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Review in EMBO Molecular Medicine

8/3/16 - NIJMEGEN - the March edition of EMBO Molecular Medicine features a review about the development of small-molecule treatment strategies for mitochondrial disorders in children. In this collaboration between the RCMM, Khondrion and the University of Hong Kong, the authors describe the path from diagnosis through whole-exome sequencing and preferably functional genomics, to studies of pathophysiology, the path from drug development to trials, and the great importance of developing clinically relevant outcome measures. In addition the authors present a current overview of small-molecule drugs in development for tackling mitochondrial disorders.

Read the full paper by clicking on the link below:

Mitochondrial disorders in children: toward development of small-molecule treatment strategies. Koopman WJ, Beyrath J, Fung CW, Koene S, Rodenburg RJ, Willems PH, Smeitink JA. EMBO Mol Med. 2016 Mar 7. pii: e201506131. doi: 10.15252/emmm.201506131. [Epub ahead of print] Review. PubMed PMID: 26951622.