In addition to finding ways of building community with one another during this unprecedented time, mitochondrial disease adult patients and parents with affected children want to understand how academia is partnering with private industry to propel the development and delivery of life-giving therapeutics. Dr. Falk will help to unpack the therapeutic development process across an integrated spectrum from basic science to translational research and from natural history studies to clinical trial design and implementation in the pursuit of expediting the discovery of ‘next generation’ mitochondrial disease therapies that meaningful improve the health and well-being of individuals living with mitochondrial disease.
Focus will be given on how CHOP partners with a range of internal and external partners, including diverse life science companies, to identify, develop, evaluate, and implement effective therapies for diverse aspects of mitochondrial disease in pediatric and adult patients. Families are encouraged to submit questions in advance, which will be discussed in a live chat by a panel of CHOP Mitochondrial Medicine experts, regulatory colleagues, and external life science partners.
Precision medicine for mitochondrial disease would not be possible without the continued support, drive, commitment, and collaborative spirit between our patients, parents, clinicians, researchers, and private industry. We look forward to a lively discussion driven by families with expert partners across the therapeutic development pipeline.