Pompe's disease (Q37302098)

19 August 2017

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title

Pompe's disease. (English)

1 reference

19 August 2017

Ans T van der Ploeg

1

1 reference

PubMed ID

18929906

19 August 2017

Arnold J J Reuser

series ordinal

2

1 reference

PubMed ID

18929906

19 August 2017

language of work or name

English

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publication date

1 October 2008

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19 August 2017

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19 August 2017

372

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19 August 2017

1342-1353

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19 August 2017

9646

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Frequency of glycogen storage disease type II in The Netherlands: implications for diagnosis and genetic counselling

19 August 2017

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Pompe's disease in Chinese and prenatal diagnosis by determination of alpha-glucosidase activity

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Carrier frequency for glycogen storage disease type II in New York and estimates of affected individuals born with the disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

The frequency of lysosomal storage diseases in The Netherlands.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Prevalence of lysosomal storage disorders

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

The spectrum and diagnosis of acid maltase deficiency.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

A retrospective, multinational, multicenter study on the natural history of infantile-onset Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Identification of two subtypes of infantile acid maltase deficiency

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Diagnostic challenges for Pompe disease: an under-recognized cause of floppy baby syndrome

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Development of obstruction to ventricular outflow and impairment of inflow in glycogen storage disease of the heart: serial echocardiographic studies from birth to death at 6 months

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Electrocardiographic response to enzyme replacement therapy for Pompe disease.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Hearing loss in infantile Pompe's disease and determination of underlying pathology in the knockout mouse

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Pompe disease diagnosis and management guideline.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Fractures in children with Pompe disease: a potential long-term complication

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Juvenile and adult-onset acid maltase deficiency in France: genotype-phenotype correlation

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Genotype-phenotype correlation in adult-onset acid maltase deficiency

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

The natural course of non-classic Pompe's disease; a review of 225 published cases

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Disease severity in children and adults with Pompe disease related to age and disease duration.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

A rare presentation of childhood pompe disease: cardiac involvement provoked by Epstein-Barr virus infection

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Efficacy of multidisciplinary approach in the treatment of two cases of nonclassical infantile glycogenosis type II.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Juvenile onset acid maltase deficiency presenting as a rigid spine syndrome

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Clinical manifestation and natural course of late-onset Pompe's disease in 54 Dutch patients

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Respiratory failure in Pompe disease: treatment with noninvasive ventilation.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Sleep-disordered breathing and respiratory failure in acid maltase deficiency.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Respiratory insufficiency and limb muscle weakness in adults with Pompe's disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Course of disability and respiratory function in untreated late-onset Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Late-onset acid maltase deficiency associated with intracranial aneurysm

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Fusiform aneurysm of basilar artery and ectatic internal carotid arteries associated with glycogenosis type 2 (Pompe's disease)

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

alpha-Glucosidase deficiency in generalized glycogenstorage disease (Pompe's disease).

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Residual acid maltase activity in late-onset acid maltase deficiency

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Correlation of acid alpha-glucosidase and glycogen content in skin fibroblasts with age of onset in Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Glycogenosis type II (acid maltase deficiency)

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

A new diagnostic assay for glycogen storage disease type II in mixed leukocytes.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

The use of acarbose inhibition in the measurement of acid alpha-glucosidase activity in blood lymphocytes for the diagnosis of Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Glycogen storage disease type II: enzymatic screening in dried blood spots on filter paper

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Direct multiplex assay of enzymes in dried blood spots by tandem mass spectrometry for the newborn screening of lysosomal storage disorders

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Direct multiplex assay of lysosomal enzymes in dried blood spots for newborn screening

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Comparison of maltose and acarbose as inhibitors of maltase-glucoamylase activity in assaying acid alpha-glucosidase activity in dried blood spots for the diagnosis of infantile Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Newborn screening for lysosomal storage disorders

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Rapid diagnosis of late-onset Pompe disease by fluorometric assay of alpha-glucosidase activities in dried blood spots

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

High incidence of later-onset fabry disease revealed by newborn screening

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Isolation of a cDNA for human acid alpha-glucosidase and detection of genetic heterogeneity for mRNA in three alpha-glucosidase-deficient patients

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

A de novo 13 nt deletion, a newly identified C647W missense mutation and a deletion of exon 18 in infantile onset glycogen storage disease type II (GSDII).

