Small-molecule Inhibition of Glycogen Synthase 1 for the Treatment of Pompe Disease and Other Glycogen Storage Disorders
Julie C. Ullman,Kevin T. Mellem,Yannan Xi,Vyas Ramanan,Hanne Merritt,Rebeca Choy,Tarunmeet Gujral,Lyndsay E. A. Young, Kerrigan Blake,Samnang Tep,Julian R. Homburger, Adam O'Regan,Sandya Ganesh, Perryn Wong,Terrence F. Satterfield,Baiwei Lin,Eva Situ,Cecile Yu, Bryan Espanol, Richa Sarwaikar, Nathan Fastman,Christos Tzitzilonis,Patrick Lee,Daniel Reiton,Vivian Morton,Pam Santiago,Walter Won,Hannah Powers,Beryl B. Cummings,Maarten Hoek,Robert R. Graham,Sanjay J. Chandriani,Russell Bainer,Anna A. DePaoli-Roach,Peter J. Roach,Thomas D. Hurley,Ramon C. Sun,Matthew S. Gentry,Christopher Sinz,Ryan A. Dick,Sarah B. Noonberg,David T. Beattie,David J. Morgans Jr,Eric M. Green SCIENCE TRANSLATIONAL MEDICINE（2024）
Key words
Glycogen Storage Diseases,Enzyme Replacement Therapy
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