News

The first patient has been dosed in a Phase 1 clinical study testing AMX0114, an experimental therapy being developed by Almylyx Pharmaceuticals for amyotrophic lateral sclerosis (ALS). The treatment targets calpain-2, a protein believed to contribute to nerve cell damage in ALS. Called LUMINA (NCT06665165), the study was cleared…

The first patient has been enrolled in an expanded access program (EAP) trial evaluating MN-166 (ibudilast), Medicinova’s oral anti-inflammatory drug, in people with amyotrophic lateral sclerosis (ALS). EAPs, also known as compassionate use programs, allow patients with serious or life-threatening conditions to access investigational therapies outside of clinical…

Brainstorm Cell Therapeutics is moving forward in its efforts to get needed regulatory clearances to launch a Phase 3b clinical trial in the U.S. to test its stem cell therapy NurOwn (debamestrocel) in people with amyotrophic lateral sclerosis (ALS) with less advanced disease. The company has submitted to…

An experimental candidate by Ractigen Therapeutics to treat amyotrophic lateral sclerosis (ALS) linked to mutations in the SOD1 gene has shown promising signs of slowed functional decline and stable or improved breathing in ALS patients of a small early trial. The therapy, RAG-17, also significantly reduced blood levels…

Neurosense Therapeutics’ oral therapy PrimeC significantly reduces blood levels of miRNAs — molecules involved in regulating gene activity — associated with disease progression and survival in people with amyotrophic lateral sclerosis (ALS). That’s according to six-month biomarker data from the Phase 2b PARADIGM clinical trial (NCT05357950), in which…

Daily treatment with IPL344 for up to three years was safe and appeared to slow amyotrophic lateral sclerosis (ALS) progression, according to recently published data from a small Phase 2a clinical trial. Patients undergoing treatment gained weight and experienced slower declines in functional abilities and lung function. The…

The ALS Association is teaming with the workplace learning company Schoox to provide education resources for patients, caregivers, healthcare providers, and others affected by amyotrophic lateral sclerosis (ALS). With this partnership, Schoox will provide fast and easy access to a range of resources, including educational materials and…

Verge Genomics has identified multiple digital clinical biomarkers that can be used to assess short-term disease progression in the pre-treatment run-in period of a Phase 1b trial testing VRG50635, its treatment candidate for amyotrophic lateral sclerosis (ALS), in patients. These digital biomarkers measure changes in key functions, such…

A rare mutation that leads to abnormally high activity of the CREB3 protein is associated with a reduced risk of developing amyotrophic lateral sclerosis (ALS) and people with ALS who carry this mutation progress significantly more slowly and live longer than those who don’t. That’s according to the study,…

A patient with late-stage amyotrophic lateral sclerosis (ALS) was treated with TRE-515, Trethera’s investigational therapy, under a U.S. Food and Drug Administration (FDA) expanded access program, the company has announced. The program enables patients with life-threatening conditions, including ALS, to receive experimental therapies outside clinical trials when no…