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  1. Article

    Open Access

    AAV gene therapy for hereditary spastic paraplegia type 50: a phase 1 trial in a single patient

    There are more than 10,000 individual rare diseases and most are without therapy. Personalized genetic therapy represents one promising approach for their treatment. We present a road map for individualized tr...

    James J. Dowling, Terry Pirovolakis, Keshini Devakandan, Ana Stosic… in Nature Medicine (2024)

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  2. No Access

    Article

    Muscle MRI patterns for limb girdle muscle dystrophies: systematic review

    Limb girdle muscle dystrophies (LGMDs) are a group of inherited neuromuscular disorders comprising more than 20 genes. There have been increasing efforts to characterize this group with Muscle MRI. However, du...

    Issa Alawneh, Ana Stosic, Hernan Gonorazky in Journal of Neurology (2023)