RAG-17, an investigational therapy from Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA). Orphan drug status is given to medicines intended to treat life-threatening or chronically debilitating diseases that affect fewer than 200,000 individuals…

In a recent column, I described how I panicked following my ALS diagnosis. With the prognosis of having only two to three years to live, suddenly there weren’t enough hours in the day to do what I wanted to do. My mind hyperventilated with seemingly endless thought…

Amyotrophic lateral sclerosis (ALS) patients treated earlier with CNM-Au8 in a clinical trial experienced a significantly slower disease progression than those who started treatment nine months later. These latest findings from the RESCUE-ALS Phase 2 trial (NCT04098406) and its open-label extension (OLE) study (NCT05299658) add to earlier reports that…

Levels of the TDP-43 protein in spinal fluid show promise as biomarkers for amyotrophic lateral sclerosis (ALS) and may aid in diagnosing the neurological disease, according to a new review of several published studies. That review discovered that these protein levels are increased in…

When I was in my early teens, I was awakened by what sounded like a rushing wind, followed by the inability to move any part of my body. It was a frightening sensation, and worse was the feeling of someone standing over me with the notion of harming me. I…

Enrollment is still ongoing for a Phase 2 clinical trial testing the safety and efficacy of Corcept Therapeutics‘ cortisol modulator, called dazucorilant, in people with amyotrophic lateral sclerosis (ALS). The DAZALS trial (NCT05407324), launched in November 2022, is expected to enroll an estimated 198 adults with…

I enjoy reading motivational blogs written by people from all walks of life outside the ALS community. What they write not only inspires me, but also gives me lots of new ideas. However, one recent blog post had me fuming. The author described how she was transforming her life…

To help those who have lost their ability to speak easily, Georgia Southern University’s RiteCare Center for Communications Disorders again is recruiting students, faculty, and staff members to make recordings for submission to a voice bank. The center’s third annual Voice Drive, which runs through May 2, is…

Today I had a mammogram. We have a family history of certain cancers, so I’m careful to get recommended screenings. After losing my husband, Jeff, to ALS, I feel a bittersweet commitment to taking care of myself as he would want. Looking after my health after he’s gone,…

Kadimastem’s experimental cell-based therapy AstroRx was safe and significantly slowed disease progression in the first three months among people with amyotrophic lateral sclerosis (ALS), a Phase 1/2 clinical study shows. The findings are in line with initial data released by the company in 2020, with AstroRx more…