Beneath the floor: re-analysis of neurodevelopmental outcomes in untreated Hurler syndrome

Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison

(2018) Julie B Eisengart et al.   GENETICS IN MEDICINE

Cognitive outcomes and age of detection of severe mucopolysaccharidosis type 1

(2017) Scott D. Grosse et al.   GENETICS IN MEDICINE

Mucopolysaccharidosis Type I Newborn Screening: Best Practices for Diagnosis and Management

(2017) Lorne A. Clarke et al.   JOURNAL OF PEDIATRICS

Cognitive endpoints for therapy development for neuronopathic mucopolysaccharidoses: Results of a consensus procedure

(2017) Johanna H. van der Lee et al.   MOLECULAR GENETICS AND METABOLISM

Cognitive and adaptive measurement endpoints for clinical trials in mucopolysaccharidoses types I, II, and III: A review of the literature

(2017) Darren Janzen et al.   MOLECULAR GENETICS AND METABOLISM

Early disease progression of Hurler syndrome

(2017) Bridget T. Kiely et al.   Orphanet Journal of Rare Diseases

A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA

(2016) Elsa G. Shapiro et al.   JOURNAL OF PEDIATRICS

Neurocognitive clinical outcome assessments for inborn errors of metabolism and other rare conditions

(2016) Elsa Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

Long-term outcome of Hurler syndrome patients after hematopoietic cell transplantation: an international multicenter study

(2015) M. Aldenhoven et al.   BLOOD

Neurocognition across the spectrum of mucopolysaccharidosis type I: Age, severity, and treatment

(2015) Elsa G. Shapiro et al.   MOLECULAR GENETICS AND METABOLISM

Early treatment is associated with improved cognition in Hurler syndrome

(2014) Michele D. Poe et al.   ANNALS OF NEUROLOGY

Enzyme replacement therapy for Mucopolysaccharidosis Type I among patients followed within the MPS Brazil Network

(2014) Alícia Dorneles Dornelles et al.   GENETICS AND MOLECULAR BIOLOGY

Natural history of Sanfilippo syndrome type A

(2013) Dakota Buhrman et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Cardiac disease in patients with mucopolysaccharidosis: presentation, diagnosis and management

(2011) Elizabeth A. Braunlin et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Methods for assessing neurodevelopment in lysosomal storage diseases and related disorders: a multidisciplinary perspective

(2008) Holly R Martin et al.   ACTA PAEDIATRICA

Growth and endocrine function in patients with Hurler syndrome after hematopoietic stem cell transplantation

(2008) L E Polgreen et al.   BONE MARROW TRANSPLANTATION