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<record><TEI><teiHeader><fileDesc><titleStmt><title xml:lang="en">Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human <italic>N</italic>-acetylgalactosamine 4-sulfatase</title><author><name sortKey="Decker, Celeste" sort="Decker, Celeste" uniqKey="Decker C" first="Celeste" last="Decker">Celeste Decker</name><affiliation><nlm:aff id="A1">BioMarin Pharmaceutical Inc, Novato, CA, USA</nlm:aff></affiliation></author><author><name sortKey="Yu, Zi Fan" sort="Yu, Zi Fan" uniqKey="Yu Z" first="Zi-Fan" last="Yu">Zi-Fan Yu</name><affiliation><nlm:aff id="A2">Statistics Collaborative, Inc., Washington, DC, USA</nlm:aff></affiliation></author><author><name sortKey="Giugliani, Roberto" sort="Giugliani, Roberto" uniqKey="Giugliani R" first="Roberto" last="Giugliani">Roberto Giugliani</name><affiliation><nlm:aff id="A3">Serviço de Genética Médica/ HCPA and Department of Genetics/UFRGS, Porto Alegre, Brazil</nlm:aff></affiliation></author><author><name sortKey="Schwartz, Ida Vanessa D" sort="Schwartz, Ida Vanessa D" uniqKey="Schwartz I" first="Ida Vanessa D." last="Schwartz">Ida Vanessa D. Schwartz</name><affiliation><nlm:aff id="A3">Serviço de Genética Médica/ HCPA and Department of Genetics/UFRGS, Porto Alegre, Brazil</nlm:aff></affiliation></author><author><name sortKey="Guffon, Nathalie" sort="Guffon, Nathalie" uniqKey="Guffon N" first="Nathalie" last="Guffon">Nathalie Guffon</name><affiliation><nlm:aff id="A4">Hôpital Femme Mère Enfant, Service Maladies Métaboliques, BRON, France</nlm:aff></affiliation></author><author><name sortKey="Teles, Elisa Leao" sort="Teles, Elisa Leao" uniqKey="Teles E" first="Elisa Leão" last="Teles">Elisa Leão Teles</name><affiliation><nlm:aff id="A5">Unidade de Doenças Metabólicas, Departamento Pediatria, Hospital de Sao João, Porto, Portugal</nlm:aff></affiliation></author><author><name sortKey="Miranda, M Clara Sa" sort="Miranda, M Clara Sa" uniqKey="Miranda M" first="M. Clara Sá" last="Miranda">M. Clara Sá Miranda</name><affiliation><nlm:aff id="A6">Unidade de Biologia do Lisossoma e Peroxisoma, Instituto de Biologia Molecular e Celular, Porto, Portugal</nlm:aff></affiliation></author><author><name sortKey="Wraith, J Edmond" sort="Wraith, J Edmond" uniqKey="Wraith J" first="J. Edmond" last="Wraith">J. Edmond Wraith</name><affiliation><nlm:aff id="A7">Royal Manchester Children’s Hospital, Manchester, UK</nlm:aff></affiliation></author><author><name sortKey="Beck, Michael" sort="Beck, Michael" uniqKey="Beck M" first="Michael" last="Beck">Michael Beck</name><affiliation><nlm:aff id="A8">Children’s Hospital, University of Mainz, Germany</nlm:aff></affiliation></author><author><name sortKey="Arash, Laila" sort="Arash, Laila" uniqKey="Arash L" first="Laila" last="Arash">Laila Arash</name><affiliation><nlm:aff id="A8">Children’s Hospital, University of Mainz, Germany</nlm:aff></affiliation></author><author><name sortKey="Scarpa, Maurizio" sort="Scarpa, Maurizio" uniqKey="Scarpa M" first="Maurizio" last="Scarpa">Maurizio Scarpa</name><affiliation><nlm:aff id="A9">Department of Pediatrics, University of Padova, Padova, Italy</nlm:aff></affiliation></author><author><name sortKey="Ketteridge, David" sort="Ketteridge, David" uniqKey="Ketteridge D" first="David" last="Ketteridge">David Ketteridge</name><affiliation><nlm:aff id="A10">SA Pathology at Women’s and Children’s Hospital, North Adelaide, Australia</nlm:aff></affiliation></author><author><name sortKey="Hopwood, John J" sort="Hopwood, John J" uniqKey="Hopwood J" first="John J." last="Hopwood">John J. Hopwood</name><affiliation><nlm:aff id="A11">Department of Genetic Medicine, Women’s and Children’s Hospital Adelaide, North Adelaide, Australia</nlm:aff></affiliation></author><author><name sortKey="Plecko, Barbara" sort="Plecko, Barbara" uniqKey="Plecko