Eleanor and Lou Gehrig ALS Center at Columbia University

06/06/2025

https://www.openaccessgovernment.org/experimental-drug-ulefnersen-offers-unprecedented-hope-for-rare-form-of-als/193097/

An experimental drug known as ulefnersen has shown remarkable efficacy in treating a rare, aggressive form of ALS

05/10/2024

https://alsnewstoday.com/news/columbia-gets-15m-design-gene-therapies-rare-als-forms/

Columbia researchers will use a $15 million grant to design tailored gene therapies for nine people with ultra-rare forms of ALS.

03/22/2024

Silence ALS is a collaborative effort between Columbia University's Eleanor and Lou Gehrig ALS Center at Columbia University and the n-Lorem Foundation.

The research aims to help an underserved portion of ALS patients by creating customized therapies.

05/16/2023

FDA Grants Accelerated Approval for QALSODY (tofersen) for SOD1-ALS

Though limited to patients with a rare genetic mutation, the newest drug for ALS could be most effective when given before symptoms emerge.

09/03/2022

Anna's Story of Hope and Help

In this episode, Stan talks with Sonja and Dr. Neil Shneider, associate professor of motor neuron disorders at Columbia Medical School, about Sonja’s daughter, Anna. She is 17 years old and suffers an aggressive, fatal form of ALS.

Quickly and easily listen and subscribe to Patient Empowerment Program: A Rare Disease Podcast for free in your podcast app of choice.

07/15/2022

We are so pleased and proud to be part of this extraordinary experience. What a strong and courageous young woman you are, Anna.

+-+-+-+-+ BREAKING NEWS +-+-+-+-+Hallo zusammen, nachdem ich nun die offizielle Freigabe habe, möchte ich Euch das nicht länger vorenthalten. Neben Tofersen ...

05/13/2022

https://www.cuimc.columbia.edu/news/silence-als-develop-personalized-therapies-people-rare-genetic-forms-als

A new initiative led by Columbia University and the n-Lorem Foundation will develop personalized therapies for individuals with "nano-rare" genetic forms of ALS and treat them for free, for life.

05/04/2022

https://www.nlorem.org/n-lorem-foundation-and-columbia-university-establish-silence-als-initiative/

n-Lorem Foundation established to charitably provide antisense medicines to treat patients with ultra-rare diseases

02/25/2022

An abstract is unavailable.

02/14/2022

FUSION study now active in Europe!

Reading Time: 4 minutes Researchers at King’s College London led by Professor Christopher Shaw, are the first in Europe to embark on a clinical trial targeting a specific genetic form of ALS caused by mutations in the fused in sarcoma (FUS) gene. What is FUS-ALS? Mutations in FUS were first linked...

01/24/2022

Compassionate use of an antisense drug first used at Columbia to treat an aggressive type of ALS caused by mutations in the FUS gene markedly reduces toxic proteins in the nervous system.

01/24/2022

Very proud of my team. Our paper on FUS-ALS and the antisense therapeutic jacifusen (ION363) was published today in Nature Medicine. Read it here: https://rdcu.be/cFCqV

NIH-funded preclinical study identifies potential therapeutic approach to treat ALS.