Safety and efficacy evaluation of investigational liver gene transfer for secretable GAA in the treatment of Pompe disease
Sean M. Armour(Spark Therapeutics (United States)), Federico Mingozzi(Spark Therapeutics (United States)), Xavier M. Anguela(Children's Hospital of Philadelphia), Marco Crosariol(Spark Therapeutics (United States)), Haley Hanby(Spark Therapeutics (United States)), Helena Costa Verdera(Spark Therapeutics (United States)), Virginia Haurigot(Spark Therapeutics (United States)), Giuseppe Ronzitti(Inserm), Pasqualina Colella(Stanford Medicine), Francesco Puzzo(Inserm), Umut Cagin(Inserm), Fanny Collaud(Inserm), Jayme M.L. Nordin(Spark Therapeutics (United States))
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