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    • Therapeutic Benefit of Autophagy Modulation in Pompe Disease. 

      Sun, Baodong; Lim, Jeong-A; Puertollano, Rosa; Raben, Nina (Molecular therapy : the journal of the American Society of Gene Therapy, 2018-05-03)
      The complexity of the pathogenic cascade in lysosomal storage disorders suggests that combination therapy will be needed to target various aspects of pathogenesis. The standard of care for Pompe disease (glycogen storage ...