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Gene therapy halts progression of rare genetic condition in young boy

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Scienceblog.com 
Michael is checked by a nurse at SickKids before receiving gene therapy in a single-patient clinical trial for spastic paraplegia type 50 (SPG50).
Michael is checked by a nurse at SickKids before receiving gene therapy in a single-patient clinical trial for spastic paraplegia type 50 (SPG50).