An article in the Sunshine Coast Daily, Australia, describes why a Pompe sufferer goes to the U.S. to participate in a clinical trial. As Myozyme, a drug for enzyme replacement therapy of Pompe disease, is not funded in Australia, the participation in a clinical trial is a chance to receive a drug, at least for the duration of the clinical trial. The clinical trial is run by BioMarin to evaluate a new drug that may be competitive to Genzyme’s Myozyme (ClinicalTrials.gov identifier: NCT01230801).
- Read the article online (Sunshine Coast Daily, Australia)