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news.Solid Biosciences’ SGT-212 gains FDA rare paediatric disease status
The therapy aims to restore therapeutic frataxin protein levels to address cardiac, neurological and systemic symptoms of FA. Credit: Gorodenkoff/Shutterstock.com.
The therapy aims to restore therapeutic frataxin protein levels to address cardiac, neurological and systemic symptoms of FA. Credit: Gorodenkoff/Shutterstock.com.