Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres. - Wikidata - wikimedia - TriplyDB

Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres.

Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres.

http://www.wikidata.org/entity/Q35254540

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Therapies for the bone in mucopolysaccharidoses Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review.Corneal hysteresis in mucopolysaccharidosis I and VI.Growth patterns and the use of growth hormone in the mucopolysaccharidoses.From gene transfer to gene therapy in lysosomal storage diseases affecting the central nervous system.Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.Heparin cofactor II-thrombin complex and dermatan sulphate:chondroitin sulphate ratio are biomarkers of short- and long-term treatment effects in mucopolysaccharide diseases Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation Lysosomal storage disorders: diagnostic dilemmas and prospects for therapy.Hematopoietic cell transplantation for inherited metabolic diseases: an overview of outcomes and practice guidelines.Can mucopolysaccharidosis type I disease severity be predicted based on a patient's genotype? A comprehensive review of the literature.Long-term functional outcomes of children with hurler syndrome treated with unrelated umbilical cord blood transplantation.Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome.Physical performance testing in mucopolysaccharidosis I: a pilot study.Prevalence and development of orthopaedic symptoms in the dutch hurler patient population after haematopoietic stem cell transplantation.AAV Gene Therapy for MPS1-associated Corneal Blindness Management of otolaryngological manifestations in mucopolysaccharidoses: our experience.Long-Term Cognitive and Functional Outcomes in Children with Mucopolysaccharidosis (MPS)-IH (Hurler Syndrome) Treated with Hematopoietic Cell Transplantation.Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure.Anterior segment OCT imaging in mucopolysaccharidoses type I, II, and VI.Strategies for delivery of therapeutics into the central nervous system for treatment of lysosomal storage disorders.New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders Orthopaedic aspects of mucopolysaccharidoses.Cognitive outcomes and age of detection of severe mucopolysaccharidosis type 1.Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience.Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modifying factor 1.alpha-L-iduronidase therapy for mucopolysaccharidosis type I.Characterization of knee alignment in children with mucopolysaccharidosis types I and II and outcome of treatment with guided growth.Precocious initiation of spermatogenesis in a 19-month-old boy with Hurler syndrome.An algorithm to predict phenotypic severity in mucopolysaccharidosis type I in the first month of life.In vitro gene therapy of mucopolysaccharidosis type I by lentiviral vectors.Haematopoietic cell transplantation (HCT) in combination with enzyme replacement therapy (ERT) in patients with Hurler syndrome.Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H).The effect of haemopoietic stem cell transplantation on the ocular phenotype in mucopolysaccharidosis type I (Hurler).Early treatment is associated with improved cognition in Hurler syndrome.Variable disease progression after successful stem cell transplantation: prospective follow-up investigations in eight patients with Hurler syndrome.Outcome of second hematopoietic cell transplantation in Hurler syndrome.Mucopolysaccharidosis type I in 21 Czech and Slovak patients: mutation analysis suggests a functional importance of C-terminus of the IDUA protein.

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Bone marrow transplantation for mucopolysaccharidosis type I: experience of two British centres.

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P2860

A simple salting out procedure for extracting DNA from human nucleated cells

Rapid typing of tandemly repeated hypervariable loci by the polymerase chain reaction: application to the apolipoprotein B 3' hypervariable region.

Long-term effects of bone marrow transplantation in dogs with mucopolysaccharidosis I.

Cardiac involvement in mucopolysaccharidoses: effects of allogeneic bone marrow transplantation.

Bone marrow transplantation corrects the enzyme defect in neurons of the central nervous system in a lysosomal storage disease

The systemic mucopolysaccharidoses.

Molecular genetics of mucopolysaccharidosis type I: diagnostic, clinical, and biological implications.

The laboratory diagnosis of Sanfilippo disease.

Amplification of a variable number of tandem repeats (VNTR) locus (pMCT118) by the polymerase chain reaction (PCR) and its application to forensic science.

Mutation in Scheie syndrome (MPS IS): a G-->A transition creates new splice site in intron 5 of one IDUA allele.

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