Thank you to every person who donated over the past month to support PPMD’s Gene Therapy Initiative. Thanks to your generosity, we can continue answering the difficult questions we face when it comes to the future of Gene Therapy.
We are also grateful to the Small Heroes Foundation for matching all gifts up to $50,000. Their commitment to this vital research is deeply appreciated.
As you’ve heard throughout the campaign, there are several obstacles we face in bringing the remarkable potential of Gene Therapy to all who will benefit from it. There are challenges regarding neutralizing antibodies, redosing, accessibility to older or non-ambulatory individuals and barriers to approval.
PPMD will continue to work with the best and the brightest in their fields to approach solutions from every angle. We will continue to discuss the important issues surrounding Gene Therapy, fund immune response research and related projects, support the Pediatric Gene Therapy Medical Ethics (PGTME) working group at New York University, and work with the FDA and our champions in Congress to incorporate patient and caregiver preferences in their decision making and approval process.
It is because of you that we will turn obstacles into possibilities. Thank you.Donate >