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BioMarin is currently seeking patients for a Phase 3 clinical trial in Late Onset Pompe Disease
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...
Amicus Therapeutics Announces First Patient in Phase 2 Study for Pompe Disease
Amicus Therapeutics Announces First Patient in Phase 2 Study for Pompe Disease
MEDICALERT BROCHURE
MedicAlert Brochure
SARI
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...
BartJan
Our son BartJan was 6 weeks old (born 14-10-1998) when we heard the diagnoses ''Pompe´s disease''. The doctor didn’t trust some signs BartJan was showing and did some tests to find out what was wrong. Our world collapsed when the seriousness of this disease sank in...
Survey for Grandparents of Children with Pompe
Grandparent's Survey
UNITED STATES POMPE COMMUNITY UPDATE, AUGUST 1, 2014
United States Pompe Community Update
Expression of Hope 2014
Expression of Hope 2014
Genzyme Receives Label Expansion for Lumizyme® (alglucosidase alfa) in the United States for the treatment of Pompe Disease
Genzyme Label Expansion for Lumizyme
Share your thoughts by participating in an interview
Share your thoughts by participating in an interview… EVIDERA is seeking participants for interviews about their experience with late-onset Pompe disease • Interviews will be conducted August 21st to August 24th in Santa Monica, California • Interviews will involve...