Simon and Nina Frost had spared no expense, taking Annabel to all the best neurologists around the country. Finally a potential diagnosis emerged: alternating hemiplegia of childhood, an ultrarare genetic disorder.
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They also learned that there is no effective treatment or cure, [and] that any one of Annabel’s episodes has the potential to lead to permanent brain damage or death.
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The real estate investor and developer and former rugby player had no background in science, “but honestly,” [Simon] says, “I felt like the only way to get towards any type of therapy for Annabel and AHC kids was to do it myself.”
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Over the next two years, Frost pursued the science of AHC.
He is now listed as an inventor on U.S. and international patent applications for a developing gene therapy and its delivery method. Frost also has become a project manager, coordinating many parts of the research — and playing a role in designing some of it — that could lead to new treatment for his daughter. He is now heading up an effort to develop a novel gene therapy with the goal of getting it to clinical trial.
Read full, original post: His daughter Annabel as a rare disorder. He’s developing a novel gene therapy
