Rare is more common than you think.
If all people with rare diseases lived in one country, it would be the world’s third most populous country.
More than 30 million children and families suffer from rare pediatric diseases. The Take Part Foundation was established to raise money to support those researchers who work tirelessly and endlessly to find answers.
Unfortunately, traditional funding sources for these projects simply don’t exist due to the small number of children impacted. As a result, research for cures or advances for these rare pediatric diseases often experience stops and starts that push progress off by years, if at all. Take Part bridges the funding gap so researchers can make enough progress to publish and apply for larger grants that sustain their research.
No longer do families need to feel sidelined and helpless, dependent on others to do something – anything to make progress that might help their child. These children are warriors, fighters—so are we. Join us and “take part” in the fight.
Give hope. Give possibilities.
WHAT WE DO
Help Kids & Their Parents Fight for Possible
Project Based Research
Our approach to funding research is unique. Similar to how an idea becomes a sustainable business, our projects have a plan, anticipated milestones and are reviewed by our advisors to ensure validity.
Sharing Research Findings
We aspire to be a hub of information for families and doctors around the world who are working on rare pediatric diseases. Sharing findings mitigates the communication disconnect that often occurs. Our approach leads to collaboration and potential advancement.
Life Saving Individual Profiles for Kids and Parents
All too often parents are told, “just wait” and left to navigate this journey on their, struggling to help their child survive long enough for science to catch up. We help families share their stories so they can connect with others who are also looking for answers.
How Our Projects Work
Each project is required to present a research case description, anticipated funding needs, project timing and proposed milestones. We also require that the team applying have 20% of the initial project funding in place. Our Take Part Foundation advisors (comprised of doctors, business executives and leaders within partner organizations) then review the application for validity of project model and medical assumptions, financial evaluation and proof of concept.
Once accepted, each Take Part project is assigned a project manager by the foundation. Our project manager will be responsible for oversight, including collaborating to set agreed upon project milestones and metrics, ensuring the receipt of updates on results, reporting to and validating with the foundation’s advisory board, as well as allocating project funding to the designated institution. It’s important to note that funding will only be issued to the institution affiliated with the project; funding will not be given directly to the research team or to any individual.
Our Current Project
Project 0001: PyroXD1
PYROXD1 (Pyridine Nucleotide-Disulphide Oxidoreductase Domain 1) is a Protein Coding gene. Diseases associated with it present as extremely rare and progressive forms of muscular dystrophy. Less than 20 people worldwide have this specific mutation, one of those people is our daughter Natalie. Her case is considered extremely severe, as her symptoms set in at a very young age. Natalie cannot walk, crawl, or do any of the things any other 3-year-old can do. She can barely use her muscles at all. A simple cold means endless trips to the emergency room. Natalie needs constant round-the-clock care, receives hyperbaric oxygen therapy twice a week ,and uses a G tube to eat. She fights every day.
Her mutation has no cure. And, due to the small number of cases, traditional research funding from pharmaceutical companies and others simply doesn’t exist. It doesn’t make financial sense.
Funding for this project, estimated at $250,000, will allow researchers to discover insights into the origin of this mutation, how it affects the body and possible treatment options. We know this won’t save Natalie, but we hope it will lead the way for finding a cure for PYROXD-1 and help others with rare pediatric diseases.
How Can You Help?
Your donation will help us save and improve lives with research, education and emergency care.