The purpose of the webinar is to provide the Batten disease community with information on current Batten disease research efforts including gene therapy, the importance of patient involvement in clinical research, and the availability of community resources and support. Amicus Therapeutics is committed to ensuring that the Batten disease community is provided with accurate and up to date information on Batten disease research.

If you are unable to attend the live webinar, still register so you can receive a link to the recorded content.

Don’t forget, to access this webinar use the password: BDwebinar


  • Welcome
  • Overview of Amicus Therapeutics and Advocacy Programs
  • Clinical Overview of Batten Disease
  • Current Efforts in Batten Disease Research
  • Batten Disease and Gene Therapy
  • A Patient’s Perspective on Clinical Trial Participation
  • Batten Disease and Other Rare Disease Resources


Jayne Gershkowitz, Chief Patient Advocate, Amicus Therapeutics
Emily de los Reyes, MD, Nationwide Children’s Hospital
Noreen Murphy, Batten Disease Support and Research Association (BDSRA)
Mary Beth Kiser, Beyond Batten
Kathrin Meyer, PhD, Nationwide Children’s Hospital
Maria Graham, Parent


Meredith Cagle, Senior Director of Patient Engagement, Global Genes