The U.S. Food and Drug Administration (FDA) has given orphan drug designation to CB03, Zhimeng Biopharma’s small molecule treatment candidate for amyotrophic lateral sclerosis (ALS). CB03 is designed to bolster nerve cell health by regulating potassium channels on these cells, reducing the hyperexcitability that is damaging to…
People with amyotrophic lateral sclerosis (ALS) who are treated daily with 1,000 mg or higher doses of tauroursodeoxycholic acid (TUDCA) tend to live longer than those given standard care alone, according to an analysis of registry data. “In our ‘real-world’ study, patients who received TUDCA at the higher…
Back in middle school, I was taught that sound comprises three components: something to make the sound, something to transport the sound, and something to receive the sound. It’s been a while since I learned that, but I assume it’s still true. The principle reminds me of a related statement…
BrainStorm Cell Therapeutics is withdrawing the application requesting U.S. approval of its cell-based therapy NurOwn for the treatment of amyotrophic lateral sclerosis (ALS). The decision comes on the heels of a recent U.S. Food and Drug Administration (FDA) advisory committee meeting, in which experts ruled nearly unanimously…
After my husband, Todd, was diagnosed with ALS, I was overwhelmed by grief and filled with anxiety about the future. How was I going to manage as a single parent with two small children? How would they cope with the loss of a parent? What would I do…
Continuing its annual support, BluSky Restoration Contractors has raised $140,000 to advance research at the Miller Laboratory at Washington University School of Medicine in St. Louis, Missouri, for new and better amyotrophic lateral sclerosis (ALS) treatments. The funds were raised through the sixth annual Rakers Classic…
Treatment with Relyvrio (sodium phenylbutyrate and taurursodiol) in the CENTAUR trial significantly extended the median survival — by 10.4 months — of amyotrophic lateral sclerosis (ALS) patients compared with an historical patient control group, a post-hoc analysis of study data reported. This finding more than doubles the 4.8…
Yay, it’s October! Fall colors, pumpkin pie, Halloween, and my birthday month. Not only am I feeling “another year older, another year wiser,” I’m also grateful and appreciative that I’ve had 72 birthdays, even though I’m living with ALS. But reaching this particular milestone has me pondering the complicated…
The National Institutes of Health (NIH) has awarded about $45.1 million toward an expanded access program (EAP) of CNM-Au8, an oral therapy candidate for amyotrophic lateral sclerosis (ALS). The EAP, commonly referred to as compassionate use, will make CNM-Au8 available to U.S. patients who aren’t eligible for clinical…
An advisory committee of the European Medicines Agency has maintained its recommendation not to approve AMX0035 for amyotrophic lateral sclerosis (ALS). In its initial assessment issued in June, the Committee for Medicinal Products for Human Use (CHMP) said data from the CENTAUR Phase 2 trial (NCT03127514), which formed…
