by United Pompe | Jul 18, 2016 | News
In an effort to complete the AGLU02704 study (Late-Onset Treatment Study, LOTS) as responsibly as possible, Genzyme has amended the study protocol to incorporate the use of an adaptive design. Genzyme utilized this method to help ensure a conclusive trial outcome in...
by United Pompe | Jul 18, 2016 | News
Expensive drug gives hope to rare disease sufferer
by United Pompe | Jan 18, 2016 | News
BioMarin is currently seeking patients for a Phase 3 clinical trial in Late Onset Pompe Disease. This phase 3 study incorporates a switchover design in which all patients switch directly from Myozyme® or Lumizyme®* to the experimental drug BMN 701 without missing...
by United Pompe | Jan 18, 2016 | News
Amicus Therapeutics Announces First Patient in Phase 2 Study for Pompe Disease
by United Pompe | Jan 18, 2016 | News
MedicAlert Brochure
by United Pompe | Jan 18, 2016 | News
Background We are the parents of Sari, a Belgian girl of 4.5 years old suffering from infantile Pompe´s disease. Based on the normal development of the disease, Sari would have passed away already, probably before her 1st birthday. However she has been receiving an...