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Diagnostic journey and impact of enzyme replacement therapy for mucopolysaccharidosis IVA: a sibling control study.
Ficicioglu, Can; Matalon, Dena R; Luongo, Nicole; Menello, Caitlin; Kornafel, Tracy; Degnan, Andrew J.
Affiliation
  • Ficicioglu C; Division of Human Genetics/Metabolism, Lysosomal Storage Diseases Program, The Children's Hospital of Philadelphia, Perelman School of Medicine, The University of Pennsylvania, 3401 Civic Center Blvd., Philadelphia, PA, 19104, USA. ficicioglu@email.chop.edu.
  • Matalon DR; Stanford University, Lucile Packard Children's Hospital, Palo Alto, CA, USA.
  • Luongo N; Division of Human Genetics/Metabolism, Lysosomal Storage Diseases Program, The Children's Hospital of Philadelphia, Perelman School of Medicine, The University of Pennsylvania, 3401 Civic Center Blvd., Philadelphia, PA, 19104, USA.
  • Menello C; Division of Human Genetics/Metabolism, Lysosomal Storage Diseases Program, The Children's Hospital of Philadelphia, Perelman School of Medicine, The University of Pennsylvania, 3401 Civic Center Blvd., Philadelphia, PA, 19104, USA.
  • Kornafel T; Division of Human Genetics/Metabolism, Lysosomal Storage Diseases Program, The Children's Hospital of Philadelphia, Perelman School of Medicine, The University of Pennsylvania, 3401 Civic Center Blvd., Philadelphia, PA, 19104, USA.
  • Degnan AJ; Abington Hospital - Jefferson Health, Abington, PA, USA.
Orphanet J Rare Dis ; 15(1): 336, 2020 11 30.
Article in En | MEDLINE | ID: mdl-33256811
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Full text: 1 Database: MEDLINE Main subject: Mucopolysaccharidosis IV / Siblings Type of study: Diagnostic_studies / Screening_studies Limits: Humans / Newborn Language: En Journal: Orphanet J Rare Dis Journal subject: MEDICINA Year: 2020 Type: Article Affiliation country: United States
Fulltext
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Full text: 1 Database: MEDLINE Main subject: Mucopolysaccharidosis IV / Siblings Type of study: Diagnostic_studies / Screening_studies Limits: Humans / Newborn Language: En Journal: Orphanet J Rare Dis Journal subject: MEDICINA Year: 2020 Type: Article Affiliation country: United States