Note: This story was updated Aug. 10, 2022, to clarify the U.S. Food and Drug Administration advisory committee voted there is no substantial evidence supporting AMX0035 efficacy. When added to standard of care, both the experimental oral therapy AMX0035 and the U.S.-approved Radicava ORS oral suspension provide comparable…

The Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database, the largest collection of de-identified records on amyotrophic lateral sclerosis (ALS) patients in clinical trials, has been updated with records of participants in three Cytokinetics-sponsored studies. Newly added findings derive from a decade of trials involving nearly 600 ALS patients. Those…

There I was, just living each day and minding my own business. I was following all the guidelines from the U.S. Centers for Disease Control and Prevention and wearing a mask everywhere I went, when all of a sudden, BAM! The COVID-19 virus found me. What followed were…

Early use of Clene Nanomedicine’s experimental therapy CNM-Au8 reduces the risk of death by 70% in people with amyotrophic lateral sclerosis (ALS), compared with patients who experienced a nine-month delay in starting treatment. These are the findings of an updated analysis of the RESCUE-ALS Phase 2 trial…

Nonprofits, scientists, governmental organizations, and the rare disease drug development industry have long cited 7,000 as the average number of rare diseases in the world. But a new analysis shows there are as many as 10,867 rare diseases globally. And that…

ABBV-CLS-7262, an experimental compound being developed by Calico Life Sciences and AbbVie for amyotrophic lateral sclerosis (ALS), will likely serve as the sixth therapy in the multi-regimen HEALEY ALS platform trial. HEALEY (NCT04297683) is testing the potential of several ALS treatment candidates simultaneously with a goal of speeding…

Corestem is still enrolling participants in its Phase 3 clinical trial of NeuroNata-R (lenzumestrocel), a stem cell therapy that is conditionally approved to treat amyotrophic lateral sclerosis (ALS) in South Korea. The trial, ALSummit (NCT04745299), is evaluating the safety of repeat NeuroNata-R injections and how well…

A U.S. Food and Drug Administration (FDA) advisory committee will meet  Sept. 7 to discuss the approval of AMX0035 for the treatment of amyotrophic lateral sclerosis (ALS), Amylyx Pharmaceuticals announced. While the application was granted priority review earlier this year, with a final decision expected by June…

Using the approved treatment Radicava (edaravone) in a real-world setting resulted in a similar safety profile as that reported in clinical trials, with no new safety signals identified, according to a new report. The analysis involved more than 5,000 people with amyotrophic lateral sclerosis (ALS) who received the…

When my 12-year-old son, Isaac, went to mow the grass last week, he noticed that one of the tires had come off the rim. My husband, Todd, purchased the zero-turn lawn mower nine years ago, a few years after his ALS diagnosis, when his arms were too weak to…