A previously unknown form of amyotrophic lateral sclerosis (ALS) — one with onset during childhood — is caused by mutations that alter the production of certain lipids (fat molecules), scientists report. “We found that a genetic form of the disease can also threaten children. Our results show for the first…

Cedars-Sinai Medical Center, in California, has been awarded an $11.99 million grant to support a clinical trial that will test specifically engineered neural progenitor cells as a potential therapy for amyotrophic lateral sclerosis (ALS). The work, funded by the California Institute for Regenerative Medicine, or CIRM, will build…

My husband, Todd, came down with a cold last week. At this stage of his ALS progression, any cold is life-threatening. His lungs fill up with mucus, and he needs me to push on his abdomen so he can cough it out. Todd had a sore throat and a runny…

In a response letter to The ALS Association, the U.S. Food and Drug Administration (FDA) has recognized the unmet therapeutic need of people with amyotrophic lateral sclerosis (ALS) and reaffirmed its commitment to the 2019 ALS clinical trial guidance. However, the agency did not provide any details about how it…

To help me get through the ups and downs of living with ALS, I’ve created several mental games. Perhaps they’ll help you, too. For example, when life around me isn’t cooperating, I imagine I’m in a driver’s ed training film. When my body isn’t following orders, I tell…

A gay 39-year-old entrepreneur from Mexico and a retired Jewish football player-turned-bank-CEO in the early stages of amyotrophic lateral sclerosis (ALS) come from different places and are in different stages of life. But through art, Octavio Molina and Ken Brenner have become fast friends, discussing life at its deepest…

CRISPR Therapeutics and Capsida Biotherapeutics have partnered to develop gene-editing therapies for familial amyotrophic lateral sclerosis (ALS) and Friedreich’s ataxia. Capsida specializes in designing virus-based means of delivering therapies to specific cells, while CRISPR’s expertise lies in gene-editing technology, particularly the CRISPR gene-editing system. “Bringing together…

Clene and its subsidiary Clene Nanomedicine are negotiating to increase their manufacturing capacity as they prepare to release findings — expected early next year — from a pivotal and enrolling Phase 3 trial of CNM-Au8 as an oral therapy aiming to slow amyotrophic lateral sclerosis (ALS) progression. Negotiations include the…

Levels of the protein tau found in the cerebrospinal fluid (CSF) — the transparent liquid surrounding the brain and spinal cord — can help in diagnosing amyotrophic lateral sclerosis (ALS), a study reported. Tau levels in ALS patients also correlated with a faster progression rate and poorer survival, suggesting this…

An investigational RNA molecule called an RNA aptamer halted the death of motor neurons and helped preserve motor function in a mouse model of amyotrophic lateral sclerosis (ALS). Further research is ongoing to confirm the findings in additional ALS models before testing it in humans, “but I’m optimistic that this…