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How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years

How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years
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SPG50: Toronto boy receives new treatment for rare disease

After a Toronto boy became the first person to receive a new gene therapy to treat the rare genetic disease spastic paraplegia type 50 or SPG50 earlier this year, his family hopes to connect sick children around the world with the treatment too. ....

United States , Terry Pirovolakis , Us Federal Drug Administration , Drug Administration ,

Four-Year-Old Boy Receives Cure for Rare Disease

Michael Pirovolakis, a four-year-old boy in Toronto, is now the only child in the world to receive a potential cure for his extremely rare disease. After his parents desperately raised $3 million over a period of three years, they finally have faith for their son’s possible recovery.

He is the on ....

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