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…

Stem cells derived from fat tissue significantly improved motor function and delayed disease onset by protecting motor neurons in a mouse model of amyotrophic lateral sclerosis (ALS), a study shows. The study, “Adipose derived stem cells protect motor neurons and reduce glial activation in both in…

AcuraStem has been awarded a $1 million grant from the U.S. Department of Defense (DOD) to advance AS-202, a potentially disease-modifying therapy for sporadic amyotrophic lateral sclerosis (ALS), according to a press release. The candidate therapy is designed to increase the number of lysosomes — cell compartments…

PharmaTher has filed an application with the U.S. Food and Drug Administration (FDA) requesting that ketamine be designated an orphan drug as a potential treatment of amyotrophic lateral sclerosis (ALS). Orphan drug status is granted by the FDA to encourage the development of therapies for conditions that affect fewer…

Today, the National ALS Registry is joining the Major League Baseball to celebrate Lou Gehrig’s life and legacy and to raise greater awareness of amyotrophic lateral sclerosis (ALS). This year’s inaugural “Lou Gehrig Day” on June 2 honors the famous New York Yankees baseball player whose career…

Everyone likes being noticed — for the right things, of course. I like it when others recognize my unique talents and skills or a job well done. I don’t like being noticed because I have to use a rollator to help me walk. Or, because I move more slowly than…

People living with amyotrophic lateral sclerosis (ALS) recently shared their views on the importance of new therapies and incremental gains, and the risks they are willing to take with U.S. Food and Drug Administration (FDA) and industry officials in a virtual meeting hosted by The ALS Association. The…