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

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

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

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

A disease progression model for trials in mucopolysaccharidosis type IIIA

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

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

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

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

2020 ◽  
Author(s):  
Jennifer Saville ◽  
Ainslie Derrick-Roberts ◽  
Chantelle McIntyre ◽  
Maria Fuller
Keyword(s):  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

scholarly journals Mucopolysaccharidosis Type IIIA

2020 ◽  
Author(s):  
Keyword(s):  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia

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)

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

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

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

Validation of an expert system-generated checklist for the early diagnosis of mucopolysaccharidosis type IIIA and IIIB

2020 ◽  
Vol 129 (2) ◽  
pp. S118-S119
Author(s):  
Loreanne S. Oh ◽  
Michele D. Poe ◽  
Maria L. Escolar
Keyword(s):  
Expert System ◽  
Early Diagnosis ◽  
Mucopolysaccharidosis Type ◽  
Type Iiia ◽  
Mucopolysaccharidosis Type Iiia
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