Long-term major clinical outcomes in patients with long chain fatty acid oxidation disorders before and after transition to triheptanoin treatment--A retrospective chart review.

Vockley, Jerry; Marsden, Deborah; McCracken, Elizabeth; DeWard, Stephanie; Barone, Amanda; Hsu, Kristen; Kakkis, Emil.
Mol Genet Metab; 116(1-2): 53-60, 2015 Sep-Oct.
Artigo em Inglês | MEDLINE | ID: mdl-26116311