(Q34545883)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

title

Therapies of mucopolysaccharidosis IVA (Morquio A syndrome) (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

author

Carlos J Alméciga-Díaz

2

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

author name string

Shunji Tomatsu

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Hector Barbosa

3

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Adriana M Montaño

4

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Luis A Barrera

5

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Tsutomu Shimada

6

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Eriko Yasuda

7

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

William G Mackenzie

8

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Robert W Mason

9

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Yasuyuki Suzuki

10

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Kenji E Orii

11

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

Tadao Orii

12

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

publication date

1 October 2013

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

Expert opinion on orphan drugs

23 February 2020

published in

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

volume

1

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

issue

10

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

page(s)

805-818

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

Current and emerging treatments and surgical interventions for Morquio A syndrome: a review

23 February 2020

cites work

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Bone-targeting endogenous secretory receptor for advanced glycation end products rescues rheumatoid arthritis

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

The effect of neonatal gene therapy on skeletal manifestations in mucopolysaccharidosis VII dogs after a decade

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Gene therapy for mucopolysaccharidosis type VI is effective in cats without pre-existing immunity to AAV8.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Correction of pathological accumulation of glycosaminoglycans in central nervous system and peripheral tissues of MPSIIIA mice through systemic AAV9 gene transfer

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Long circulating enzyme replacement therapy rescues bone pathology in mucopolysaccharidosis VII murine model

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Sialic acid deposition impairs the utility of AAV9, but not peptide-modified AAVs for brain gene therapy in a mouse model of lysosomal storage disease

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Effect of neonatal gene therapy on lumbar spine disease in mucopolysaccharidosis VII dogs

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Neonatal gene therapy with a gamma retroviral vector in mucopolysaccharidosis VI cats.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Clinical overview and treatment options for non-skeletal manifestations of mucopolysaccharidosis type IVA

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Gene therapy for lysosomal storage diseases: progress, challenges and future prospects

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Mucopolysaccharidosis type IVA (Morquio A disease): clinical review and current treatment

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Enzyme replacement therapy with galsulfase for mucopolysaccharidosis VI: clinical facts and figures

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Direct gene transfer to the CNS prevents emergence of neurologic disease in a murine model of mucopolysaccharidosis type I.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Correction of neurological disease of mucopolysaccharidosis IIIB in adult mice by rAAV9 trans-blood-brain barrier gene delivery

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Safe, efficient, and reproducible gene therapy of the brain in the dog models of Sanfilippo and Hurler syndromes.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Long-term amelioration of feline Mucopolysaccharidosis VI after AAV-mediated liver gene transfer

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Minicircle DNA-based gene therapy coupled with immune modulation permits long-term expression of α-L-iduronidase in mice with mucopolysaccharidosis type I

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Comparison of ventricular and intravenous lentiviral-mediated gene therapy for murine MPS VII.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Adeno-associated virus gene transfer in Morquio A disease - effect of promoters and sulfatase-modifying factor 1.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Correction of mucopolysaccharidosis type IIIA somatic and central nervous system pathology by lentiviral-mediated gene transfer.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Characterization of a novel mucopolysaccharidosis type II mouse model and recombinant AAV2/8 vector-mediated gene therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Enhancement of drug delivery: enzyme-replacement therapy for murine Morquio A syndrome.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Efficacy of recombinant human alpha-L-iduronidase (laronidase) on restricted range of motion of upper extremities in mucopolysaccharidosis type I patients

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Therapeutic efficacy of bone marrow transplant, intracranial AAV-mediated gene therapy, or both in the mouse model of MPS IIIB

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

A self-inactivating gamma-retroviral vector reduces manifestations of mucopolysaccharidosis I in mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Long-term AAV vector gene and protein expression in mouse brain from a small pan-cellular promoter is similar to neural cell promoters

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Systemic correction of storage disease in MPS I NOD/SCID mice using the sleeping beauty transposon system

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Effect of elongation factor 1alpha promoter and SUMF1 over in vitro expression of N-acetylgalactosamine-6-sulfate sulfatase

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Immune tolerance improves the efficacy of enzyme replacement therapy in canine mucopolysaccharidosis I.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Targeted drug delivery to bone: pharmacokinetic and pharmacological properties of acidic oligopeptide-tagged drugs

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Targeting of the CNS in MPS-IH using a nonviral transferrin-alpha-L-iduronidase fusion gene product

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Acidic amino acid tag enhances response to enzyme replacement in mucopolysaccharidosis type VII mice.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Enzyme replacement therapy for murine hypophosphatasia

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Treatment of lysosomal storage disorders : progress with enzyme replacement therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Engineering adeno-associated virus 2 vectors for targeted gene delivery to atherosclerotic lesions.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Gene therapy for mucopolysaccharidosis

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Correction of clinical manifestations of canine mucopolysaccharidosis I with neonatal retroviral vector gene therapy

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Nanomedicine: clinical applications of polyethylene glycol conjugated proteins and drugs

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

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

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

A systematic review of the clinical effectiveness and cost-effectiveness of enzyme replacement therapies for Fabry's disease and mucopolysaccharidosis type 1.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Enhancement of drug delivery to bone: characterization of human tissue-nonspecific alkaline phosphatase tagged with an acidic oligopeptide

