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

Final results of the first-in-human open-label study of intravenous SBC-103 in children with mucopolysaccharidosis type IIIB

(2018) Chester Whitley et al.   MOLECULAR GENETICS AND METABOLISM

Whole Body and CNS Biodistribution of rhHNS in Cynomolgus Monkeys after Intrathecal Lumbar Administration: Treatment Implications for Patients with MPS IIIA

(2017) Jou-Ku Chung et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Clearance of Heparan Sulfate and Attenuation of CNS Pathology by Intracerebroventricular BMN 250 in Sanfilippo Type B Mice

(2017) Mika Aoyagi-Scharber et al.   Molecular Therapy-Methods & Clinical Development

From blood–brain barrier to blood–brain interface: new opportunities for CNS drug delivery

(2016) William A. Banks   NATURE REVIEWS DRUG DISCOVERY

Evaluation of blood–brain barrier integrity and structural abnormalities in MPS IIIB patients using cerebrospinal fluid/serum albumin index (CSF-AI) and multimodal MRI

(2015) Suresh Vijay et al.   MOLECULAR GENETICS AND METABOLISM

Pharmacokinetics and Bioavailability of a Therapeutic Enzyme (Idursulfase) in Cynomolgus Monkeys after Intrathecal and Intravenous Administration

(2015) Hongsheng Xie et al.   PLoS One

Intravenous SBC-103, a recombinant human alpha-N-acetylglucosaminidase reduces CNS heparan sulfate content in a mucopolysaccharidosis type IIIB mouse model

(2014) Joseph V. Rutkowski et al.   MOLECULAR GENETICS AND METABOLISM

Signaling molecules regulating phenotypic conversions of astrocytes and glial scar formation in damaged nerve tissues

(2014) Yutaka Koyama   NEUROCHEMISTRY INTERNATIONAL

Recombinant human tripeptidyl peptidase-1 infusion to the monkey CNS: Safety, pharmacokinetics, and distribution

(2014) Brian R. Vuillemenot et al.   TOXICOLOGY AND APPLIED PHARMACOLOGY

Astrogliosis

(2014) Michael V. Sofroniew   Cold Spring Harbor Perspectives in Biology

Glycan-based biomarkers for mucopolysaccharidoses

(2013) Roger Lawrence et al.   MOLECULAR GENETICS AND METABOLISM

Astrocyte-derived VEGF-A drives blood-brain barrier disruption in CNS inflammatory disease

(2012) Azeb Tadesse Argaw et al.   JOURNAL OF CLINICAL INVESTIGATION

Disease-specific non–reducing end carbohydrate biomarkers for mucopolysaccharidoses

(2012) Roger Lawrence et al.   Nature Chemical Biology

NIH Image to ImageJ: 25 years of image analysis

(2012) Caroline A Schneider et al.   NATURE METHODS

Blood-Brain Barrier Impairment in an Animal Model of MPS III B

(2011) Svitlana Garbuzova-Davis et al.   PLoS One

Defects in the Medial Entorhinal Cortex and Dentate Gyrus in the Mouse Model of Sanfilippo Syndrome Type B

(2011) Kazuhiro Ohmi et al.   PLoS One

Astrocytes: biology and pathology

(2009) Michael V. Sofroniew et al.   ACTA NEUROPATHOLOGICA

Effect of cisternal sulfamidase delivery in MPS IIIA Huntaway dogs—A proof of principle study

(2009) Kim M. Hemsley et al.   MOLECULAR GENETICS AND METABOLISM

The bright side of the glial scar in CNS repair

(2009) Asya Rolls et al.   NATURE REVIEWS NEUROSCIENCE

Mannose 6-Phosphate Receptor–mediated Transport of Sulfamidase Across the Blood–brain Barrier in the Newborn Mouse

(2008) Akihiko Urayama et al.   MOLECULAR THERAPY

Disaccharide structure code for the easy representation of constituent oligosaccharides from glycosaminoglycans

(2008) Roger Lawrence et al.   NATURE METHODS