Approches génétiques intégrées et nouvelles thérapies pour les maladies rares (INTEGRARE)
… Pompe disease in a rat model with muscle-directed AAV gene therapy Elena Barbon, Giuseppe Ronzitti, Federico Mingozzi 01/03/2024 Molecular Metabolism The myotubular and centronuclear myopathy patient registry: a … Pompe disease in a rat model with muscle-directed AAV gene therapy Elena Barbon, Giuseppe Ronzitti, Federico Mingozzi …Published on: