We are extremely excited about the treatments we are working hard to bring to the clinic… but that process is incredibly costly. That’s why this year we have a HUGE goal:

We need to raise $1,000,000!

Here is a taste of what we are funding with this $1,000,000…

  • GENE THERAPY

    Our incredibly promising work from Muotri Lab at @ UCSD and the Cogram Lab at the University of Chile has resulted in biotech company www.Mahzi.com licensing our work and launching their company with Pitt Hopkins as one of their first 3 pipeline indications. To move gene therapy forward our starting costs are $500K.

  • MTT THERAPY

    We have completed our first clinical trial of microbiota transfer therapy with great success in reducing the severe and debilitating gut issues our children suffer.The FACT THAT WE HAVE A TREATMENT TO BRING TO THE FDA FOR APPROVAL IS UNPRECEDENTED FOR SUCH A YOUNG FOUNDATION. Our hope is to bring this to market ASAP. Starting Costs are $250K.

  • ASO GENETIC THERAPY

    In addition to traditional gene therapy, our ASO (antisense oligonucleotide approach) is a promising avenue we must continue moving forward if we want to have all shots on goal. In this approach, we upregulate the TCF4 protein production of the working gene copy. Annual cost to continue the program is $250K.

WAYS YOU CAN GET INVOLVED TO HELP REACH THE GOAL…

• Host a fundraising event

• Start an e-mail campaign

• Begin a Facebook fundraiser

• Create a postcard or letter campaign

DONATE TODAY

$1,000,000 by December 31, 2022… we got this!

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