1-13C-propionate breath testing as a surrogate endpoint to assess efficacy of liver-directed therapies in methylmalonic acidemia (MMA)

Genetics in Medicine, Published online: 05 April 2021; doi:10.1038/s41436-021-01143-8

Source: Genetics in Medicine - April 5, 2021 Category: Genetics & Stem Cells Authors: Irini Manoli Alexandra R. Pass Elizabeth A. Harrington Jennifer L. Sloan Jack Gagn é Samantha McCoy Sarah L. Bell Jacob D. Hattenbach Brooks P. Leitner Courtney J. Duckworth Laura A. Fletcher Thomas M. Cassimatis Carolina I. Galarreta Audrey Thurm Joseph Source Type: research