Repeated muscle injections with Engensis (VM202), Helixmith’s investigational non-viral gene therapy, were generally safe and well-tolerated in people with amyotrophic lateral sclerosis (ALS), according to top-line data from a Phase 2a clinical trial. While the sample size was too small to determine the therapy’s efficacy, muscle biopsies were…

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…

To help expand research into the treatment and cure of amyotrophic lateral sclerosis (ALS), the Blazeman Foundation for ALS has donated $800,000 to the University of Maryland School of Medicine (UMSOM). The funds will be used to boost research at the University of Maryland Brain and Tissue Bank,…

Treating patients with their own immune regulatory T-cells (Tregs), in combination with IL-2 — a protein that boosts the cells’ immunosuppressive function and survival — is generally safe and shows potential to slow disease progression in people with amyotrophic lateral sclerosis (ALS). That’s according to a small Phase…

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…

The European Medicines Agency (EMA) has agreed to review an application seeking the conditional approval of AB Science’s masitinib, now Alsitek, for the treatment of amyotrophic lateral sclerosis (ALS). Conditional approval is granted to a medication whose immediate availability fulfills an unmet medical need, when its preliminary benefits are…

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 clinical trial is evaluating whether a new, implantable brain computer interface (BCI) is able to decode brain signals — ones research has shown are related to tasks relying on the hands or speech — and transform them into actions for people with neurological disease or injury. Up to 15…

Whole-genome sequencing, a type of genetic screening that examines a person’s complete DNA sequence, may be a promising way to identify disease-causing mutations in people with amyotrophic lateral sclerosis (ALS), according to a study of Italian patients. Disease-associated mutations were found in more than a quarter of 1,043 Italian…