B" first="Barbara" last="Plecko">Barbara Plecko</name><affiliation><nlm:aff id="A12">University Klinik für Kinder und Jugendheilkunde, Graz, Austria</nlm:aff></affiliation></author><author><name sortKey="Steiner, Robert" sort="Steiner, Robert" uniqKey="Steiner R" first="Robert" last="Steiner">Robert Steiner</name><affiliation><nlm:aff id="A13">Departments of Pediatrics and Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR, USA</nlm:aff></affiliation></author><author><name sortKey="Whitley, Chester B" sort="Whitley, Chester B" uniqKey="Whitley C" first="Chester B." last="Whitley">Chester B. Whitley</name><affiliation><nlm:aff id="A14">University of Minnesota Medical School, Minneapolis, MN, USA</nlm:aff></affiliation></author><author><name sortKey="Kaplan, Paige" sort="Kaplan, Paige" uniqKey="Kaplan P" first="Paige" last="Kaplan">Paige Kaplan</name><affiliation><nlm:aff id="A15">Children’s Hospital of Philadelphia, Philadelphia, PA, USA</nlm:aff></affiliation></author><author><name sortKey="Swiedler, Stuart J" sort="Swiedler, Stuart J" uniqKey="Swiedler S" first="Stuart J." last="Swiedler">Stuart J. Swiedler</name><affiliation><nlm:aff id="A1">BioMarin Pharmaceutical Inc, Novato, CA, USA</nlm:aff></affiliation></author><author><name sortKey="Conrad, Susan" sort="Conrad, Susan" uniqKey="Conrad S" first="Susan" last="Conrad">Susan Conrad</name><affiliation><nlm:aff id="A16">Children’s Hospital & Research Center Oakland, Oakland, CA, USA</nlm:aff></affiliation></author><author><name sortKey="Harmatz, Paul" sort="Harmatz, Paul" uniqKey="Harmatz P" first="Paul" last="Harmatz">Paul Harmatz</name><affiliation><nlm:aff id="A16">Children’s Hospital & Research Center Oakland, Oakland, CA, USA</nlm:aff></affiliation></author></titleStmt><publicationStmt><idno type="wicri:source">PMC</idno><idno type="pmid">20634905</idno><idno type="pmc">2904323</idno><idno type="url">http://www.ncbi.nlm.nih.gov/pmc/articles/PMC2904323</idno><idno type="RBID">PMC:2904323</idno><idno type="doi">10.3233/PRM-2010-0113</idno><date when="2010">2010</date><idno type="wicri:Area/Pmc/Corpus">001438</idno><idno type="wicri:explorRef" wicri:stream="Pmc" wicri:step="Corpus" wicri:corpus="PMC">001438</idno></publicationStmt><sourceDesc><biblStruct><analytic><title xml:lang="en" level="a" type="main">Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human <italic>N</italic>-acetylgalactosamine 4-sulfatase</title><author><name sortKey="Decker, Celeste" sort="Decker, Celeste" uniqKey="Decker C" first="Celeste" last="Decker">Celeste Decker</name><affiliation><nlm:aff id="A1">BioMarin Pharmaceutical Inc, Novato, CA, USA</nlm:aff></affiliation></author><author><name sortKey="Yu, Zi Fan" sort="Yu, Zi Fan" uniqKey="Yu Z" first="Zi-Fan" last="Yu">Zi-Fan Yu</name><affiliation><nlm:aff id="A2">Statistics Collaborative, Inc., Washington, DC, USA</nlm:aff></affiliation></author><author><name sortKey="Giugliani, Roberto" sort="Giugliani, Roberto" uniqKey="Giugliani R" first="Roberto" last="Giugliani">Roberto Giugliani</name><affiliation><nlm:aff id="A3">Serviço de Genética Médica/ HCPA and Department of Genetics/UFRGS, Porto Alegre, Brazil</nlm:aff></affiliation></author><author><name sortKey="Schwartz, Ida Vanessa D" sort="Schwartz, Ida Vanessa D" uniqKey="Schwartz I" first="Ida Vanessa D." last="Schwartz">Ida Vanessa D. Schwartz</name><affiliation><nlm:aff id="A3">Serviço de Genética Médica/ HCPA and Department of Genetics/UFRGS, Porto Alegre, Brazil</nlm:aff></affiliation></author><author><name sortKey="Guffon, Nathalie" sort="Guffon, Nathalie" uniqKey="Guffon N" first="Nathalie" last="Guffon">Nathalie Guffon</name><affiliation><nlm:aff id="A4">Hôpital Femme Mère Enfant, Service Maladies Métaboliques, BRON, France</nlm:aff></affiliation></author><author><name sortKey="Teles, Elisa Leao" sort="Teles, Elisa Leao" uniqKey="Teles E" first="Elisa