On 6/29/22, the Hope for Harvey Foundation signed a contract with Everlum Bio, a rare disease lab who believes that everyone deserves a cure, no matter how big or small the disease. Everlum Bio is based in Austin, TX, and we are excited to partner with them on creating a treatment for DLG4-related Synaptopathy.
We are currently working on these specific tasks with funds from this GoFundMe Campaign and HOPE for Harvey Foundation Fundraising Efforts:
Currently there is no treatment options for DLG4-related Synaptopathy. We hope to change that with your help! 100% of donations will support the development of various treatment modalities.
Due to advocacy by HOPE for Harvey Foundation, two academic labs recently started research projects on DLG4 Synaptopathy:
"Collins concluded by thanking his audience while urging them to do more. 'Of nearly 7,000 genetic disorders, only 500–600 have an FDA-approved therapy,' he said. The goal is to develop therapies for the thousands of disorders for which patients and their families are still waiting in vain."
"After excluding genetic variants that were also found in the control group and in a separate large cohort of more than 60,000 individuals without autism, Doan, Yu and colleagues were left with 41 genes that were knocked out only in individuals with autism. Overall, the researchers estimate that these genes explain another 3 to 5 percent of all cases of autism (2 percent from loss-of-function mutations, and 1 to 3 percent from missense mutations)."
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