Mitochondrial Disease | CHOP Research Institute

Mitochondrial Disease

Primary mitochondrial diseases (PMD) are a group of energy deficiency disorders that are typically progressive and range in onset and severity.
Published on
Nov 16, 2023
The Connor Initiative, a multimillion dollar gift from the Holveck Family, will support precision therapeutics for osteosarcoma and rare cancers.

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.

Published on
Nov 9, 2022
A U.S. Department of Defense grant will support CHOP researchers in their study of noninvasive methods to diagnose mitochondrial diseases.
Published on
Oct 28, 2022
In the News: Researcher Awards, Hearing Loss in Mitochondrial Disease, Abu Dhabi Partnership

Evaluate the safety and efficacy of an investigational drug called "Elamipretide" in adult patients with mitochondrial disease.

Study focusing on non-invasive MRI procedures to measure biological activity within leg muscles of individuals, including both healthy volunteers and those with mitochondrial disease.

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 16, 2022
This week’s In the News highlights research on mitochondrial disease, acute lymphoblastic leukemia, type 2 diabetes, and sleep apnea.
Published on
Apr 29, 2022
Read about the latest discoveries and awards at Children’s Hospital of Philadelphia Research Institute.