Leão" last="Teles">Elisa Leão Teles</name><affiliation><nlm:aff id="A5">Unidade de Doenças Metabólicas, Departamento Pediatria, Hospital de Sao João, Porto, Portugal</nlm:aff></affiliation></author><author><name sortKey="Miranda, M Clara Sa" sort="Miranda, M Clara Sa" uniqKey="Miranda M" first="M. Clara Sá" last="Miranda">M. Clara Sá Miranda</name><affiliation><nlm:aff id="A6">Unidade de Biologia do Lisossoma e Peroxisoma, Instituto de Biologia Molecular e Celular, Porto, Portugal</nlm:aff></affiliation></author><author><name sortKey="Wraith, J Edmond" sort="Wraith, J Edmond" uniqKey="Wraith J" first="J. Edmond" last="Wraith">J. Edmond Wraith</name><affiliation><nlm:aff id="A7">Royal Manchester Children’s Hospital, Manchester, UK</nlm:aff></affiliation></author><author><name sortKey="Beck, Michael" sort="Beck, Michael" uniqKey="Beck M" first="Michael" last="Beck">Michael Beck</name><affiliation><nlm:aff id="A8">Children’s Hospital, University of Mainz, Germany</nlm:aff></affiliation></author><author><name sortKey="Arash, Laila" sort="Arash, Laila" uniqKey="Arash L" first="Laila" last="Arash">Laila Arash</name><affiliation><nlm:aff id="A8">Children’s Hospital, University of Mainz, Germany</nlm:aff></affiliation></author><author><name sortKey="Scarpa, Maurizio" sort="Scarpa, Maurizio" uniqKey="Scarpa M" first="Maurizio" last="Scarpa">Maurizio Scarpa</name><affiliation><nlm:aff id="A9">Department of Pediatrics, University of Padova, Padova, Italy</nlm:aff></affiliation></author><author><name sortKey="Ketteridge, David" sort="Ketteridge, David" uniqKey="Ketteridge D" first="David" last="Ketteridge">David Ketteridge</name><affiliation><nlm:aff id="A10">SA Pathology at Women’s and Children’s Hospital, North Adelaide, Australia</nlm:aff></affiliation></author><author><name sortKey="Hopwood, John J" sort="Hopwood, John J" uniqKey="Hopwood J" first="John J." last="Hopwood">John J. Hopwood</name><affiliation><nlm:aff id="A11">Department of Genetic Medicine, Women’s and Children’s Hospital Adelaide, North Adelaide, Australia</nlm:aff></affiliation></author><author><name sortKey="Plecko, Barbara" sort="Plecko, Barbara" uniqKey="Plecko B" first="Barbara" last="Plecko">Barbara Plecko</name><affiliation><nlm:aff id="A12">University Klinik für Kinder und Jugendheilkunde, Graz, Austria</nlm:aff></affiliation></author><author><name sortKey="Steiner, Robert" sort="Steiner, Robert" uniqKey="Steiner R" first="Robert" last="Steiner">Robert Steiner</name><affiliation><nlm:aff id="A13">Departments of Pediatrics and Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR, USA</nlm:aff></affiliation></author><author><name sortKey="Whitley, Chester B" sort="Whitley, Chester B" uniqKey="Whitley C" first="Chester B." last="Whitley">Chester B. Whitley</name><affiliation><nlm:aff id="A14">University of Minnesota Medical School, Minneapolis, MN, USA</nlm:aff></affiliation></author><author><name sortKey="Kaplan, Paige" sort="Kaplan, Paige" uniqKey="Kaplan P" first="Paige" last="Kaplan">Paige Kaplan</name><affiliation><nlm:aff id="A15">Children’s Hospital of Philadelphia, Philadelphia, PA, USA</nlm:aff></affiliation></author><author><name sortKey="Swiedler, Stuart J" sort="Swiedler, Stuart J" uniqKey="Swiedler S" first="Stuart J." last="Swiedler">Stuart J. Swiedler</name><affiliation><nlm:aff id="A1">BioMarin Pharmaceutical Inc, Novato, CA, USA</nlm:aff></affiliation></author><author><name sortKey="Conrad, Susan" sort="Conrad, Susan" uniqKey="Conrad S" first="Susan" last="Conrad">Susan Conrad</name><affiliation><nlm:aff id="A16">Children’s Hospital & Research Center Oakland, Oakland, CA, USA</nlm:aff></affiliation></author><author><name sortKey="Harmatz, Paul" sort="Harmatz, Paul" uniqKey="Harmatz P" first="Paul" last="Harmatz">Paul Harmatz</name><affiliation><nlm:aff id="A16">Children’s Hospital & Research Center