Our Stance
Everyone has genetic mutations, our genetic code is what makes us unique and creates the full spectrum of humanity. It’s only when the mutation occurs on a gene that is essential for the human body to function, like PDHA1, that there is disease. Children with PDCD are perfectly made AND they have disabilities. They have rich, full lives with families that love them AND their bodies are fighting a progressive disease. For the families of PDCD, gene therapy is not about “fixing” our kids, but giving them the best therapeutic science has to offer. Gene therapy will extend their lifespan, alleviate the most painful symptoms of their disease, and improve quality of life. Our hope for gene therapy is that our children will spend less time in the hospital and more time living their lives.
If you’d like to improve the life of a child with PDCD, please visit our Get Involved page or consider making a small monthly donation.
Our Core Values
EMPOWER PDCD FAMILIES
We hold researchers accountable with milestones and reporting and, in turn, we hold ourselves accountable to our most important stakeholders: PDCD families. As a PDCD-patient run advocacy group we seek to own PDCD-related assets and provide input on our disease whenever possible. We seek to make decisions as a community, empower PDCD families with knowledge and encourage them to ask hard questions.
BUILD A BETTER FUTURE FOR PDCD
Our goal is to build something that future generations can improve upon. We appreciate the incredible progress in genetic and precision medicine over the last few decades, but we acknowledge that we still have much farther to go. We understand that any potential therapeutics we help develop will be the first of its kind. For the families of PDCD, gene therapy is not about “fixing” our kids, but giving them the best therapeutic science has to offer.
STRONGER TOGETHER
We know that PDCD is an incredibly complex disease, with a vast spectrum of clinical outcomes. We also know that as a mostly spontaneous disease, PDCD does not favor any single population over another. We advocate for fairness and equality for all of our babies. We believe we will reach a cure by working with a cross functional team of stakeholders from academia, industry, and the families themselves. We acknowledge that everyone has an incredibly personal experience with the diagnosis, how they handle the disease, and which barriers they face. Finally, we rise above political and cultural differences to collaborate on our most important shared goal, a good outcome for patients with PDCD.
NEVER. GIVE. UP.
We wouldn’t change our kids for the world. But we WILL change the world for our kids. Our kids face impossible odds everyday. Our work with Hope for PDCD is a tribute to them. We know this disability and rare disease life is not easy. We take breaks when we need to. We accept a bad day, bad month, bad year, and then we get back up and get in the fight to achieve the impossible - a cure for PDCD.