Inês Martins, PhD,  managing science editor—

The U.S. Food and Drug Administration (FDA) has granted conditional approval to Biogen’s tofersen, now named Qalsody, for the treatment of amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The decision, which marks the first conditional approval for ALS in the country, comes about eight…

AC Immune is planning to advance its investigational anti-TDP-43 antibody into clinical testing for neurodegenerative diseases, like amyotrophic lateral sclerosis (ALS), in which TPD-43 protein aggregates play a major role in brain damage. The company announced the start of preclinical studies into this first-in-class antibody. This work will…

The first platform trial for amyotrophic lateral sclerosis (ALS), designed to assess multiple treatment candidates simultaneously with a goal of accelerating the development of ALS therapies, has enrolled its first patients. The HEALEY ALS Platform Trial (NCT04297683) will start by evaluating UCB’s…

Kadimastem’s cell therapy candidate, AstroRx, significantly lowers the rate of disease progression in people with amyotrophic lateral sclerosis (ALS) for at least three months after treatment, updated findings from the company’s Phase 1/2 clinical trial show. The company already had reported promising…

The clear, colorless liquid that fills and surrounds the brain and spinal cord in people with amyotrophic lateral sclerosis (ALS) seems to induce early neurodegeneration events in healthy motor neurons, a study has found. When motor neurons were continuously cultured with cerebrospinal fluid  from ALS patients, cells showed…

A new mutation in the FUS gene, which results in a shorter version of the FUS protein that cannot enter the cell nucleus to work as it should, may cause an aggressive from of juvenile amyotrophic lateral sclerosis (ALS), the case of an adolescent girl shows. The…

Tofersen, Biogen’s investigational therapy for people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, was safe and generally well-tolerated over three months, and appeared to lower SOD1 protein levels in the central nervous system, a Phase 1/2 clinical trial has found. There…

Amyotrophic lateral sclerosis (ALS) patients who never regularly engaged in sports or physical activity have more extensive metabolic changes in the brain that likely help them to better cope with the disease’s neurodegenerative processes, a study from Europe suggests. These changes can reflect either greater metabolic activity in a…

Mutations in the UBQLN2 gene, known to cause amyotrophic lateral sclerosis (ALS), promote the buildup of toxic waste in brain cells by preventing the normal function of two cellular degradation mechanisms, a study has found. In addition to its known role in the proteasome, a mechanism used…

Denali Therapeutics and Sanofi announced a pause in clinical testing of their small molecule inhibitor DNL747 in favor of work on a possibly more effective compound, DNL788, for neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), Alzheimer’s, and multiple sclerosis. DNL747 was seen to be safe…