Inventors:
Nina N. Raben - N. Bethesda MD, US
Cynthia Schreiner - Gowanda NY, US
Rebecca Baum - Highland UT, US
Shoichi Takikita - Rockville MD, US
Tao Xie - Rockville MD, US
Paul H. Plotz - Washington DC, US
International Classification:
A61K 31/7088, A61P 3/00, A61K 31/675
Abstract:
Provided herein are methods of treating lysosomal storage disease, for instance Pompe disease, through inhibition of autophagy. Optionally, treatment is administered as an adjunct to enzyme replacement therapy (ERT).