Therapy for the mucopolysaccharidoses

Idursulfase treatment of Hunter syndrome in children younger than 6 years: Results from the Hunter Outcome Survey

(2011) Joseph Muenzer et al.   GENETICS IN MEDICINE

Genistein: a natural isoflavone with a potential for treatment of genetic diseases

(2010) Grzegorz Węgrzyn et al.   BIOCHEMICAL SOCIETY TRANSACTIONS

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

(2010) Joseph Muenzer et al.   GENETICS IN MEDICINE

Enzyme replacement therapy for mucopolysaccharidosis VI: evaluation of long-term pulmonary function in patients treated with recombinant human N-acetylgalactosamine 4-sulfatase

(2010) Paul Harmatz et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Mucopolysaccharidosis VI

(2010) Vassili Valayannopoulos et al.   Orphanet Journal of Rare Diseases

Polysomnographic characteristics in patients with mucopolysaccharidoses

(2010) Hsiang-Yu Lin et al.   PEDIATRIC PULMONOLOGY

Laronidase for Cardiopulmonary Disease in Hurler Syndrome 12 Years After Bone Marrow Transplantation

(2010) V. Valayannopoulos et al.   PEDIATRICS

Risk Factor Analysis of Outcomes after Unrelated Cord Blood Transplantation in Patients with Hurler Syndrome

(2009) Jaap Jan Boelens et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Clinical overview of children with mucopolysaccharidosis type III A and effect of Risperidone treatment on children and their mothers psychological status

(2009) Sema Kalkan Ucar et al.   BRAIN & DEVELOPMENT

Human α-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution

(2009) Carine Ciron et al.   HUMAN GENE THERAPY

AGT-181: Expression in CHO cells and pharmacokinetics, safety, and plasma iduronidase enzyme activity in Rhesus monkeys

(2009) Ruben J. Boado et al.   JOURNAL OF BIOTECHNOLOGY

Development of Novel Aminoglycoside (NB54) with Reduced Toxicity and Enhanced Suppression of Disease-Causing Premature Stop Mutations

(2009) Igor Nudelman et al.   JOURNAL OF MEDICINAL CHEMISTRY

Combined enzyme replacement and haematopoietic stem cell transplantation in Hurler syndrome

(2009) Sunita Bijarnia et al.   JOURNAL OF PAEDIATRICS AND CHILD HEALTH

Bone Marrow Transplantation in Children with Hunter Syndrome: Outcome after 7 to 17 Years

(2009) Nathalie Guffon et al.   JOURNAL OF PEDIATRICS

Treatment reduces or stabilizes brain imaging abnormalities in patients with MPS I and II

(2009) Raymond Y. Wang et al.   MOLECULAR GENETICS AND METABOLISM

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

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

Enzyme-Replacement Therapy in a 5-Month-Old Boy With Attenuated Presymptomatic MPS I: 5-Year Follow-up

(2009) O. Gabrielli et al.   PEDIATRICS

Multidisciplinary Management of Hunter Syndrome

(2009) J. Muenzer et al.   PEDIATRICS

Transplant Outcomes in Mucopolysaccharidoses

(2009) Vinod K. Prasad et al.   SEMINARS IN HEMATOLOGY

Progression of organ manifestations upon enzyme replacement therapy in a patient with mucopolysaccharidosis type I/Hurler

(2009) Saadet Mercimek-Mahmutoglu et al.   World Journal of Pediatrics

Intrathecal enzyme replacement therapy in a patient with mucopolysaccharidosis type I and symptomatic spinal cord compression

(2008) Maria-Veronica Munoz-Rojas et al.   AMERICAN JOURNAL OF MEDICAL GENETICS PART A

Incidence and prevalence of mucopolysaccharidosis type 1 in the Irish republic

(2008) A M Murphy et al.   ARCHIVES OF DISEASE IN CHILDHOOD

Treating lysosomal storage disorders: Current practice and future prospects

(2008) Frances M. Platt et al.   BIOCHIMICA ET BIOPHYSICA ACTA-MOLECULAR CELL RESEARCH

The Clinical Outcome of Hurler Syndrome after Stem Cell Transplantation

(2008) Mieke Aldenhoven et al.   BIOLOGY OF BLOOD AND MARROW TRANSPLANTATION

Sanfilippo syndrome: A mini-review

(2008) M. J. Valstar et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Use of Enzyme Replacement Therapy (Laronidase) before Hematopoietic Stem Cell Transplantation for Mucopolysaccharidosis I: Experience in 18 Patients

(2008) Robert F. Wynn et al.   JOURNAL OF PEDIATRICS

Clinical trials of orphan medicines

(2008) Brendan M Buckley   LANCET

Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy

(2008) Ramin Sedaghat Herati et al.   MOLECULAR GENETICS AND METABOLISM

Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase

(2008) Paul Harmatz et al.   MOLECULAR GENETICS AND METABOLISM

A dose-optimization trial of laronidase (Aldurazyme®) in patients with mucopolysaccharidosis I

(2008) Roberto Giugliani et al.   MOLECULAR GENETICS AND METABOLISM

The prevalence of and survival in Mucopolysaccharidosis I: Hurler, Hurler-Scheie and Scheie syndromes in the UK

(2008) David Moore et al.   Orphanet Journal of Rare Diseases

Recognition and Diagnosis of Mucopolysaccharidosis II (Hunter Syndrome)

(2008) R. Martin et al.   PEDIATRICS

Long-term Efficacy and Safety of Laronidase in the Treatment of Mucopolysaccharidosis I

(2008) L. A. Clarke et al.   PEDIATRICS

Mucopolysaccharidosis I: Management and Treatment Guidelines

(2008) J. Muenzer et al.   PEDIATRICS

Current Strategies in the Management of Lysosomal Storage Diseases

(2008) Bryce A. Heese   Seminars in Pediatric Neurology

A counterintuitive approach to treat enzyme deficiencies: use of enzyme inhibitors for restoring mutant enzyme activity

(2007) Jian-Qiang Fan   BIOLOGICAL CHEMISTRY