Oakland, Oakland, CA, USA</nlm:aff></affiliation></author></analytic><series><title level="j">Journal of pediatric rehabilitation medicine</title><idno type="ISSN">1874-5393</idno><idno type="eISSN">1875-8894</idno><imprint><date when="2010">2010</date></imprint></series></biblStruct></sourceDesc></fileDesc><profileDesc><textClass></textClass></profileDesc></teiHeader><front><div type="abstract" xml:lang="en"><sec id="S1"><title>Background and Methods</title><p id="P1">Growth failure is characteristic of untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to and for up to 240 weeks of weekly infusions of recombinant human arylsulfatase B (rhASB) at 1 mg/kg during Phase 1/2, Phase 2, Phase 3 or Phase 3 Extension clinical trials. Height, weight, and Tanner stage data were collected. Pooled data were analyzed to determine mean height increase by treatment week, growth impacts of pubertal status, baseline urinary GAG, and age at treatment initiation. Growth rate for approximately 2 years prior to and following treatment initiation was analyzed using longitudinal modeling.</p></sec><sec id="S2"><title>Results</title><p id="P2">Mean height increased by 2.9 cm after 48 weeks and 4.3 cm after 96 weeks on enzyme replacement therapy (ERT). Growth on ERT was not correlated with baseline urinary GAG. Patients under 16 years of age showed greatest increases in height on treatment. Model results based on pooled data showed significant improvement in growth rate during 96 weeks of ERT when compared to the equivalent pretreatment time period. Delayed pubertal onset or progression was noted in 10 patients entering the clinical trials; all of whom showed progression of at least one Tanner stage during 2 years on ERT, and 6 of whom (60%) completed puberty.</p></sec><sec id="S3"><title>Conclusion</title><p id="P3">Analysis of mean height by treatment week and longitudinal modeling demonstrate significant increase in height and growth rate in MPS VI patients receiving long-term ERT. This impact was greatest in patients aged below 16 years. Height increase may result from bone growth and/or reduction in joint contractures. Bone growth and resolution of delayed puberty may be related to improvements in general health, bone cell health, nutrition, endocrine gland function and reduced inflammation.</p></sec></div></front></TEI><pmc article-type="research-article" xml:lang="EN"><pmc-comment>The publisher of this article does not allow downloading of the full text in XML form.</pmc-comment>
<pmc-dir>properties manuscript</pmc-dir>
<front><journal-meta><journal-id journal-id-type="nlm-journal-id">101490944</journal-id><journal-id journal-id-type="pubmed-jr-id">36349</journal-id><journal-id journal-id-type="nlm-ta">J Pediatr Rehabil Med</journal-id><journal-title>Journal of pediatric rehabilitation medicine</journal-title><issn pub-type="ppub">1874-5393</issn><issn pub-type="epub">1875-8894</issn></journal-meta><article-meta><article-id pub-id-type="pmid">20634905</article-id><article-id pub-id-type="pmc">2904323</article-id><article-id pub-id-type="doi">10.3233/PRM-2010-0113</article-id><article-id pub-id-type="manuscript">NIHMS214597</article-id><article-categories><subj-group subj-group-type="heading"><subject>Article</subject></subj-group></article-categories><title-group><article-title>Enzyme replacement therapy for mucopolysaccharidosis VI: Growth and pubertal development in patients treated with recombinant human <italic>N</italic>-acetylgalactosamine 4-sulfatase</article-title></title-group><contrib-group><contrib contrib-type="author"><name><surname>Decker</surname><given-names>Celeste</given-names></name><xref ref-type="aff" rid="A1">a</xref></contrib><contrib contrib-type="author"><name><surname>Yu</surname><given-names>Zi-Fan</given-names></name><xref ref-type="aff" rid="A2">b</xref></contrib><contrib contrib-type="author"><name><surname>Giugliani</surname><given-names>Roberto</given-names></name><xref ref-type="aff" rid="A3">c</xref></contrib><contrib contrib-type="author"><name><surname>Schwartz</surname><given-names>Ida Vanessa D.