Mitochondrial Medicine | CHOP Research Institute
 

Mitochondrial Medicine

Published on
Sep 5, 2025
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The Mitochondria and Cancer Connections (MC2) Research program leverages knowledge from existing centers to establish a novel, cutting-edge research initiative and enable the development of mechanistic understanding and novel therapeutic opportunities at the intersection of mitochondrial disease and pediatric cancer.

We are recruiting patients with genetically confirmed primary mitochondrial myopathy who are at least 18 years old for a randomized, double-blind, placebo-controlled study of a new drug to treat this disorder.

Published on
Sep 22, 2021
Marni Falk, MD, and Adam Resnick, PhD, to lead the Mitochondria and Cancer Connections (MC²) Research Program, a new CHOP research initiative.

We are studying an investigational drug called sodium dichloroacetate (DCA).

The Mitochondrial Medicine Research Laboratory investigates the genetic etiologies, pathophysiologic mechanisms, and therapeutic targets of mitochondrial disease.  The lab applies state-of-the-art technologies and approaches in diverse animals (C. elegans worms, D. rerio zebrafish, M.

The Resuscitation Science Center is a multifaceted, collaborative scientific platform dedicated to understanding critical illnesses and accelerating discoveries to improve resuscitation care and outcomes for children. By partnering translational and clinical science, the Center takes a true bench-to-bedside approach to pediatric research.

The Ganetzky Lab focuses on inherited mitochondrial respiratory chain disorders with a goal of applying biochemical approaches to develop improved diagnostic modalities, especially for mitochondrial Complex V (ATP Synthase) deficiency.

Dr. Ganetzky is passionate about developing new biochemical strategies for accurate diagnosis and classification of inborn errors of energy metabolism. She has a particular focus on diseases of mitochondrial complex V (ATP Synthase).

E-mail:
ganetzkyr [at] chop.edu

Dr. Falk is a Clinical Geneticist who serves as executive director of the Mitochondrial Medicine Frontier Program. Her translational research lab investigates the causes and global metabolic consequences of mitochondrial disease, as well as targeted therapies, in C. elegans, zebrafish, mouse, and human tissue models of genetic-based respiratory chain dysfunction.

E-mail:
falkm [at] chop.edu