Terry Pirovolakis shares his personal journey as the father of Michael, who has the rare neurodegenerative disease SPG50, and details the extensive efforts undertaken to develop a gene therapy treatment.
After liquidating his life savings to fund 4 million in research, Terry succeeded in creating a charity and collaborating with experts, leading to a gene therapy treatment for Michael at age four. He discussed the gene therapy process, challenges faced during clinical studies and FDA approval, as well as the formation of Elpida Therapeutics to expand their focus to treat additional rare diseases (10,000 globally).
Highlighting the financial sustainability model through the Priority Review Voucher system, Terry expressed ambitions to scale treatment efforts significantly. He emphasized the need for community support and revealed plans for future fundraising initiatives, including a 100 million grant application to advance multiple clinical programs. Terry provides continuing education for families starting their rare disease journeys and disseminating fundraising information to assist more children in need. https://www.elpidatx.com/

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