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Cell and gene-based therapies for the lysosomal storage diseases

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

AAV hybrid serotypes: improved vectors for gene delivery

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Characterizing biological products and assessing comparability following manufacturing changes.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Gene therapy for lysosomal storage diseases: the lessons and promise of animal models

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Promoters and control elements: designing expression cassettes for gene therapy.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Receptor targeting of adeno-associated virus vectors

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

The multiple sulfatase deficiency gene encodes an essential and limiting factor for the activity of sulfatases

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Multiple sulfatase deficiency is caused by mutations in the gene encoding the human C(alpha)-formylglycine generating enzyme

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Enzyme replacement therapy in mucopolysaccharidosis type II (Hunter syndrome): a preliminary report

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Selective delivery of estradiol to bone by aspartic acid oligopeptide and its effects on ovariectomized mice

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Selective drug delivery system to bone: small peptide (Asp)6 conjugation

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Enzyme replacement therapy from birth in a feline model of mucopolysaccharidosis type VI.

7 July 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Mouse model of N-acetylgalactosamine-6-sulfate sulfatase deficiency (Galns-/-) produced by targeted disruption of the gene defective in Morquio A disease

7 July 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Development of MPS IVA mouse (Galnstm(hC79S.mC76S)slu) tolerant to human N-acetylgalactosamine-6-sulfate sulfatase

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Murine model (Galns(tm(C76S)slu)) of MPS IVA with missense mutation at the active site cysteine conserved among sulfatase proteins.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

International Morquio A Registry: clinical manifestation and natural course of Morquio A disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Deficiency in N-acetylgalactosamine-6-sulfate sulfatase results in collagen perturbations in cartilage of Morquio syndrome A patients.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Pathogenesis of Morquio A syndrome: an autopsied case reveals systemic storage disorder.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Enzyme-replacement therapy in mucopolysaccharidosis I.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Direct comparison of measures of endurance, mobility, and joint function during enzyme-replacement therapy of mucopolysaccharidosis VI (Maroteaux-Lamy syndrome): results after 48 weeks in a phase 2 open-label clinical study of recombinant human N-ac

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

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

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Enzyme replacement therapy in a murine model of Morquio A syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Long-term follow-up following bone marrow transplantation for Hunter disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Anesthetic care and perioperative complications of children with Morquio syndrome.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Joint and bone disease in mucopolysaccharidoses VI and VII: identification of new therapeutic targets and biomarkers using animal models

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Europe gives gene therapy the green light

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Gene therapy of Hunter syndrome: evaluation of the efficiency of muscle electro gene transfer for the production and release of recombinant iduronate-2-sulfatase (IDS).

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Significantly increased lifespan and improved behavioral performances by rAAV gene delivery in adult mucopolysaccharidosis IIIB mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Restoration of central nervous system alpha-N-acetylglucosaminidase activity and therapeutic benefits in mucopolysaccharidosis IIIB mice by a single intracisternal recombinant adeno-associated viral type 2 vector delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Lentiviral-mediated gene therapy for murine mucopolysaccharidosis type IIIA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

SGSH gene transfer in mucopolysaccharidosis type IIIA mice using canine adenovirus vectors.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Helper-dependent canine adenovirus vector-mediated transgene expression in a neurodegenerative lysosomal storage disorder

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Worldwide epidemiology of neutralizing antibodies to adeno-associated viruses.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Lentiviral-mediated correction of MPS VI cells and gene transfer to joint tissues.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Widespread biochemical correction of murine mucopolysaccharidosis type VII pathology by liver hydrodynamic plasmid delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Skeletal response to lentiviral mediated gene therapy in a mouse model of MPS VII.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Various cells retrovirally transduced with N-acetylgalactosoamine-6-sulfate sulfatase correct Morquio skin fibroblasts in vitro

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Targeting recombinant adeno-associated virus vectors to enhance gene transfer to pancreatic islets and liver

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Improvements in mucopolysaccharidosis I mice after adult retroviral vector-mediated gene therapy with immunomodulation.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Disease correction by combined neonatal intracranial AAV and systemic lentiviral gene therapy in Sanfilippo Syndrome type B mice.

21 January 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Enzyme-replacement therapy in life-threatening hypophosphatasia.

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

A molecular and histological characterization of cartilage from patients with Morquio syndrome

27 October 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4238063

Sacral dimple: incidental findings from newborn evaluation. Mucopolysaccharidosis IVA disease

27 October 2018

1 reference

https://api.crossref.org/works/10.1517%2F21678707.2013.846853

Fetal presentation of Morquio disease type A

21 January 2018

1 reference

12 December 2020

Limb-saving medicines sought to prevent amputations

1 reference

12 December 2020

Enzyme replacement therapy for mucopolysaccharidosis II from 3 months of age: a 3-year follow-up

1 reference

12 December 2020

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

12 December 2020

[Construction of an adenoassociated, viral derived, expression vector to correct the genetic defect in Morquio A disease]

1 reference

12 December 2020

Intracranial gene delivery of LV-NAGLU vector corrects neuropathology in murine MPS IIIB

1 reference

12 December 2020

10.1517/21678707.2013.846853

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json

23 February 2020

PubMed publication ID

25419501

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:25419501%20AND%20SRC:MED&resulttype=core&format=json