L-alanine supplementation in Pompe disease (IOPD): a potential therapeutic implementation for patients on ERT? A case report

• Main Authors: Valentina Rovelli (Author), Juri Zuvadelli (Author), Marta Piotto (Author), Andrea Scopari (Author), Alice Re Dionigi (Author), Vittoria Ercoli (Author), Sabrina Paci (Author), Graziella Cefalo (Author), Elisabetta Salvatici (Author), Giuseppe Banderali (Author)
• Format: Book
• Published: BMC, 2022-03-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

Abstract Background Pompe disease (PD) is a disorder of glycogen metabolism conditioning a progressive and life conditioning myopathy. Enzyme replacement therapy (ERT) is currently the best treatment option for PD, but is not resolutive. While other potential therapeutic approaches have been reporte...