A Novel Gene Therapy Approach for GSD III Using an AAV Vector Encoding a Bacterial Glycogen Debranching Enzyme

Por Jeong-A Lim, Su Jin Choi, Fengqin Gao, Priya S. Kishnani, Baodong Sun
Publicado em 2020-09-01

Immune Modulation for Enzyme Replacement Therapy in A Female Patient With Hunter Syndrome

Por Daniel C. Julien, Kara Woolgar, Laura Pollard, Holly Miller, Ankit Desai, Kristin Lindstrom, Priya S. Kishnani
Publicado em 2020-05-01

Pregnancy Outcomes in Late Onset Pompe Disease

Por Ozlem Goker-Alpan, Vellore G. Kasturi, Maninder K. Sohi, Renuka P. Limgala, Stephanie L. Austin, Tabitha Jennelle, Maryam Banikazemi, Priya S. Kishnani
Publicado em 2020-09-01

Assessment of cognitive scales to examine memory, executive function and language in individuals with Down syndrome: implications of a 6-month observational study

Por Xavier Liogier D’Ardhuy, Jamie Ogline Edgin, Charles eBouis, Susana ede Sola, Susana ede Sola, Celia eGoeldner, Priya eKishnani, Jana eNöeldeke, Sydney eRice, Silvia eSacco, Lisa eSquassante, Gail eSpiridigliozzi, Jeannie eVisootsak, James eHeller, Omar eKhwaja
Publicado em 2015-11-01