May 14, 2014

The FDA is looking for patient advocate voices at their Public Meeting on Neurologic Manifestations of Inborn Errors of Metabolism. Read on for information about attending the meeting in person or via webcast on June 10, 2014:

Dear patient stakeholder,

We invite you to a...

May 9, 2014

Recently, I had the privilege of interviewing Jen Menard, mother to this year’s Ilan-a-thon honoree, 3-year old Livi Menard. As a mother of two, and godmother to a 19-month old nephew with PBD, Jen’s thoughtful answers meant a great deal. I’m inspired by her honesty, a...

May 1, 2014

Congratulations to the Pound the Pavement for Peter team for another successful 5k run in honor of Peter Hopkins! Learn about Peter and view pictures from this year’s event at