The Committee for Medicinal Products for Human Use (CHMP) has issued an opinion opposing Amylyx Pharmaceuticals’ bid for conditional European Union approval of AMX0035, its oral treatment to slow the progression of amyotrophic lateral sclerosis (ALS). The negative recommendation from the advisory committee, an arm of the…

Ever since my husband Todd’s ALS led to his paralysis, it has been hard for him to get comfortable. This is especially true now that he wears a mask nearly 24/7 for noninvasive ventilation support. For years, I’ve jumped into action when he calls me to swat a…

The first text message my late husband, Jeff, ever sent me was just one word. We’d had our first date the previous evening, and our dinner at a sidewalk restaurant on a mild autumn evening in Alexandria, Virginia, was full of laughter. I was happy to see his text the…

BenevolentAI is advancing BEN-34712, its experimental oral therapy for amyotrophic lateral sclerosis (ALS), to late-stage preclinical studies. The studies are expected to support a future investigational new drug (IND) application, a formal request to the U.S. Food and Drug Administration (FDA) to test a potential new treatment in clinical…

QurAlis soon expects to open its ongoing Phase 1 clinical trial testing QRL-201 in people with amyotrophic lateral sclerosis (ALS) at sites in various European Union countries, including Germany, Belgium, the Netherlands, and Ireland. The company’s announcement comes after these countries reviewed and accepted an application for…

Today marks the third annual Major League Baseball (MLB) Lou Gehrig Day, set aside to honor the legacy of the famed New York Yankees first baseman whose career was shortened by amyotrophic lateral sclerosis (ALS). For the first time since the event began, all 30 MLB teams have…

A committee of the European Medicines Agency (EMA) is leaning toward not recommending a conditional approval of Amylyx Pharmaceuticals’ AMX0035 for treating amyotrophic lateral sclerosis (ALS) in the European Union. The drug is already marketed in the U.S. under the name Relyvrio (sodium phenylbutyrate and taurursodiol) and…

WVE-004, Wave Life Sciences‘ investigational treatment for amyotrophic lateral sclerosis (ALS), significantly reduces toxic proteins associated with C9orf72 genetic mutations, but that doesn’t seem to translate into functional status gains in patients. Based on findings from the Phase 1b/2a FOCUS-C9 trial (NCT04931862), the company has decided to discontinue developing…

Mary Kate Karam and Brian Jeansonne at Jazz Fest 2023, watching Mumford & Sons, Trombone Shorty, and John Batiste. Incredibly hot, but incredibly fun! (Photo courtesy of Mary Kate Karam) This is Mary Kate Karam’s story: Brian Jeansonne, a father of five, was diagnosed…

“You should burn the beaver,” my husband, Todd, said. Over the last several years, since he has been paralyzed due to ALS, he has given me a lot of advice on various house projects, but that was not something I ever anticipated hearing. Last weekend, I was walking with…