Improvement of CNS Defects Via Continuous Intrathecal Enzyme Replacement by Osmotic Pump in Mucopolysaccharidosis Type II Mice

Intrathecal recombinant human 4-sulfatase reduces accumulation of glycosaminoglycans in dura of mucopolysaccharidosis VI cats

(2012) Dyane Auclair et al.   PEDIATRIC RESEARCH

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

Retrospective analysis of the clinical manifestations and survival of Korean patients with mucopolysaccharidosis type II: Emphasis on the cardiovascular complication and mortality cases

(2011) Young Bae Sohn et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Controlled study of the effects of continuous intrathecal baclofen infusion in non-ambulant children with cerebral palsy

(2011) RICHARD E MORTON 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

THE NATURAL HISTORY OF THE SEVERE FORM OF HUNTER'S SYNDROME: A STUDY BASED ON 52 CASES

(2010) I. D. Young et al.   DEVELOPMENTAL MEDICINE AND CHILD NEUROLOGY

Correction of CNS defects in the MPSII mouse model via systemic enzyme replacement therapy

(2010) Vinicia Assunta Polito et al.   HUMAN MOLECULAR GENETICS

Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy

(2010) Sung-Chul Jung et al.   MOLECULES AND CELLS

Morphologic Changes Associated with Intrathecal Catheters for Direct Delivery to the Central Nervous System in Preclinical Studies

(2010) Mark T. Butt   TOXICOLOGIC PATHOLOGY

IDS Crossing of the Blood-Brain Barrier Corrects CNS Defects in MPSII Mice

(2009) Vinicia Assunta Polito et al.   AMERICAN JOURNAL OF HUMAN GENETICS

Mortality and cause of death in mucopolysaccharidosis type II—a historical review based on data from the Hunter Outcome Survey (HOS)

(2009) S. A. Jones et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Intrathecal administration of recombinant human N-acetylgalactosamine 4-sulfatase to a MPS VI patient with pachymeningitis cervicalis

(2009) María Verónica Muñoz-Rojas et al.   MOLECULAR GENETICS AND METABOLISM

Repeated intrathecal injections of recombinant human 4-sulphatase remove dural storage in mature mucopolysaccharidosis VI cats primed with a short-course tolerisation regimen

(2009) Dyane Auclair et al.   MOLECULAR GENETICS AND METABOLISM

Japan Elaprase® Treatment (JET) study: Idursulfase enzyme replacement therapy in adult patients with attenuated Hunter syndrome (Mucopolysaccharidosis II, MPS II)

(2009) Torayuki Okuyama et al.   MOLECULAR GENETICS AND METABOLISM

Safety and one-year efficacy of intrathecal baclofen therapy in children with intractable spastic cerebral palsy

(2008) Marjanke A. Hoving et al.   EUROPEAN JOURNAL OF PAEDIATRIC NEUROLOGY

Neurological findings in Hunter disease: Pathology and possible therapeutic effects reviewed

(2008) S. Al Sawaf et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Acidic amino acid tag enhances response to enzyme replacement in mucopolysaccharidosis type VII mice

(2008) Adriana M. Montaño et al.   MOLECULAR GENETICS AND METABOLISM