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Homozygosity for multiple contiguous single-nucleotide polymorphisms as an indicator of large heterozygous deletions: identification of a novel heterozygous 8-kb intragenic deletion (IVS7-19 to IVS15-17) in a patient with glycogen storage disease ty

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Deletion of exon 18 is a frequent mutation in glycogen storage disease type II

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Aberrant splicing in adult onset glycogen storage disease type II (GSDII): molecular identification of an IVS1 (-13T-->G) mutation in a majority of patients and a novel IVS10 (+1GT-->CT) mutation

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Mutation profile of the GAA gene in 40 Italian patients with late onset glycogen storage disease type II

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Broad spectrum of Pompe disease in patients with the same c.-32-13T->G haplotype

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Frequent mutation in Chinese patients with infantile type of GSD II in Taiwan: evidence for a founder effect

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Evidence for a founder effect in Sicilian patients with glycogen storage disease type II

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

The effect of a single base pair deletion (delta T525) and a C1634T missense mutation (pro545leu) on the expression of lysosomal alpha-glucosidase in patients with glycogen storage disease type II.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

The African origin of the common mutation in African American patients with glycogen-storage disease type II.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Clinical diversity in glycogenosis type II. Biosynthesis and in situ localization of acid alpha-glucosidase in mutant fibroblasts

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Twenty-two novel mutations in the lysosomal ?-glucosidase gene (GAA) underscore the genotype-phenotype correlation in glycogen storage disease type II

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Modification of the natural history of adult-onset acid maltase deficiency by nutrition and exercise therapy

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Benign course of glycogen storage disease type IIb in two brothers: nature or nurture?

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Systemic correction of the muscle disorder glycogen storage disease type II after hepatic targeting of a modified adenovirus vector encoding human acid-alpha-glucosidase

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Physiological correction of Pompe disease by systemic delivery of adeno-associated virus serotype 1 vectors

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Multiple muscles in the AMD quail can be “cross‐corrected” of pathologic glycogen accumulation after intravenous injection of an [E1‐, polymerase‐] adenovirus vector encoding human acid‐α‐glucosidase

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Intercellular transfer of the virally derived precursor form of acid alpha-glucosidase corrects the enzyme deficiency in inherited cardioskeletal myopathy Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enhanced efficacy of an AAV vector encoding chimeric, highly secreted acid alpha-glucosidase in glycogen storage disease type II.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Impact of humoral immune response on distribution and efficacy of recombinant adeno-associated virus-derived acid alpha-glucosidase in a model of glycogen storage disease type II.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Chemical chaperones improve transport and enhance stability of mutant alpha-glucosidases in glycogen storage disease type II

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Pharmacological enhancement of mutated alpha-glucosidase activity in fibroblasts from patients with Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Targeted disruption of the acid alpha-glucosidase gene in mice causes an illness with critical features of both infantile and adult human glycogen storage disease type II

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Generalized glycogen storage and cardiomegaly in a knockout mouse model of Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

The role of autophagy during the early neonatal starvation period

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Autophagy and lysosomes in Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Pathological features of glycogen storage disease type II highlighted in the knockout mouse model

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Impaired performance of skeletal muscle in alpha-glucosidase knockout mice

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Morphological changes in muscle tissue of patients with infantile Pompe's disease receiving enzyme replacement therapy

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Dysfunction of endocytic and autophagic pathways in a lysosomal storage disease.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Characterization of pre- and post-treatment pathology after enzyme replacement therapy for Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Effects of non-contractile inclusions on mechanical performance of skeletal muscle

