Concepedia

Publication | Open Access

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

DOIFull Paper Access

28

Citations

12

References

2020

Year

Darius J. Adams, Mark Midei, Jahannaz Dastgir, Christina Flora, Robert J. Molinari, Frederic Heerinckx, Sarah Endemann, Paldeep S. Atwal, Peter Milner, Mikhail S. Shchepinov
JIMD Reports

Abstract

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.

References

YearCitations

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