Michele Herndon’s son Mitchell began developing symptoms of an ultra-rare neurological condition in 2012. He went for five years without answers and in 2017, enrolled in the Undiagnosed Diseases Network, an NIH-funded research study that seeks to diagnose people with the most puzzling conditions. He was diagnosed with the genetic disease Mitchell syndrome, which was named for him. He died from the disease in 2019. Herndon not only started the Mitchell & Friends Foundation but is today the program director of the Undiagnosed Diseases Network Foundation. There she has helped establish the UDNF’s new Patient Navigator Program, which is designed to answer general question undiagnosed patients may have, connect them to resources, and guide them through the UDN process. We spoke to Herndon and F. Sessions Cole, secretary of Undiagnosed Disease Network Foundation and professor of Pediatrics at Washington University School of Medicine, about the diagnostic odyssey, the work of the Undiagnosed Disease Network and the Undiagnosed Disease Network Foundation, and how the new Patient Navigator program works.
How a Rare Diagnosis Impacts a Family
Transforming the Rare Disease Landscape with Data
Creating a Playbook for Bespoke Gene Therapies
Finding Answers for Undiagnosed Patients with Rare Genetic Diseases
How a Familiar Face Can Lead to a Rare Disease Diagnosis
Bringing the Trial to the Patient
Targeting Rare and Chronic Kidney Diseases
Addressing the Delivery Challenges of Genetic Medicines
Targeting Tissues Throughout the Body with RNA Therapies
Delivering Rare Disease Therapies to Patients in Need
An Advocate's Journey through Diagnosis, Loss, and Hope
Using Model Systems to Find Drugs to Repurpose for Rare Diseases
How a Small and Young Foundation Catalyzed Research into a Rare Disease
Overcoming the Limitations of Conventional Cell Engineering
Experimental Therapy for Rare Endocrine Disorder Offers Hope of Improved Care
Forging Gene Therapy Capacity and a Pipeline at the Same Time
Using A Natural DNA Repair Process to Improve Genetic Medicines
Empowering Rare Disease Patients with their Own Health Records
Targeting CNS Disease with Gene Therapies
A New Therapy Offers A Different Approach to Inhibiting the Complement System
Create your
podcast in
minutes
It is Free
The emPOWERed Half Hour
NABOR® TALKS
U.S Property Podcast
Aligned Money Show
The Ramsey Show
Planet Money