Early treatment with laronidase improves clinical outcomes in patients with attenuated MPS I: a retrospective case series analysis of nine sibships

Conclusions:
These findings suggest early initiation of laronidase, prior to the onset of symptoms in patients with attenuated MPS I, can slow or prevent the development of severe clinical manifestations.

Source: Orphanet Journal of Rare Diseases - October 7, 2015 Category: Internal Medicine Authors: Nouriya Al-SannaaLuisa BayDeborah BarbouthYoussef BenhayounCyril GoizetNorberto GuelbertSimon JonesSandra KyosenAna MartinsChanika PhornphutkulCelia ReigRebecca PleatShari FalletIva Ivanovska Holder Source Type: research