Mary Kay Koenig Sam Nick Russo Kim L McBride Hans Tomas Bjornsson Brynja Bjork Gunnarsdottir Amy Goldstein Scott A Falk
10.1002/jmd2.12335
JIMD Rep
2022 Sep 21;64(1):65-70.
barth syndrome cardiolipin elamipretide megdel sengers syndrome mitochondrial disease
CC BY
https://pubmed.ncbi.nlm.nih.gov/36636586/
PMC9830009
36636586
Use of Elamipretide in patients assigned treatment in the compassionate use program: Case series in pediatric patients with rare orphan diseases
2022