More than 25 million Americans are affected by one of more than 7,000 rare genetic diseases, impacting how they perceive and interact with the world around them. Less than 600 of these diseases have an FDA-approved therapy. Our son, Harvey, was diagnosed with an ultra-rare genetic disorder, a DLG4 mutation, with more than 60 identified cases.
We are fundraising to create a treatment for this disease. We are exploring various approaches, including repurposed drugs or small molecules, Allele-specific oligonucleotide (ASO), and any other viable modality. We are just getting started but are already in the process of looking at micro labs and research organizations to begin this work and are engaged with other established consulting companies to guide us in our decisions. All funds raised here will go directly toward research and into the HOPEforHarvey Foundation, a nonprofit 501c3 organization, which we are currently in the process of forming.