Niemann-Pick B - RS - 5th infusion.
Por um escritor misterioso
Descrição
Interstitial lung disease in lysosomal storage disorders
Pediatric hepatocellular carcinoma associated with Niemann–Pick disease type C: Case report and literature review - Hwang - 2023 - JIMD Reports - Wiley Online Library
Natural history and management of liver dysfunction in lysosomal storage disorders
Advances in therapies for neurological lysosomal storage disorders - Ellison - 2023 - Journal of Inherited Metabolic Disease - Wiley Online Library
Niemann-Pick disease type C
Pediatric hepatocellular carcinoma associated with Niemann–Pick disease type C: Case report and literature review - Hwang - 2023 - JIMD Reports - Wiley Online Library
Expanded access with intravenous hydroxypropyl-β-cyclodextrin to treat children and young adults with Niemann-Pick disease type C1: a case report analysis, Orphanet Journal of Rare Diseases
Lysosomal storage diseases: Diagnostic confirmation and management of presymptomatic individuals - Genetics in Medicine
WO2022026410A2 - Compositions and methods for the treatment of niemann-pick type c1 disease - Google Patents
Olipudase alfa enzyme replacement therapy for acid sphingomyelinase deficiency (ASMD): sustained improvements in clinical outcomes after 6.5 years of treatment in adults, Orphanet Journal of Rare Diseases
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por adulto (o preço varia de acordo com o tamanho do grupo)
