Patient Empowerment Program: A Rare Disease Podcast

n-Lorem Foundation (Dr. Stan Crooke, Amy Williford, Kim Butler, Andrew Serrano, Jon Magnuson, and Kira Dineen)

Silencing ALS with Neil Shneider

AUG 5, 202629 MIN

Description

Silence ALS is an initiative that brings together Columbia University and n-Lorem to support the discovery, development, and treatment of individuals living with nano-rare genetic forms of amyotrophic lateral sclerosis (ALS) through personalized antisense oligonucleotide (ASO) medicines. Learn more about this initiative and one of its co-founders, leading ALS physician-scientist Dr. Neil Shneider in this episode of the Patient Empowerment Program podcast. On this episode we discuss: 4:00 – Developing tools to discuss with patients about the real and scary outcomes of ALS, and changing the course of a neurogenerative and fatal disease 6:52 – Experimental ASO treatments for genetic forms of ALS 9:30 – The creation of Silence ALS to treat extremely rare forms of genetically caused ALS like CHCHD10 and TARDBP and moving the collaboration forward 12:36 – Serving present day and future patient populations with the Silence ALS initiative 17:30 – Learnings gained from treated nano-rare ALS patients may translate to broader ALS groups 22:33 – To ALS patients and families, hope is powerful and makes a difference   Links:  Donate - https://www.nlorem.org/donate/  NRCP26 - https://www.nlorem.org/nano-rare-patient-colloquium-2026/  Hongene - https://www.hongene.com/