YBRP 2021 HNRNPH2 Virtual Walk
The Yellow Brick Road Project is so excited to be hosting our first virtual walk on July 31st, during this year's HNRNPH2 Awareness Week!
YBRP completed our funding of two-year $420k research project with Andlit Therapeutics. But that is not the end! Completion of this initial project places us at the "Disease Concept/Disease Burden" phase of the overall process to reach clinical trials, as illustrated below. The next steps are to identify outcomes and to fund research projects to conduct efficacy & toxicity tests, with a total estimated cost of $1.4M - $1.8M (see below for details). This research would place us in the "Mechanisms ID'd" phase of the clinical trial process.
How do I take part in the walk?
- Register for the walk
Choose the "Click Here to Register for the Walk" option below and you will be taken to the registration page. As an official participant, you’ll receive a printable ‘bib’ to attach to your shirt, showing your support for the YBRP and HNRNPH2 research. You’ll also receive a discount code for YBRP products through the store on our website. Get your "swag" in time to wear for the walk!
- Spread the Word
Once you’re signed, up, invite your friends and families to walk or to sponsor you and your family! You can also set up a Facebook fundraiser for your walk as well. Each dollar brings us closer to completing our mission and finding a potential treatment.
- Walk Your Way!
On July 31, go for a walk. This can be any kind of walk you like. If local restrictions allow, gather with friends and family. Take a walk around your local track, walking trail, or park. Walk for a block, walk for a mile, or ten miles. Roll or wheel if walking is difficult. Can't get out or gather? Walk on your treadmill or elliptical! Walk YOUR Way! The only requirement is that you have fun! We’d also love for you to take a photo of your walk and share it with us on social media, tagging #2021ybrpwalk #hnrnph2 #walkyourway
What should I do if I'd like to support the walk, but am unable to take part myself? You can sponsor any walkathon participant or HNRNPH2 patient and/or family! The choice is yours, just click below to register for or sponsor this year's walk!
Our Research and Fundraising Goals
To accelerate the progression of the project with a significant funding resource, Andlit will focus on:
1. Characterizing, establishing an ASO campaign and building a mouse model for HNRNPH2 mutations. These mutations are ~15% of the patients and were not included in the initial project plan. They add significant amount of patient to this rare syndrome.
2. Adding manpower to the project (experienced scientist and two junior scientist) to facilitate the work progress.
3. Performing pre-clinical experiments on the mouse models with lead molecules:
A. Characterizing the neurological phenotype caused by HNRNPH2 mutations.
B. Performing efficacy experiments (phenotype/disease correction) by lead ASO.
C. Toxicity studies of the lead ASO.
Total cost of this plan is estimated to be $1,400,000 - $1,800,000
*illustration courtesy of COMBINEDBrain.