Depleting a protein called Staufen1 improved motor function and lowered protein aggregation in a mouse model of spinocerebellar ataxia type 2 (SCA2). The findings suggest that targeting Staufen1 could lead to therapies for amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. The study “Staufen1 links…

Survival and quality of life of patients with more advanced amyotrophic lateral sclerosis (ALS) can be improved significantly with the use of noninvasive mechanical ventilation (NIV). But many factors can contribute to a delayed decision on when to start mechanical ventilation, as well as on the effectiveness of the…

A man with progressive non-fluent aphasia (PNFA), one of the three types of frontotemporal dementia (FTD), was unusual in developing amyotrophic lateral sclerosis (ALS) with bulbar-onset one year later, a case study from Italy reports. The study, “A case of Progressive Non-Fluent Aphasia as onset of Amyotrophic Lateral…

The U.S. Food and Drug Administration (FDA) has approved Tiglutik, an oral suspension of riluzole, for the treatment of amyotrophic lateral sclerosis (ALS). Rilutek (riluzole, by Sanofi) has been available in the U.S. as 50 mg tablets since December 1995. However, ALS patients can have difficulties with…

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…

Stem cell transplants given to amyotrophic lateral sclerosis (ALS) patients were safe and feasible whether infused intravenously or via the spinal cord, data from two small Phase 1 trials from Iran show. However, the treatment was not seen to slow disease worsening in efficacy measures studied as secondary trial goals…

The Medicines and Healthcare products Regulatory Agency in the United Kingdom has granted permission to Tikomed to begin a Phase 2 clinical trial to evaluate the safety and effectiveness of ILB, an investigational therapy for amyotrophic lateral sclerosis (ALS). The trial will take place at…

An exploratory anti-cancer therapy may halt the characteristic toxic accumulation of TDP-43 in amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD), researchers suggest. The preclinical study, “Poly(ADP-Ribose) Prevents Pathological Phase Separation of TDP-43 by Promoting Liquid Demixing and Stress Granule Localization,” was published in the journal…

The first patient has been dosed in a Phase 3 clinical trial (NCT03491462) evaluating the effectiveness of Orphazyme’s investigational therapy arimoclomol in the treatment of amyotrophic lateral sclerosis (ALS). One of the disease mechanisms known to be involved in the development of ALS is protein misfolding and aggregation…

Physicians who help amyotrophic lateral sclerosis (ALS) patients die should carefully evaluate their motivations, capacity, and care goals, while also discussing alternatives with their patient, according to researchers. An ALS patient case and commentary, “How Should Physicians Care for Dying Patients with Amyotrophic Lateral Sclerosis?” appeared in the…