SOD1

The enzyme superoxide dismutase 1 (SOD1) forms clumps inside the cells of some people with sporadic amyotrophic lateral sclerosis (ALS), making them more vulnerable to DNA damage — which may contribute to the neurodegeneration that marks the disease, an early study suggests. In other patients, SOD1 is transported…

Biogen’s investigational therapy tofersen can significantly reduce toxic levels of SOD1 protein and may slow disability progression in people with familial amyotrophic lateral sclerosis (ALS) caused by SOD1 mutations, interim study results show. The positive data from a Phase 1/2 clinical trial support advancing the experimental compound into Phase 3…

Masitinib, the lead experimental medicine being developed by AB Science, can modulate the activity of different cell types known to be involved in inflammation and nerve degeneration in amyotrophic lateral sclerosis (ALS), according to results from a preclinical study. The findings were discussed in a presentation, titled “Post-paralysis treatment with…

Stem cell therapy using a certain type of stem cell called human mesenchymal stromal cells (hMSCs) may have the potential to improve outcomes and prevent progression of familial amyotrophic lateral sclerosis (ALS), a mouse study suggests. Repeated administrations of these stem cells in the spinal cord prolonged the lifespan…

Small nerve injury may be the trigger for early manifestation or faster progression of amyotrophic lateral sclerosis (ALS) motor symptoms in the presence of disease-associated genetic contributing factors, a study with rats suggests. The study, “Mutant SOD1 prevents normal functional recovery through enhanced glial activation and loss…

Suppressing an enzyme known as MAP4K4 extended the survival of motor neurons collected from mice and patients with amyotrophic lateral sclerosis (ALS), and decreased the accumulation of toxic proteins associated with the disease, a study reports. These findings from the study, titled “MAP4K4 Activation Mediates Motor…

Biogen has decided to exercise its option to acquire from Ionis Pharmaceuticals the exclusive rights to develop and commercialize BIIB067 as a potential treatment for amyotrophic lateral sclerosis (ALS). The decision to license the investigational therapy is covered under the terms of a collaborative agreement between the two companies…

Sensory nerves carrying mutations in the TDP-43 or SOD1 genes — associated with the development of a…

SOD1 gene mutations can contribute differently to the degenerative process of nerve cells involved in the progression of amyotrophic lateral sclerosis (ALS), newly developed worm models of the disease reveal. This finding was reported in the study, “Single copy/knock-in models…

A newly developed method can help characterize the types of misfolded proteins present in patients with amyotrophic lateral sclerosis (ALS), according to findings in a thesis. The recently published doctoral thesis, titled “Structural investigation of SOD1 aggregates in ALS: identification of prion strains using anti-peptide antibodies,” was written…