| Wednesday, September 18, 2024 |
| 12:45 AM - 04:30 PM Eastern Daylight Time |
We are pleased to announce that Philadelphia partner Andrew R. Mariniello will moderate the Patient Advocacy Spotlight panel at Life Sciences PA’s annual Life Sciences Future.
Panelists Maria Kefalas and Matt Hammond from the Calliope Joy Foundation will share how their advocacy led to the passing of newborn screening bills, the historic FDA approval of gene therapy to treat metachromatic leukodystrophy (MLD), and critical fundraising for the treatment and research of the disease that has personally affected their lives.
Dr. Paul Orchard will discuss his work on gene therapy on MLD patients.
Dr. Amy Waldman will discuss research for new treatments being developed at the Children’s Hospital of Philadelphia’s new Leukodystrophy Center.