Welcome to the HOPE for Harvey Foundation for DLG4
Helping Overcome PSD-95 Errors for the DLG4 Community
Welcome to the HOPE for Harvey Foundation for DLG4
Helping Overcome PSD-95 Errors for the DLG4 Community
Amazing HOPE for Harvey Update!
I am beyond grateful to share Grann Pharmaceuticals in collaboration with HOPE for Harvey have filed our final Investigational New Drug (IND) Application to the FDA to seek approval to start treatments for our mRNA lipid nano particle (LNP) treatment for DLG4.
This would be the first-ever targeted treatment for DLG4 mutations, which cause seizures, motor, language, and cognitive delays. Another huge blessing, is the foundation will own the IP rights, so our community can never lose access to this life-changing treatment.
Without the amazing support of our donors, the incredible work by Grann Pharmaceuticals in creation of this new type of technology, Harvey's amazing neurologist who has been working with our team to ensure Harvey can receive these treatments close to home, as well as our research partners, like Dr. Anna Pfalzer and so many more, we could never have accomplished this milestone.
This was truly a hope and a prayer just 4.5 years ago. I knew that my son's only hope for a treatment in a timeframe that would make a difference in his life was truly on my shoulders. Parents and small foundations are leading the way in creating these treatments for ultra-rare diseases, because big pharma has no interest in diseases that won't reap them billions in profit. Even through so many ups and downs and wondering if this day would ever come, I knew I would never regret trying. To all of the DLG4 families who regularly reach out to me, hoping and praying as well, I am hopeful. I am hopeful for my son, for our community, and for the children yet to be born with DLG4 mutations for a life-changing treatment.
We can't wait to share next steps and updates along the way for my sweet boy and so many others just like him.
