A businessman’s daughter has an ‘ultra-rare’ disease, so he immersed himself in science to find a gene therapy cure

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Annabel Frost, 3, experiences pain delay because of a rare neurological disease called AHC. Annabel takes medicine to help reduce symptoms. Image: Marlena Sloss/The Washington Post

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.

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.

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.”

Related article:  Washington Post editorial: We have an 'urgent need' for international rules on gene-edited babies

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

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