info@amda-pompe.org

Valerion Initiates VAL-1221 Dosing in Patients with Pompe Disease

Jul 11, 2017 | NEWS

CONCORD, Mass., July 11, 2017—Valerion Therapeutics, a clinical-stage biotechnology company that specializes in the development of therapies for orphan genetic diseases, today announced that it has initiated dosing in a Phase 1/2 clinical trial evaluating VAL-1221 in...

In Memory of Henri Termeer

May 19, 2017 | NEWS

he AMDA was saddened to learn of the sudden passing of Henri Termeer, former CEO of Genzyme. We have known and worked with Henri since the late-90s through his role as the CEO of Genzyme. We will never forget how he always took the time to talk to patients and their...

Pompe in the News: What Happened to the Truth?

Feb 28, 2017 | EDITORIALS

By: Tiffany House–AMDA President posted on: March 03, 2017   On February 28, 2017—Rare Disease Day—Pompe disease was once again the subject of national media attention. During his Joint Address to Congress, President Trump brought national attention to patients...

2017 PCMA of Texas Pull for Pompe!

Jan 5, 2017 | NEWS

The AMDA is excited to announce that the 2017 PCMA of Texas Pull for Pompe will take place on Saturday, March 25th at the National Shooting Complex in San Antonio, Texas! This is the 7th Annual Pull for Pompe hosted by the PCMA of Texas. The Pull for Pompe clay...