Aperture advances RNA-targeting treatment for ALS

Aperture Therapeutics said it is advancing its RNA-targeting therapy APRTX-003 as a potential treatment for amyotrophic lateral sclerosis (ALS). The therapy, currently in preclinical development, is designed to reduce the production of matrix metalloproteinase-9 (MMP-9) by targeting its template RNA molecule. When overactive, this enzyme may lead to…

“I feel like crying,” I wrote in my journal after a second caregiver decided to leave in December. “I can do 18 hours a day on duty, but I need to sleep sometimes.” For a few months, we had had a reliable team of nighttime caregivers for my husband,…

Tanabe Pharma plans to establish a new U.S.-based company to hold the global rights to Radicava and Radicava ORS, two edaravone formulations approved to treat amyotrophic lateral sclerosis (ALS), which Shionogi plans to acquire and operate as a wholly owned subsidiary. “We are pleased to welcome Radicava…

My recent holiday season was overall fun and festive. It did, however, offer a pointed lesson in living with ALS. It began one morning as I was getting ready for a special lunch with relatives. I sat on a folding chair, struggling to put on a sock, and from…

Both people living with amyotrophic lateral sclerosis (ALS) and their caregivers benefit most from flexible, personalized psychological support that combines emotional care and open dialogue, a new study from Germany suggests. Critical, the researchers say, is to create a framework that can be easily adjusted to a patient and…

QRL-101, an experimental oral small molecule being developed by Quralis for amyotrophic lateral sclerosis (ALS), was found in an early clinical trial to engage its intended biological target and reduce nerve cell overactivity in people with ALS. These results come from a proof-of-mechanism Phase 1 clinical study…

Health Canada has cleared the way for people with amyotrophic lateral sclerosis (ALS) living in Canada to join an ongoing clinical trial evaluating Coya Therapeutics’ therapy candidate COYA 302. The Canadian regulatory agency issued a “no objection” letter that will allow the developer to start enrolling adults in…

Some people with amyotrophic lateral sclerosis (ALS) who received Qalsody (tofersen) in the VALOR trial experienced improvements in physical function, lung function, and muscle strength over about three years of treatment, a new analysis found. Gains were observed more often among those who started treatment in the main…

Dear Dagmar, I know the news is still fresh. There are so many things I want to tell you, but I’ll keep this letter focused. That’s mainly because I know being contacted by your future self can be startling enough, but added to that, being told you have ALS…

I feel a sense of accomplishment as this year ends. We made it. My husband, Todd, has hung on, with much effort to keep his lungs clear and filled with air, and we’ve kept persevering even as his ALS keeps progressing. Todd hasn’t had his breathing tested since December…