标题
Olipudase Alfa: First Approval
作者
关键词
-
出版物
DRUGS
Volume 82, Issue 8, Pages 941-947
出版商
Springer Science and Business Media LLC
发表日期
2022-05-31
DOI
10.1007/s40265-022-01727-x
参考文献
相关参考文献
注意：仅列出部分参考文献，下载原文获取全部文献信息。- Sustained and continued improvements in pulmonary function, hepatosplenomegaly, dyslipidemia, and disease biomarkers in 5 adults with chronic acid sphingomyelinase deficiency after 6.5 years of olipudase alfa enzyme replacement therapy
- (2022) Robin Lachmann et al. MOLECULAR GENETICS AND METABOLISM
- Continued improvement in pulmonary, visceral, biomarker and growth outcomes in children with chronic acid sphingomyelinase deficiency treated with olipudase alfa enzyme replacement therapy: 2-year results of ASCEND-Peds
- (2022) George A. Diaz et al. MOLECULAR GENETICS AND METABOLISM
- A randomized, placebo-controlled clinical trial evaluating olipudase alfa enzyme replacement therapy for chronic acid sphingomyelinase deficiency (ASMD) in adults: One-year results
- (2022) Melissa Wasserstein et al. GENETICS IN MEDICINE
- eP280: Continued improvement in adults with acid sphingomyelinase deficiency after 2 years of olipudase alfa in the ASCEND placebo-controlled trial
- (2022) Melissa Wasserstein et al. GENETICS IN MEDICINE
- Changes in PCSK 9 and apolipoprotein B100 in Niemann–Pick disease after enzyme replacement therapy with olipudase alfa
- (2021) Bethanie Garside et al. Orphanet Journal of Rare Diseases
- One-year results of a clinical trial of olipudase alfa enzyme replacement therapy in pediatric patients with acid sphingomyelinase deficiency
- (2021) George A. Diaz et al. GENETICS IN MEDICINE
- Long-term efficacy of olipudase alfa in adults with acid sphingomyelinase deficiency (ASMD): Further clearance of hepatic sphingomyelin is associated with additional improvements in pro- and anti-atherogenic lipid profiles after 42 months of treatment
- (2020) Beth L. Thurberg et al. MOLECULAR GENETICS AND METABOLISM
- Olipudase alfa for treatment of acid sphingomyelinase deficiency (ASMD): safety and efficacy in adults treated for 30 months
- (2018) Melissa P. Wasserstein et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Recommendations for clinical monitoring of patients with acid sphingomyelinase deficiency (ASMD)
- (2018) Melissa Wasserstein et al. MOLECULAR GENETICS AND METABOLISM
- Consensus recommendation for a diagnostic guideline for acid sphingomyelinase deficiency
- (2017) Margaret M. McGovern et al. GENETICS IN MEDICINE
- Novel first-dose adverse drug reactions during a phase I trial of olipudase alfa (recombinant human acid sphingomyelinase) in adults with Niemann–Pick disease type B (acid sphingomyelinase deficiency)
- (2015) Margaret M. McGovern et al. GENETICS IN MEDICINE
- Nonclinical safety assessment of recombinant human acid sphingomyelinase (rhASM) for the treatment of acid sphingomyelinase deficiency: The utility of animal models of disease in the toxicological evaluation of potential therapeutics
- (2015) James M. Murray et al. MOLECULAR GENETICS AND METABOLISM
- Successful within-patient dose escalation of olipudase alfa in acid sphingomyelinase deficiency
- (2015) Melissa P. Wasserstein et al. MOLECULAR GENETICS AND METABOLISM
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