A systematic review evaluates the therapeutic profile of velaglucerase alfa in pediatric patients with Gaucher disease across all age groups. This research provides insights into the drug's efficacy and safety in younger populations.

Sanofi's drug for Gaucher disease shows promise in recent trials, achieving a 1-for-2 success rate. This development could pave the way for its market entry, following a history of setbacks.

Sanofi plans to submit venglustat for regulatory approval in Gaucher disease following positive Phase III trial results, while the future for Fabry disease remains uncertain after a trial failure. This dual outcome highlights the complexities of rare disease drug development.