“One hundred bottles of beer on the wall, One hundred bottles of beer! Take one down, Pass it around, Ninety-nine bottles of beer on the wall!” That ditty was invariably sung, in unison, on all bus rides associated…
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 sclerosis (ALS). This follows a recent announcement that a similar application will be submitted to Health…
It all began as one of those typical online interactions when someone reaches out and asks for help. A gentleman contacted me via Facebook, explaining that his friend was recently diagnosed with ALS. He wanted to ask me a few questions. I noted that we were connected as “friends of…
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 gene editing tools and advance its pipeline of therapies for neurodegenerative diseases, such as…
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 York Yankees first baseman whose career was cut short by amyotrophic lateral sclerosis (ALS). The inaugural “Lou Gehrig…
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 diagnose the condition, and open the way to treatment. Launched by Invitae,…
I think a lot about how a parent’s ALS affects kids. Recently, an online friend lost her husband to ALS. She has small children and has been posting about their grief and the tender questions her kids have asked. It’s difficult enough for an adult to process the pain of…
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 are genes or genetic variants that can increase or reduce the severity of a condition without necessarily…
“I can see clearly now the rain is gone. I can see all obstacles in my way. Gone are the dark clouds that had me blind.” Last week, the Jimmy Cliff version of that Johnny Nash composition played over…
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 disease. The trial (NCT04768972) will enroll up to 64 people, ranging in age from 12 to 65,…
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