ArticleMolecular therapy. Methods & clinical development2017
Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice.
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Article in Molecular therapy. Methods & clinical development, 2017. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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0cells of the map it votes in
24citing papers in PubMed
3.1field-weighted citation impact, top 8% of its field
What it found
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
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Who cites it
24 citing papers in PubMed, 37 citations in OpenAlex.
- A phase I/II study on intracerebroventricular tralesinidase alfa in patients with Sanfilippo syndrome type B.The Journal of clinical investigation · 2023Trial
- Evaluation of GlcNAc-Configured Glycomimetics as Pharmacological Chaperones of NAGLU for the Treatment of Mucopolysaccharidosis IIIB.Biomolecules · 2026Article
- Intracerebroventricular administration of a modified hexosaminidase ameliorates late-stage neurodegeneration in a GM2 mouse model.PloS one · 2025Article
- Safety, pharmacokinetics and CNS distribution of tralesinidase alfa administered via intracerebroventricular infusion to juvenile cynomolgus monkeys.Toxicology reports · 2023Article
- Tralesinidase Alfa Enzyme Replacement Therapy Prevents Disease Manifestations in a Canine Model of Mucopolysaccharidosis Type IIIB.The Journal of pharmacology and experimental therapeutics · 2022Article
- Promoter considerations in the design of lentiviral vectors for use in treating lysosomal storage diseases.Molecular therapy. Methods & clinical development · 2022Review
- Molecular environment and atypical function: What do we know about enzymes associated with Mucopolysaccharidoses?Orphanet journal of rare diseases · 2022Review
- Biochemical evaluation of intracerebroventricular rhNAGLU-IGF2 enzyme replacement therapy in neonatal mice with Sanfilippo B syndrome.Molecular genetics and metabolism · 2021Article
- Enzyme Replacement Therapy for Mucopolysaccharidosis IIID using Recombinant Human α-Molecular pharmaceutics · 2021Article
- The Unifying Hypothesis of Alzheimer's Disease: Heparan Sulfate Proteoglycans/Glycosaminoglycans Are Key as First Hypothesized Over 30 Years Ago.Frontiers in aging neuroscience · 2021Article
- Two Distinct Lysosomal Targeting Strategies Afford Trojan Horse Antibodies With Pan-Filovirus Activity.Frontiers in immunology · 2021Article
- Central nervous system pathology in preclinical MPS IIIB dogs reveals progressive changes in clinically relevant brain regions.Scientific reports · 2020Article
- Intracerebroventricular enzyme replacement therapy with β-galactosidase reverses brain pathologies due to GM1 gangliosidosis in mice.The Journal of biological chemistry · 2020Article
- Translational studies of intravenous and intracerebroventricular routes of administration for CNS cellular biodistribution for BMN 250, an enzyme replacement therapy for the treatment of Sanfilippo type B.Drug delivery and translational research · 2020Article
- A Cure for Sanfilippo Syndrome? A Summary of Current Therapeutic Approaches and their Promise.Medical research archives · 2020Article
- Evaluation of non-reducing end pathologic glycosaminoglycan detection method for monitoring therapeutic response to enzyme replacement therapy in human mucopolysaccharidosis I.Molecular genetics and metabolism · 2020Article
- Enzyme replacement therapy for mucopolysaccharidoses; past, present, and future.Journal of human genetics · 2019Review
- Differential Uptake of NAGLU-IGF2 and Unmodified NAGLU in Cellular Models of Sanfilippo Syndrome Type B.Molecular therapy. Methods & clinical development · 2019Article
- Assessment of predicted enzymatic activity of α-N-acetylglucosaminidase variants of unknown significance for CAGI 2016.Human mutation · 2019Article
- Review
Corrections and comments
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Authors and funding
25 authors at 1 institution in 1 country.
Mika Aoyagi-ScharberResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Danielle Crippen-HarmonResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Roger LawrenceResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Jon VinceletteResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Gouri YogalingamResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Heather PrillResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Bryan K YipResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Brian BaridonResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Catherine VitelliResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Amanda LeeResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Olivia GorostizaResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Evan G AdintoriResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Wesley C MintoResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Jeremy L Van VleetResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Bridget YatesResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Sara RigneyResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Terri M ChristiansonResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Pascale M N TigerResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Melanie J LoResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
John HoltzingerResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Paul A FitzpatrickResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Jonathan H LeBowitzResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Sherry BullensResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Brett E CrawfordResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
Stuart BuntingResearch, BioMarin Pharmaceutical Inc., 105 Digital Drive, Novato, CA 94949, USA.
BioMarin (United States) · US
Funding
No grant is acknowledged in the PubMed record.
Abstract
Sanfilippo syndrome type B (mucopolysaccharidosis IIIB), caused by inherited deficiency of α-
Indexed as
enzyme replacement therapyglycosaminoglycanintracerebroventricular deliverylysosomal storage disordermental retardationMPS IIIBmucopolysaccharidosis IIIBneurodegenerationSanfilippo B
Identifiers
What OpenQuestion holds
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LicenceCC BY
Read underepoch 390
Registered trials
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.