Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

Enzyme replacement therapy outcomes across the disease spectrum: Findings from the mucopolysaccharidosis VI Clinical Surveillance Program

The impact of galsulfase enzyme replacement therapy in patients with mucopolysaccharidosis (MPS) VI with phenotypes at either end of the disease spectrum was evaluated. The MPS VI Clinical Surveillance Program (CSP) was established to collect long‐term observational data from routine clinical and la...

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Veröffentlicht in:Journal of inherited metabolic disease 2019-05, Vol.42 (3), p.519-526
Hauptverfasser: Harmatz, Paul R., Lampe, Christina, Parini, Rossella, Sharma, Reena, Teles, Elisa L., Johnson, Julie, Sivam, Debbie, Sisic, Zlatko
Format: Artikel
Sprache:eng
Schlagworte:
Cardiac function
Creatinine
disease severity, urinary GAG
efficacy
enzyme replacement therapy
Enzymes
galsulfase
Genetic disorders
Heart
Maroteaux-Lamy syndrome
Metabolic disorders
Mucopolysaccharidosis
mucopolysaccharidosis VI
Patients
Phenotypes
registry, safety
Respiratory function
Surveillance
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Zusammenfassung:The impact of galsulfase enzyme replacement therapy in patients with mucopolysaccharidosis (MPS) VI with phenotypes at either end of the disease spectrum was evaluated. The MPS VI Clinical Surveillance Program (CSP) was established to collect long‐term observational data from routine clinical and laboratory assessments. A subanalysis of the CSP was performed in patients with pretreatment urinary glycosaminoglycan (uGAG) levels
ISSN:0141-8955
1573-2665
DOI:10.1002/jimd.12079