The Autophagic/Lysosomal Enhancer GHF201 Ameliorates Pathological Features in Patient-Derived Fibroblasts and a Mouse Model of Pompe Disease

SSRN Electronic Journal 2026 Preprint Open access

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Authors: Uri Sprecher, Manjeet Gupta, Shireen Sarji, Or Kakhlon, See PDF
DOI: 10.2139/ssrn.6050465
Cited by: 0

Fields of Study & Subjects

Lysosomal Storage Disorders Research Autophagy in Disease and Therapy Glycogen Storage Diseases and Myoclonus

Publication details

Journal / source
SSRN Electronic Journal
Publication date
1 January 2026
Document type
Preprint