Pharmacokinetic Modeling of Intrathecally Administered Recombinant Human Arylsulfatase A (TAK‐611) in Children With Metachromatic Leukodystrophy

Nonclinical comparability studies of recombinant human arylsulfatase A addressing manufacturing process changes

(2018) Teresa Wright et al.   PLoS One

Intrathecal antibody distribution in the rat brain: surface diffusion, perivascular transport and osmotic enhancement of delivery

(2017) Michelle E. Pizzo et al.   JOURNAL OF PHYSIOLOGY-LONDON

Transependymal Cerebrospinal Fluid Flow: Opportunity for Drug Delivery?

(2017) João Casaca-Carreira et al.   MOLECULAR NEUROBIOLOGY

Ten years of the Hunter Outcome Survey (HOS): insights, achievements, and lessons learned from a global patient registry

(2017) Joseph Muenzer et al.   Orphanet Journal of Rare Diseases

Outflow of cerebrospinal fluid is predominantly through lymphatic vessels and is reduced in aged mice

(2017) Qiaoli Ma et al.   Nature Communications

The Paravascular Pathway for Brain Waste Clearance: Current Understanding, Significance and Controversy

(2017) Andrew Bacyinski et al.   Frontiers in Neuroanatomy

Lymphatic Clearance of the Brain: Perivascular, Paravascular and Significance for Neurodegenerative Diseases

(2016) Erik N. T. P. Bakker et al.   CELLULAR AND MOLECULAR NEUROBIOLOGY

Metachromatic Leukodystrophy

(2016) Florian S. Eichler et al.   JOURNAL OF CHILD NEUROLOGY

Metachromatic leukodystrophy: Disease spectrum and approaches for treatment

(2015) Diane F. van Rappard et al.   BEST PRACTICE & RESEARCH CLINICAL ENDOCRINOLOGY & METABOLISM

Enzymes Approved for Human Therapy: Indications, Mechanisms and Adverse Effects

(2015) Brian A. Baldo   BIODRUGS

Enzyme Replacement Therapies and Immunogenicity in Lysosomal Storage Diseases: Is There a Pattern?

(2015) Paul Harmatz   CLINICAL THERAPEUTICS

A phase I/II study of intrathecal idursulfase-IT in children with severe mucopolysaccharidosis II

(2015) Joseph Muenzer et al.   GENETICS IN MEDICINE

Mutation Update ofARSAandPSAPGenes Causing Metachromatic Leukodystrophy

(2015) Martina Cesani et al.   HUMAN MUTATION

Long-term outcomes after allogeneic hematopoietic stem cell transplantation for metachromatic leukodystrophy: the largest single-institution cohort report

(2015) Alexander A. Boucher et al.   Orphanet Journal of Rare Diseases

Immunogenicity of idursulfase and clinical outcomes in very young patients (16 months to 7.5 years) with mucopolysaccharidosis II (Hunter syndrome)

(2015) Arian Pano et al.   Orphanet Journal of Rare Diseases

Language and cognition in children with metachromatic leukodystrophy: onset and natural course in a nationwide cohort

(2014) Christiane Kehrer et al.   Orphanet Journal of Rare Diseases

Neurodevelopmental Outcomes of Umbilical Cord Blood Transplantation in Metachromatic Leukodystrophy

(2013) Holly R. Martin et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Cerebral gray and white matter changes and clinical course in metachromatic leukodystrophy

(2012) S. Groeschel et al.   NEUROLOGY

CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder

(2012) Pericles Calias et al.   PLoS One

A Paravascular Pathway Facilitates CSF Flow Through the Brain Parenchyma and the Clearance of Interstitial Solutes, Including Amyloid  

(2012) J. J. Iliff et al.   Science Translational Medicine

The natural course of gross motor deterioration in metachromatic leukodystrophy

(2011) CHRISTIANE KEHRER et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Safety Evaluation of Chronic Intrathecal Administration of Idursulfase-IT in Cynomolgus Monkeys

(2011) Brian R. Felice et al.   TOXICOLOGIC PATHOLOGY

Anatomy and physiology of cerebrospinal fluid

(2011) L. Sakka et al.   European Annals of Otorhinolaryngology-Head and Neck Diseases

Intracisternal enzyme replacement therapy in lysosomal storage diseases: routes of absorption into brain

(2010) R. D. Jolly et al.   NEUROPATHOLOGY AND APPLIED NEUROBIOLOGY

Metachromatic Leukodystrophy – An Update

(2010) V. Gieselmann et al.   NEUROPEDIATRICS

Metachromatic leukodystrophy: genetics, pathogenesis and therapeutic options

(2008) Volkmar Gieselmann   ACTA PAEDIATRICA

Metachromatic leukodystrophy: an overview of current and prospective treatments

(2008) A Biffi et al.   BONE MARROW TRANSPLANTATION

Likelihood based approaches to handling data below the quantification limit using NONMEM VI

(2008) Jae Eun Ahn et al.   JOURNAL OF PHARMACOKINETICS AND PHARMACODYNAMICS

Neutralizing antibodies to therapeutic enzymes: considerations for testing, prevention and treatment

(2008) Jinhai Wang et al.   NATURE BIOTECHNOLOGY