Prion Disease Clinical Trial NN112

Dr. Gregg Day and Drs. Sonia Vallabh and Eric Minikel discuss scientific insights and the future of prion disease treatment, highlighting the importance of early diagnosis, personalized medicine, and hope for affected families.

Learn about the clinical trial.

Learn more about the Prion Alliance.

Show transcript:

Dr. Gregg Day:

This is Gregg Day with Neurology Minute. I've just been speaking with Eric Minikel and Sonia Vallabh, a husband and wife team at the heart of the PRiSM trial, a first-in-human study of a prion protein-lowering, divalent, small-interfering RNA for patients with symptomatic prion disease. Eric and Sonia, could you provide us with a brief overview of the PRiSM trial and what this first-in-human study seeks to accomplish?

Dr. Eric Minikel:

The PRiSM trial is testing a short interfering RNA designed to bind the RNA that encodes the prion protein. That is the protein that causes Prion disease. We are at the heart of what causes this disease. Through doing this, we hope to make prion disease a treatable and preventable condition. We both want to stabilize symptomatic patients and prevent the disease in people who are at genetic risk. This is a personal mission for us. Sonia is a carrier of a prion disease mutation that she inherited from her mother who died of prion disease, and we, along the way, aspire to be a different kind of sponsor. We want to create our own clinical data that are shareable learnings for the entire field.

Dr. Gregg Day:

This is Gregg Day with Neurology Minute. Thanks for listening.

Denne episoden er hentet fra en åpen RSS-feed og er ikke publisert av Podme. Den kan derfor inneholde annonser.

Episoder(2051)

Facial Pain Series - Part 1

In part one of this series, Dr. Derek Stitt discusses trigeminal neuralgia and, more specifically, how to make sure that's actually the diagnosis.

9 Nov 3min

October 26, 2026 Capitol Hill Report: Conrad 30

In this episode, Dr. Jose Posas reviews the Capitol Hill Report from October 26th, discussing international medical graduates, the Conrad 30 program, and the AAN's efforts to strengthen visa pathways ...

6 Nov 2min

Youth Football and the Epidemic of Pediatric Brain Injury - Part 2

In part two of this series, Dr. Dara Albert and Isaac Thorman discuss guidance for parents weighing whether to let their children play tackle football. Show citation: Thorman IB, Feldstein E, Sackno...

5 Nov 2min

Youth Football and the Epidemic of Pediatric Brain Injury - Part 1

In part one of this series, Dr. Dara Albert and Isaac Thorman discuss football as a major contributor to pediatric traumatic brain injury and its association with subsequent head injuries. Show citat...

4 Nov 1min

Acute Optic Neuropathy in Older Adults

Dr. Justin Abbatemarco discusses an article that helps us distinguish between the causes of acute optic neuropathy. Show citation: Tisavipat N, Stiebel-Kalish H, Palevski D, et al. Acute Optic Neuro...

3 Nov 3min

Efficacy and Safety of Gefurulimab in Generalized Myasthenia Gravis

Dr. Aaron Zelikovich discusses how gefurulimab may offer an effective and convenient treatment option for adults with generalized myasthenia gravis. Show citation: Gwathmey KG, Saccà F, Howard JF Jr, ...

2 Nov 3min

Trends in Emergent Interfacility Ischemic Stroke Transfers

Dr. Andy Southerland and Dr. Matthew Gusler discuss how emergent interfacility transfers for ischemic stroke have changed and the factors associated with which patients are transferred. Show citation...

30 Okt 2min

Clinical Reasoning: Rapidly Progressive Cranial Neuropathies, Weakness, and Ataxia in a Patient With MS

Dr. Zohaib Siddiqi and Dr. Michel Toledano discuss the diagnosis and management of infectious rhombencephalitis in patients receiving immunomodulatory therapy. Show citation: Trentadue T, Martinez Sos...

29 Okt 3min