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.
Tackling the Pricing Challenges for Advanced Therapies for Rare Diseases
Addressing the Current Limitations of AAV Gene Therapies
Examining the Legislative Landscape for Rare Disease Drug Development
How a Drug Setback Became a Patient Community’s Gain
Helping Regulators and Drug Developers Understand the Challenges of Living with Fabry Disease
Addressing Racial Disparities in a Rare Blood Cancer
How New Sequencing Technology Promises to Alter the Diagnostic Odyssey
Leveraging Technology to Empower Patients and Decentralize Clinical Trials
Equipping The Next-Generation Rare Disease Patient Advocate
How a Family Raced from Diagnosis to Experimental Gene Therapy in Three Years
How One Advocate Went from Losing His Hearing to Being Heard
Building a Pipeline of Therapies to Treat Rare Mineralization Disorders
Advancing an Oral Alternative to Infused and Injected Therapies for HAE
Embracing the Promise of Patient-Centered Biotechnology
Learning to Live with Loss
Using Cryptocurrency to Tap into the Wisdom of Crowds
A Therapy for a Rare Neurodegenerative Disease Moves Towards Regulatory Review
Transforming the Treatment of Neuromuscular Diseases with Next-Gen Oligonucleotides
Teaching the Immune System to Let Medicines Do Their Job
Designing Clinical Trials with the Patient in Mind
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