Marisa Wexler, MS, senior science writer —

In a reversal, an advisory committee of the U.S. Food and Drug Administration (FDA) now says that the current evidence of efficacy of Amylyx Pharmaceuticals’ AMX0035 is sufficient to support its approval for amyotrophic lateral sclerosis (ALS). The committee voted 7–2 in favor of AMX0035, Amylyx announced…

Higher blood levels of antibodies against the viral protein HERV-K ENV are associated with a better survival rate and improved outcomes in people with amyotrophic lateral sclerosis (ALS), a new study shows. These findings further support the therapeutic potential of GNK301, an experimental antibody against HERV-K ENV being…

Altered forms of the SOD1 protein are found in the spinal cord nerve cells of people with all types of amyotrophic lateral sclerosis (ALS), not just those with mutations in the gene providing instructions for making the protein, a study reported. “The results suggest this abnormal protein contributes to cell…

Treatment with gene therapy candidate SynCav1 delayed disease onset and extended survival in a mouse model of amyotrophic lateral sclerosis (ALS), according to a new study. The experimental therapy aims to improve the survival of motor neurons in people with ALS irrespective of the underlying cause. “These data suggest that…

Treatment with terazosin, a medication approved to treat high blood pressure and enlarged prostate, was found to protect motor neurons and extend survival in animal models of amyotrophic lateral sclerosis (ALS) in a new study. “Our work shows that terazosin is protective of motor neuron cell death in multiple…

A mutation in the STMN2 gene that consists of an excessive repeat of two nucleotides, the building blocks of DNA, is not associated with the greater risk of amyotrophic lateral sclerosis (ALS), a new study reported. Its findings contradict previous research suggesting a link between this particular mutation and ALS. “Although the…

TDP-43 protein abnormalities characteristic of most amyotrophic lateral sclerosis (ALS) cases contribute to the loss of motor neurons mostly by limiting the function of a protein called stathmin-2 (STMN2), a study reported. Results indicate that boosting STMN2 levels may be a useful approach in treating ALS, according to its…

The U.S. Food and Drug Administration (FDA) has agreed to review an application from Biogen for approval of the company’s experimental therapy tofersen to treat amyotrophic lateral sclerosis (ALS) associated with mutations in the SOD1 gene. The FDA has granted the application accelerated review and a decision…

A viral protein called HERV-K ENV is often detectable in the fluid around the brains of people with amyotrophic lateral sclerosis (ALS), and is toxic to nerve cells, a new study shows. However, blocking this protein with GeNeuro‘s experimental antibody GNK301 reduced those toxic effects in cell…

Amyotrophic lateral sclerosis (ALS) type 4 — a juvenile and slowly progressive form of the neurological disease, called ALS4 — is driven by abnormal mechanisms in both the central nervous system and the immune system, a new study reports. In particular, patients with this condition have increased levels of inflammatory…