FDA names RAG-17, targeting ALS gene mutations, an orphan drug

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…

While in town the other day, I ran into an old friend. My husband, Todd, and I had gotten to know her and her husband when they first started attending our church. We had invited them over for dinner along with a few other families. Then we met weekly with…

Note: This story was updated March 10, 2023, to correct a secondary headline to note the therapy was tracked for less than a year. The story also clarifies the timing of results from a Phase 3 clinical trial and that edaravone is believed to reduce oxidative stress. Radicava ORS, an…

Pridopidine, an oral small molecule, failed to meet its primary goal of improving physical function across amyotrophic lateral sclerosis (ALS) patients who took part in a Phase 2/3 clinical trial. Changes in secondary endpoints measuring muscle strength and respiratory function also did not significantly differ between people taking pridopidine…

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…