New Deal Helps Move Gene Therapy ET-101 Closer to Clinical Testing

Eikonoklastes Therapeutics and Forge Biologics have announced a new partnership to advance the development of ET-101, an experimental gene therapy for amyotrophic lateral sclerosis (ALS), toward clinical trial testing. Eikonoklastes acquired the rights to ET-101 from the University of California San Diego earlier this year. Under…

“I don’t know what that is.” “I don’t know how to use that.” These are a couple of the statements that ALS patients do not want to hear when a medical professional walks into the room. As a matter of fact, no patient wants to hear them. Having spent…

The COURAGE-ALS Phase 3 clinical trial, testing Cytokinetic’s experimental therapy reldesemtiv in people with amyotrophic lateral sclerosis (ALS), is expected to enroll patients who are most likely to benefit from the potential treatment. That’s according to a new analysis of data from an earlier Phase 2 study of reldesemtiv called…

I filled the last page of another journal and then reread some of the entries over the last year. I had written about the fear I felt when my husband, Todd, who has ALS, stopped breathing and I had to restart his lungs; the feelings of sadness…

Higher levels of inflammatory immune cells called effector T-cells at the time of diagnosis are associated with faster disease progression and worse survival among people with amyotrophic lateral sclerosis (ALS), a new study indicates. In turn, greater numbers of regulatory T-cells, which work to dampen excessive immune responses, were…

BrainEver has joined in a long-term partnership with 3P Biopharmaceuticals to advance in development its lab-made form of the human protein Engrailed 1 (EN1) as a potential treatment of amyotrophic lateral sclerosis (ALS) and like conditions. Under the terms of the collaboration, 3P will be in charge of…

I’ll admit that because I live with ALS, some of my past “Human vs. Automation” experiences have been, um, less than perfect. Touchpads that open automated doors don’t always work for me, and voice activation software doesn’t recognize my commands. But now, I have a new sense of…

The European Medicines Agency (EMA) has agreed to review an application seeking the approval of tofersen in treating amyotrophic lateral sclerosis (ALS) caused by mutations in the SOD1 gene, the therapy’s developer, Biogen, reported. “Today’s announcement is an important milestone for the ALS community in Europe where there is a tremendous need…

EpicentRx has been awarded funding from FightMND to study how well RRx-001, its lead small molecule, may protect nerve cells against damage caused by amyotrophic lateral sclerosis (ALS), a disease also known as motor neuron disease (MND). “We are very pleased to receive this support from FightMND…

Researchers have developed a brain-machine interface (BMI) that could someday facilitate communication for people who have lost their ability to speak, including those with amyotrophic lateral sclerosis (ALS). After being trained to recognize certain patterns of nerve cell activity, the BMI could accurately predict the words a person with…