecular Genetics and Metabolism ◽

10.1016/j.ymgme.2019.08.008 ◽

2020 ◽

Vol 129 (2) ◽

pp. 111-116 ◽

Author(s):

Jennifer T. Saville ◽

Maria Fuller

Keyword(s):

Mouse Model ◽

Mucopolysaccharidosis Type ◽

Type Iiia ◽

The Brain ◽

Mucopolysaccharidosis Type Iiia

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