Gene therapy has yielded promising results for treating various life-threatening rare diseases like muscular dystrophy and cystic fibrosis. This is groundbreaking for individuals living with rare disease, whose limited treatment options often entail ongoing, high-cost medical visits. However, these new treatments are entering the market at considerably high prices and posing affordability challenges for patients. We’ve convened a group of rare disease experts to talk through the potential that gene therapy has to treat rare diseases, and how to navigate their financial barriers.

Date: Tuesday, Nov. 16th, 2021

Time: 12:00 – 1:00pm PT

Panelists:

• • Jennifer Hodge, PhD: US Rare Neurology Medical Team Lead, Pfizer
  • Ryan Fischer: Chief Advocacy Officer, Parent Project Muscular Dystrophy
  • Angela Ramirez Holmes: Founder, Cal Rare

Jennifer Hodge is the US Rare Neurology Medical Team Lead at Pfizer. Over the past 9 years, she has contributed in roles of increasing responsibility across Early Pipeline/Gene Therapy, Sickle Cell Disease, Hemophilia and I&I where she played an important role in the US & EU launches of XELJANZ for Rheumatoid arthritis. She received her PhD in Immunology and completed two Post-Doctoral Fellowships at Harvard Medical School & the Yale University School of Medicine.

Ryan Fischer serves as the Chief Advocacy Officer for Parent Project Muscular Dystrophy and has been with the organization for 16 years. Within PPMD, Ryan oversees patient advocacy, patient-focused drug development initiatives including patient-preference research, and the strategic development of the largest patient reported registry in Duchenne developed by PPMD, The Duchenne Registry.

Angela Ramirez Holmes started her advocacy in rare diseases when her 8 year old son was diagnosed with a rare brain condition, an arteriovenous malformation. Angela is the Founder and President of California Action Link for Rare Diseases (Cal Rare), a non-profit membership coalition of rare disease stakeholders that began in 2017 and is dedicated to improving the lives of rare disease patients in California.