A toxin produced by algae may contribute to the development of amyotrophic lateral sclerosis (ALS) by lowering the stability of a protein in the brain, a new study suggests. Titled “β-Methylamino-L-alanine substitution of serine in SOD1 suggests a direct role in ALS etiology,” the study was…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Apic Bio‘s APB-102, an investigational gene therapy aiming to treat familial amyotrophic lateral sclerosis (ALS) associated with mutations in the superoxide dismutase 1 (SOD1) gene. “This orphan drug designation represents an important…
A study in identical twins or triplets found that changes in DNA marks, collectively known as DNA methylation and typically associated with aging, can like genetics play an important role to determining the development of amyotrophic lateral sclerosis (ALS). The four sets of ALS-affected and unaffected siblings carried age-related differences…
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…
New genetic mutations in the coding sequence of the GLT8D1 enzyme have been identified and linked to inherited amyotrophic lateral sclerosis (ALS), a study reports. Researchers at the University of Sheffield in the U.K. and their collaborators found that these GLT8D1 gene variants prevented the normal functioning of the enzyme, which…
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…
An experimental gene therapy based on RNA interference (RNAi) shows potential to treat patients with familial amyotrophic lateral sclerosis (ALS) caused by mutations in the superoxide dismutase 1 (SOD1) gene, according to results of a preclinical study in nonhuman primates. The study, “Safe and effective superoxide…
Specific patterns of a misfolded protein — superoxide dismutase 1 (SOD1) — are found in the brain and spinal cord of patients with sporadic amyotrophic lateral sclerosis (ALS), according to a new study, supporting research suggesting that misfolded SOD1 plays a role in this ALS subtype. The research, “…
Requip (ropinirole), a medication already approved to treat Parkinson’s disease, may be a potential therapeutic agent for amyotrophic lateral sclerosis (ALS), according to a preclinical study. The study, “Modeling sporadic ALS in iPSC-derived motor neurons identifies a potential therapeutic agent,” was published in the journal…
A gene therapy that might treat familial amyotrophic lateral sclerosis (ALS), an inherited form of the disease, was recently granted orphan drug status by the European Medicines Agency (EMA), an award that carries incentives to promote potential treatments for rare diseases. The investigative therapy is in preclinical development…
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