P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry
| Main Authors: | , , , , , , , , , , |
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| Format: | Article |
| Language: | English |
| Published: |
Elsevier
2024-01-01
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| Series: | Genetics in Medicine Open |
| Online Access: | http://www.sciencedirect.com/science/article/pii/S2949774424000529 |
| _version_ | 1797267633811226624 |
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| author | Priya Kishnani David Stockton Andreas Hahn Juan Llerena Alexander Broomfield Julie Batista Meredith Foster Kathryn Wilson Susan Sparks Hannerieke van den Hout Yin-Hsiu Chien |
| author_facet | Priya Kishnani David Stockton Andreas Hahn Juan Llerena Alexander Broomfield Julie Batista Meredith Foster Kathryn Wilson Susan Sparks Hannerieke van den Hout Yin-Hsiu Chien |
| author_sort | Priya Kishnani |
| collection | DOAJ |
| first_indexed | 2024-04-25T01:19:42Z |
| format | Article |
| id | doaj.art-d259241bf6ca46faaee2ac9e829f486b |
| institution | Directory Open Access Journal |
| issn | 2949-7744 |
| language | English |
| last_indexed | 2024-04-25T01:19:42Z |
| publishDate | 2024-01-01 |
| publisher | Elsevier |
| record_format | Article |
| series | Genetics in Medicine Open |
| spelling | doaj.art-d259241bf6ca46faaee2ac9e829f486b2024-03-09T09:30:04ZengElsevierGenetics in Medicine Open2949-77442024-01-012100906P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe RegistryPriya Kishnani0David Stockton1Andreas Hahn2Juan Llerena3Alexander Broomfield4Julie Batista5Meredith Foster6Kathryn Wilson7Susan Sparks8Hannerieke van den Hout9Yin-Hsiu Chien10Division of Medical Genetics, Department of Pediatrics, Duke University Medical Center, Durham, NCDivision of Genetic, Genomic and Metabolic Disorders, Discipline of Pediatrics, Central Michigan University and Children's Hospital of Michigan, Detroit, MIDepartment of Child Neurology, University Hospital Giessen, Giessen, GermanyCentro de Genética Médica, Instituto Fernandes Figueira/FIOCRUZ, Rio de Janeiro, BrazilGreat Ormond Street Hospital, London, UKSanofi, Cambridge, MASanofi, Cambridge, MASanofi, Cambridge, MASanofi, Cambridge, MADivision of Metabolic Diseases and Genetics, Department of Pediatrics, Erasmus University Medical Center-Sophia, Rotterdam, The NetherlandsDepartment of Medical Genetics and Pediatrics, National Taiwan University Hospital, Taipei, Taiwanhttp://www.sciencedirect.com/science/article/pii/S2949774424000529 |
| spellingShingle | Priya Kishnani David Stockton Andreas Hahn Juan Llerena Alexander Broomfield Julie Batista Meredith Foster Kathryn Wilson Susan Sparks Hannerieke van den Hout Yin-Hsiu Chien P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry Genetics in Medicine Open |
| title | P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry |
| title_full | P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry |
| title_fullStr | P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry |
| title_full_unstemmed | P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry |
| title_short | P029: Early treatment with alglucosidase alfa is associated with improved survival in patients with infantile-onset Pompe disease: Data from Pompe Registry |
| title_sort | p029 early treatment with alglucosidase alfa is associated with improved survival in patients with infantile onset pompe disease data from pompe registry |
| url | http://www.sciencedirect.com/science/article/pii/S2949774424000529 |
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