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April 16, 2021April 16, 2021

EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US

News, syndicated

The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to help adults with a rare disease pursue personal goals through training and education. For a ... Read more

April 15, 2021April 15, 2021

US Students Affected by ALS Can Apply for Scholarships Until May 24

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The ALS Association is now accepting applications for the Jane Calmes ALS Scholarship Fund, aimed at supporting U.S. students whose lives are affected financially as well as personally by amyotrophic ... Read more

April 14, 2021April 14, 2021

Amylyx to Seek AMX0035’s Approval in Europe

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Amylyx Pharmaceuticals is planning to file an application by the end of this year requesting that the European Medicines Agency (EMA) approve AMX0035 as an oral treatment for amyotrophic lateral ... Read more

April 13, 2021April 16, 2021

Scribe Raises $100M to Advance CRISPR as Means of Treating ALS

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Note: This story was updated on April 16, 2021, to note that Scribe’s founding year was 2018, not 2017. Scribe Therapeutics has raised $100 million in funding to develop CRISPR-based ... Read more

April 12, 2021April 12, 2021

It’s Official: Each June 2 Will Be ‘Lou Gehrig Day’ for Major League Baseball

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After a protracted campaign to make it happen, Major League Baseball (MLB) agreed to set aside each June 2 to pay tribute to the late Lou Gehrig, the legendary New ... Read more

April 9, 2021April 9, 2021

Invitae Opens Genetic Testing to Adults With ALS Symptoms or Family Risk

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People at risk or suspected of having some of the most common neurodegenerative disorders, including amyotrophic lateral sclerosis (ALS), may now access a genetic test at no charge that could ... Read more

April 8, 2021April 8, 2021

Maze Therapeutics Advances Potential Gene Therapy Involving ATXN2

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Maze Therapeutics is advancing the development of a lead gene therapy candidate for amyotrophic lateral sclerosis (ALS), which works by suppressing the activity of a potent genetic modifier called ATXN2. Genetic modifiers ... Read more

April 7, 2021April 7, 2021

Ionis Opening Phase 3 Trial of ION363, Antisense Therapy for FUS-ALS

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Ionis Pharmaceuticals is opening a Phase 3 safety and efficacy trial of ION363 (jacifusen) in amyotrophic lateral sclerosis (ALS) patients with confirmed mutations in the FUS gene, a known cause of juvenile-onset ... Read more

April 6, 2021April 6, 2021

NIH Grant Supports Research Into FUS Mutations, Protein Toxicity

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The National Institutes of Health (NIH) has awarded a $450,000 grant to a university research team to explore mutations in the FUS gene that contribute to amyotrophic lateral sclerosis (ALS), ... Read more

April 5, 2021April 6, 2021

ALS-linked Mutations Surprising Common in Absence of Family History

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A higher than expected proportion of amyotrophic lateral sclerosis (ALS) patients — most without a family history of ALS — had mutations in genes associated with the disease, according to ... Read more

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