A post hoc analysis of Projected Retained Ability Scores (PRAS) for the longitudinal assessment of cognitive functioning in patients with neuronopathic mucopolysaccharidosis II receiving intrathecal idursulfase-IT

Intrathecal idursulfase-IT in patients with neuronopathic mucopolysaccharidosis II: Results from a phase 2/3 randomized study

(2022) Joseph Muenzer et al.   MOLECULAR GENETICS AND METABOLISM

The natural history of neurocognition in MPS disorders: A review

(2021) Elsa Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

Analysis of cognitive ability and adaptive behavior assessment tools used in an observational study of patients with mucopolysaccharidosis II

(2021) Karen S. Yee et al.   Orphanet Journal of Rare Diseases

Evaluation of the long-term treatment effects of intravenous idursulfase in patients with mucopolysaccharidosis II (MPS II) using statistical modeling: data from the Hunter Outcome Survey (HOS)

(2021) Joseph Muenzer et al.   Orphanet Journal of Rare Diseases

Treatment of mucopolysaccharidosis type II (Hunter syndrome): a Delphi derived practice resource of the American College of Medical Genetics and Genomics (ACMG)

(2020) Kim L. McBride et al.   GENETICS IN MEDICINE

Ten years of enzyme replacement therapy in paediatric onset mucopolysaccharidosis II in England

(2019) A. Broomfield et al.   MOLECULAR GENETICS AND METABOLISM

Survival in idursulfase-treated and untreated patients with mucopolysaccharidosis type II: data from the Hunter Outcome Survey (HOS)

(2017) Barbara K. Burton et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Developmental and behavioral aspects of mucopolysaccharidoses with brain manifestations — Neurological signs and symptoms

(2017) Elsa G. Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

Clinical outcomes in idursulfase-treated patients with mucopolysaccharidosis type II: 3-year data from the hunter outcome survey (HOS)

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

Development of idursulfase therapy for mucopolysaccharidosis type II (Hunter syndrome): the past, the present and the future

(2017) David Whiteman et al.   Drug Design Development and Therapy

Presentation and treatments for Mucopolysaccharidosis Type II (MPS II; Hunter Syndrome)

(2017) Molly Stapleton et al.   Expert Opinion on Orphan Drugs

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

(2015) Joseph Muenzer et al.   GENETICS IN MEDICINE

Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease

(2011) Maurizio Scarpa et al.   Orphanet Journal of Rare Diseases

Natural Progression of Neurological Disease in Mucopolysaccharidosis Type II

(2011) J. B. Holt et al.   PEDIATRICS

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

Long-term, open-labeled extension study of idursulfase in the treatment of Hunter syndrome

(2010) Joseph Muenzer et al.   GENETICS IN MEDICINE

Initial report from the Hunter Outcome Survey

(2009) J Edmond Wraith et al.   GENETICS IN MEDICINE

Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome)

(2008) R. Martin et al.   PEDIATRICS