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Chemically modified recombinant human sulfamidase (SOBI003) in mucopolysaccharidosis IIIA patients: Results from an open, non-controlled, multicenter study.
Harmatz, Paul; Muenzer, Joseph; Ezgü, Fatih; Dalén, Per; Huledal, Gunilla; Lindqvist, Daniel; Gelius, Stefan Svensson; Wikén, Margareta; Önnestam, Kristin; Bröijersén, Anders.
Affiliation
  • Harmatz P; UCSF Benioff Children's Hospital Oakland, Oakland, CA, USA. Electronic address: pharmatz@mail.cho.org.
  • Muenzer J; University of North Carolina at Chapel Hill, Chapel Hill, NC, USA.
  • Ezgü F; Gazi University Hospital, Ankara, Turkey.
  • Dalén P; Swedish Orphan Biovitrum AB, SE-112 76 Stockholm, Sweden.
  • Huledal G; Swedish Orphan Biovitrum AB, SE-112 76 Stockholm, Sweden.
  • Lindqvist D; Swedish Orphan Biovitrum AB, SE-112 76 Stockholm, Sweden.
  • Gelius SS; Swedish Orphan Biovitrum AB, SE-112 76 Stockholm, Sweden.
  • Wikén M; Swedish Orphan Biovitrum AB, SE-112 76 Stockholm, Sweden.
  • Önnestam K; Swedish Orphan Biovitrum AB, SE-112 76 Stockholm, Sweden.
  • Bröijersén A; Swedish Orphan Biovitrum AB, SE-112 76 Stockholm, Sweden.
Mol Genet Metab ; 136(4): 249-259, 2022 08.
Article in En | MEDLINE | ID: mdl-35835061
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Mucopolysaccharidosis III Type of study: Clinical_trials Aspects: Patient_preference Limits: Child / Child, preschool / Humans / Infant Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2022 Document type: Article Country of publication: United States
Fulltext
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Full text: 1 Collection: 01-internacional Database: MEDLINE Main subject: Mucopolysaccharidosis III Type of study: Clinical_trials Aspects: Patient_preference Limits: Child / Child, preschool / Humans / Infant Language: En Journal: Mol Genet Metab Journal subject: BIOLOGIA MOLECULAR / BIOQUIMICA / METABOLISMO Year: 2022 Document type: Article Country of publication: United States