The seventh annual AMDA Research Grant (the 2016 Helen Walker Research Grant) was awarded to Dr. Frédéric Lofaso with the

Sanofi Genzyme Begins Pivotal Phase 3 Trial of NeoGAA Investigational Second-Generation Therapy for Pompe Disease Cambridge, Mass. – November 4,

The AMDA is excited to announce a research grant opportunity for applicants who are involved in Pompe Disease research. This

The IPA Board was provided with the following statement regarding Biomarin’s BMN-701 study. If you have any questions or concerns