Summary

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• Elelyso (taliglucerase alfa) is indicated for the treatment of type 1 Gaucher disease in patients 4 years and older with a confirmed diagnosis.
• This summary is based on the review of one systematic review(s)/meta-analysis(es). ^[1]
• Taliglucerase alfa was effective in treating both naïve and experienced Gaucher disease (GD) patients, with effectiveness confirmed in both short- and long-term follow-ups.
• Compared to other drugs, taliglucerase alfa, along with imiglucerase and velaglucerase alfa (ERTs), was effective in treating GD, while miglustat (SRT) did not significantly improve blood outcomes in naïve patients and decreased platelet levels in experienced patients.
• No significant differences in effectiveness were noted among different population types and subgroups for taliglucerase alfa, with consistent effectiveness observed across both naïve and experienced patients.
• There is no safety information available in the reviewed studies.
• The systematic review evaluated taliglucerase alfa's effectiveness across naïve (untreated) and experienced (previously treated) Gaucher disease patients, noting that while enzyme replacement therapies (ERTs) like taliglucerase alfa were effective in both groups, miglustat showed no significant improvement in blood outcomes for naïve patients and a decrease in platelet levels for experienced patients, whereas eliglustat maintained stable outcomes in experienced patients.