FDA Gives ET-101, Gene Therapy for ALS, Orphan Drug Status

ET-101, Eikonoklastes Therapeutics’ experimental gene therapy for 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 investigational therapies with the potential to significantly benefit people with life-threatening or chronically debilitating diseases that affect…

The Lifestyle Healing Institute (LHI), which uses a complementary and alternative medicine approach to care, is touting the success of an amyotrophic lateral sclerosis (ALS) patient whose mobility and quality of life are said to have improved following treatment. The case of Tod, a 60-year-old patient, demonstrates that…

My late husband, Jeff, had a motorized scooter that gave us the freedom to move and travel as his ALS progressed during the summer of 2019, our first full summer living with the condition. The scooter was one of the first things Jeff ordered from Amazon as his…

“Find the victory in this.” For nearly three decades, I’ve carried these five provocative words in my mind. Like my life, their meaning to me has evolved over time. I first heard them spoken at the funeral of a young woman, and the comment was directed to her 15-year-old son,…

The U.S. Food and Drug Administration (FDA) notified Brainstorm Cell Therapeutics that it will not accept for review a company application asking that its cell-based therapy NurOwn be approved as a treatment for amyotrophic lateral sclerosis (ALS). The FDA decision, in the form of a refusal to file…

I sometimes dream my husband, Todd, is calling for me at night. I wake and check the time. If it’s after 5 a.m., I know his nighttime caregiver has left, so I check on him to see if he needs to be turned, have a limb adjusted, or an itch…

Radicava Oral Suspension, an oral formulation of edaravone, has been approved in Canada for treating amyotrophic lateral sclerosis (ALS). Mitsubishi Tanabe Pharma Canada (MTP-CA), the subsidiary of Mitsubishi Tanabe Pharma America that markets Radicava in Canada, has announced plans to make the oral formula available to patients…

The investigational cell-based therapy NurOwn may slow disease progression in people with amyotrophic lateral sclerosis (ALS) who have less severe disease, according to analyses from a Phase 3 trial. Researchers’ analyses excluded patients with the lowest scores on the ALS Functional Rating Scale–Revised (ALSFRS-R) or in individual…

A question I’m often asked by newly diagnosed ALS patients is, “What do you think is the one thing that helped you adjust so well to living with ALS?” My answer is always the same: There’s no one thing; it’s the synergy of many strategies and habits that have…

Early declines in neurofilament light chain (NfL), a biomarker of nerve cell damage, after treatment with tofersen may predict a slower disease progression over time in people with SOD1-associated amyotrophic lateral sclerosis (ALS). Patients who received tofersen early also experienced a significantly slower worsening of clinical function than…