</given-names></name><xref ref-type="aff" rid="A3">c</xref></contrib><contrib contrib-type="author"><name><surname>Guffon</surname><given-names>Nathalie</given-names></name><xref ref-type="aff" rid="A4">d</xref></contrib><contrib contrib-type="author"><name><surname>Teles</surname><given-names>Elisa Leão</given-names></name><xref ref-type="aff" rid="A5">e</xref></contrib><contrib contrib-type="author"><name><surname>Miranda</surname><given-names>M. Clara Sá</given-names></name><xref ref-type="aff" rid="A6">f</xref></contrib><contrib contrib-type="author"><name><surname>Wraith</surname><given-names>J. Edmond</given-names></name><xref ref-type="aff" rid="A7">g</xref></contrib><contrib contrib-type="author"><name><surname>Beck</surname><given-names>Michael</given-names></name><xref ref-type="aff" rid="A8">h</xref></contrib><contrib contrib-type="author"><name><surname>Arash</surname><given-names>Laila</given-names></name><xref ref-type="aff" rid="A8">h</xref></contrib><contrib contrib-type="author"><name><surname>Scarpa</surname><given-names>Maurizio</given-names></name><xref ref-type="aff" rid="A9">i</xref></contrib><contrib contrib-type="author"><name><surname>Ketteridge</surname><given-names>David</given-names></name><xref ref-type="aff" rid="A10">j</xref></contrib><contrib contrib-type="author"><name><surname>Hopwood</surname><given-names>John J.</given-names></name><xref ref-type="aff" rid="A11">k</xref></contrib><contrib contrib-type="author"><name><surname>Plecko</surname><given-names>Barbara</given-names></name><xref ref-type="aff" rid="A12">l</xref></contrib><contrib contrib-type="author"><name><surname>Steiner</surname><given-names>Robert</given-names></name><xref ref-type="aff" rid="A13">m</xref></contrib><contrib contrib-type="author"><name><surname>Whitley</surname><given-names>Chester B.</given-names></name><xref ref-type="aff" rid="A14">n</xref></contrib><contrib contrib-type="author"><name><surname>Kaplan</surname><given-names>Paige</given-names></name><xref ref-type="aff" rid="A15">o</xref></contrib><contrib contrib-type="author"><name><surname>Swiedler</surname><given-names>Stuart J.</given-names></name><xref ref-type="aff" rid="A1">a</xref></contrib><contrib contrib-type="author"><name><surname>Conrad</surname><given-names>Susan</given-names></name><xref ref-type="aff" rid="A16">p</xref></contrib><contrib contrib-type="author"><name><surname>Harmatz</surname><given-names>Paul</given-names></name><xref ref-type="aff" rid="A16">p</xref><xref ref-type="corresp" rid="cor1">*</xref></contrib><on-behalf-of>for the MPS VI Study Group</on-behalf-of><xref ref-type="fn" rid="FN1">**</xref></contrib-group><aff id="A1"><label>a</label>BioMarin Pharmaceutical Inc, Novato, CA, USA</aff><aff id="A2"><label>b</label>Statistics Collaborative, Inc., Washington, DC, USA</aff><aff id="A3"><label>c</label>Serviço de Genética Médica/ HCPA and Department of Genetics/UFRGS, Porto Alegre, Brazil</aff><aff id="A4"><label>d</label>Hôpital Femme Mère Enfant, Service Maladies Métaboliques, BRON, France</aff><aff id="A5"><label>e</label>Unidade de Doenças Metabólicas, Departamento Pediatria, Hospital de Sao João, Porto, Portugal</aff><aff id="A6"><label>f</label>Unidade de Biologia do Lisossoma e Peroxisoma, Instituto de Biologia Molecular e Celular, Porto, Portugal</aff><aff id="A7"><label>g</label>Royal Manchester Children’s Hospital, Manchester, UK</aff><aff id="A8"><label>h</label>Children’s Hospital, University of Mainz, Germany</aff><aff id="A9"><label>i</label>Department of Pediatrics, University of Padova, Padova, Italy</aff><aff id="A10"><label>j</label>SA Pathology at Women’s