Treatment of infantile neuroaxonal dystrophy with RT001: A di-deuterated ethyl ester of linoleic acid: Report of two cases.

Darius AdamsMark G MideiJahannaz DastgirChristina FloraRobert J MolinariFrederic HeerinckxSarah EndemannPaldeep AtwalPeter MilnerMikhail S Shchepinov

Published in: JIMD reports (2020)

Oral RT001 was administered safely in two subjects with INAD. Early findings suggest that the compound was well tolerated, metabolized and incorporated in the RBC membrane. A clinical trial is underway to assess efficacy.

Keyphrases

clinical trial
escherichia coli
phase ii
cystic fibrosis
open label
pseudomonas aeruginosa
red blood cell
combination therapy
smoking cessation
replacement therapy
placebo controlled