Treatment with NeuroSense Therapeutics’ PrimeC was found to extend patients’ time without complications or death, and to lead to clinically meaningful effects on quality of life, among those with amyotrophic lateral sclerosis (ALS). The new data comes from the PARADIGM Phase 2b study (NCT05357950), a fully enrolled…
On Feb. 17, a party was thrown in Atlanta, Georgia, in celebration of ALS patients and their caregivers. It was a chance to promote awareness about the ongoing needs of the victims of this treacherous and unrelenting disease. A party for ALS? Why not? Like the Ice…
OrphAI Therapeutics’ experimental therapy AIT-101 has been awarded orphan drug status in the European Union for amyotrophic lateral sclerosis (ALS), following a similar designation granted in the U.S. last year. In Europe, orphan drug status is given to medicines designed to treat life-threatening or chronically debilitating conditions affecting…
Treatment with CNM-Au8 was shown to significantly prolong survival — relative to the natural history of the disease — among people with advanced amyotrophic lateral sclerosis (ALS) who took part in two expanded access programs (EAP), also known as compassionate use programs. The findings from these EAPs, which…
After years of living on the edge of life and death with my husband’s ALS, I sometimes wonder if my body has quit reacting to stress in a normal way. Trying to squeeze in lunch before I ran to town for our 18-year-old daughter’s doctor appointment, I reheated…
An advisory committee of the European Medicines Agency (EMA) has recommended that Biogen’s Qalsody (tofersen) be approved in the European Union under exceptional circumstances to treat adults with amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. The positive opinion from the Committee for Medicinal Products for Human Use,…
Last week, I received an email from a newly diagnosed ALS patient asking questions about rollators (walkers with wheels). “I think it’s time I begin using one,” he wrote, “but I don’t want to. What can I do instead?” I immediately felt empathy and sympathy for his…
Health Canada has issued a notice of noncompliance-withdrawal saying it won’t approve the oral therapy masitinib as an add-on treatment for amyotrophic lateral sclerosis (ALS). Its developer, AB Science, can submit a request for reconsideration within the next month, which it plans to do, it announced in…
ProJenX has been given the green light to expand its ongoing Phase 1 clinical trial of prosetin for amyotrophic lateral sclerosis (ALS) across the Atlantic Ocean to testing sites in Europe. The PRO-101 trial (NCT05279755), launched in 2022, is a three-part study evaluating prosetin’s safety, tolerability, and pharmacological…
After a great deal of introspection, I decided for this month’s column to reflect on my life as it stands now. I wanted to write about it without being too ominous and dreadful, but I don’t think that’s possible. My love for music prompted the headline, because within the…
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