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Lysosomes in type II glycogenosis. Changes during administration of extract from Aspergillus niger

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Phosphohexosyl components of a lysosomal enzyme are recognized by pinocytosis receptors on human fibroblasts

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Hurler and Hunter syndromes: mutual correction of the defect in cultured fibroblasts

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Uptake and stability of human and bovine acid α-glucosidase in cultured fibroblasts and skeletal muscle cells from glycogenosis type II patients

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Receptor-mediated uptake of acid alpha-glucosidase corrects lysosomal glycogen storage in cultured skeletal muscle

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Rat heart perfusion as model system for enzyme replacement therapy in glycogenosis type II

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Intravenous administration of phosphorylated acid alpha-glucosidase leads to uptake of enzyme in heart and skeletal muscle of mice

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Clinical and metabolic correction of pompe disease by enzyme therapy in acid maltase-deficient quail

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Human acid alpha-glucosidase from rabbit milk has therapeutic effect in mice with glycogen storage disease type II

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

High-level production of recombinant human lysosomal acid alpha-glucosidase in Chinese hamster ovary cells which targets to heart muscle and corrects glycogen accumulation in fibroblasts from patients with Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme replacement therapy in the mouse model of Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Biodistribution, kinetics, and efficacy of highly phosphorylated and non-phosphorylated beta-glucuronidase in the murine model of mucopolysaccharidosis VII.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Therapeutic response to intravenous infusions of glucocerebrosidase in a patient with Gaucher disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Gaucher disease: lessons from a decade of therapy

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme therapy in type 1 Gaucher disease: comparative efficacy of mannose-terminated glucocerebrosidase from natural and recombinant sources

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Isolation and characterisation of a recombinant, precursor form of lysosomal acid alpha-glucosidase

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Recombinant human acid alpha-glucosidase: high level production in mouse milk, biochemical characteristics, correction of enzyme deficiency in GSDII KO mice

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Recombinant human alpha-glucosidase from rabbit milk in Pompe patients.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme replacement therapy in late-onset Pompe's disease: a three-year follow-up

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Safety and efficacy of recombinant acid alpha-glucosidase (rhGAA) in patients with classical infantile Pompe disease: results of a phase II clinical trial.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme replacement therapy in classical infantile pompe disease: results of a ten-month follow-up study

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Nephrotic syndrome complicating alpha-glucosidase replacement therapy for Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Recombinant human acid [alpha]-glucosidase: major clinical benefits in infantile-onset Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Anaesthetic management of infants with glycogen storage disease type II: a physiological approach.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Ambulatory electrocardiogram analysis in infants treated with recombinant human acid alpha-glucosidase enzyme replacement therapy for Pompe disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

A phase II/III clinical study of enzyme replacement therapy with idursulfase in mucopolysaccharidosis II (Hunter syndrome)

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Replacement therapy for inherited enzyme deficiency--macrophage-targeted glucocerebrosidase for Gaucher's disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Safety and efficacy of recombinant human alpha-galactosidase A replacement therapy in Fabry's disease

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme replacement therapy in Fabry disease: a randomized controlled trial

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme-replacement therapy in mucopolysaccharidosis I.

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme replacement therapy for mucopolysaccharidosis I: a randomized, double-blinded, placebo-controlled, multinational study of recombinant human alpha-L-iduronidase (laronidase).

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome).

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

Enzyme replacement therapy for mucopolysaccharidosis VI: A phase 3, randomized, double-blind, placebo-controlled, multinational study of recombinant human N-acetylgalactosamine 4-sulfatase (recombinant human arylsulfatase B or rhASB) and follow-on, o

1 reference

https://api.crossref.org/works/10.1016%2FS0140-6736%2808%2961555-X

7 January 2021

10.1016/S0140-6736(08)61555-X

Identifiers

DOI

1 reference

19 August 2017

1 reference

PubMed ID

18929906