and Children’s Hospital, North Adelaide, Australia</aff><aff id="A11"><label>k</label>Department of Genetic Medicine, Women’s and Children’s Hospital Adelaide, North Adelaide, Australia</aff><aff id="A12"><label>l</label>University Klinik für Kinder und Jugendheilkunde, Graz, Austria</aff><aff id="A13"><label>m</label>Departments of Pediatrics and Molecular and Medical Genetics, Oregon Health & Science University, Portland, OR, USA</aff><aff id="A14"><label>n</label>University of Minnesota Medical School, Minneapolis, MN, USA</aff><aff id="A15"><label>o</label>Children’s Hospital of Philadelphia, Philadelphia, PA, USA</aff><aff id="A16"><label>p</label>Children’s Hospital & Research Center Oakland, Oakland, CA, USA</aff><author-notes><corresp id="cor1"><label>*</label>Address for correspondence: Paul Harmatz, MD, Children’s Hospital & Research Center Oakland, 747 52nd Street, Oakland, CA 94609, USA. Tel.: +1 510 428 3058; Fax: +1 510 450 5813; <email>Pharmatz@mail.cho.org</email></corresp></author-notes><pub-date pub-type="nihms-submitted"><day>29</day><month>6</month><year>2010</year></pub-date><pub-date pub-type="ppub"><year>2010</year></pub-date><pub-date pub-type="pmc-release"><day>14</day><month>7</month><year>2010</year></pub-date><volume>3</volume><issue>2</issue><fpage>89</fpage><lpage>100</lpage><permissions><copyright-statement>© 2010 – IOS Press and the authors. All rights reserved</copyright-statement><copyright-year>2010</copyright-year></permissions><abstract><sec id="S1"><title>Background and Methods</title><p id="P1">Growth failure is characteristic of untreated mucopolysaccharidosis type VI (MPS VI: Maroteaux-Lamy syndrome). Growth was studied in fifty-six MPS VI patients (5 to 29 years old) prior to and for up to 240 weeks of weekly infusions of recombinant human arylsulfatase B (rhASB) at 1 mg/kg during Phase 1/2, Phase 2, Phase 3 or Phase 3 Extension clinical trials. Height, weight, and Tanner stage data were collected. Pooled data were analyzed to determine mean height increase by treatment week, growth impacts of pubertal status, baseline urinary GAG, and age at treatment initiation. Growth rate for approximately 2 years prior to and following treatment initiation was analyzed using longitudinal modeling.</p></sec><sec id="S2"><title>Results</title><p id="P2">Mean height increased by 2.9 cm after 48 weeks and 4.3 cm after 96 weeks on enzyme replacement therapy (ERT). Growth on ERT was not correlated with baseline urinary GAG. Patients under 16 years of age showed greatest increases in height on treatment. Model results based on pooled data showed significant improvement in growth rate during 96 weeks of ERT when compared to the equivalent pretreatment time period. Delayed pubertal onset or progression was noted in 10 patients entering the clinical trials; all of whom showed progression of at least one Tanner stage during 2 years on ERT, and 6 of whom (60%) completed puberty.</p></sec><sec id="S3"><title>Conclusion</title><p id="P3">Analysis of mean height by treatment week and longitudinal modeling demonstrate significant increase in height and growth rate in MPS VI patients receiving long-term ERT. This impact was greatest in patients aged below 16 years. Height increase may result from bone growth and/or reduction in joint contractures. Bone growth and resolution of delayed puberty may be related to improvements in general health, bone cell health, nutrition, endocrine gland function and reduced inflammation.</p></sec></abstract><kwd-group><kwd>Mucopolysaccharidosis VI</kwd><kwd><italic>N</italic>-acetylgalactosamine 4-sulfatase</kwd><kwd>arylsulfatase B</kwd><kwd>enzyme replacement therapy</kwd><kwd>glycosaminoglycans</kwd><kwd>growth</kwd><kwd>puberty</kwd></kwd-group><contract-num rid="RR1">M01 RR001271-21
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