Patricia Inácio, PhD,  science writer—

Bringing artificial intelligence (AI) to the development of small molecule therapies for amyotrophic lateral sclerosis (ALS) and other neurological diseases is the goal of a business partnership in Asia. The newly signed agreement is between Insilico Medicine, an AI-driven drug discovery company based in Hong Kong, and 4B Technologies of Suzhou,…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to ketamine, PharmaTher’s investigational therapy for amyotrophic lateral sclerosis (ALS). Orphan drug status is intended to encourage the development of therapies for rare diseases affecting fewer than 200,000 people in the U.S. It provides…

Patient enrollment has begun for Cytokinetics‘ pivotal Phase 3 clinical trial investigating reldesemtiv for the treatment of people with early amyotrophic lateral sclerosis (ALS). The global COURAGE-ALS trial (NCT04944784) will enroll approximately 555 adults who are within two years of their first symptom of muscle weakness.

Coya Therapeutics’ ALS001, a potential regulatory T-cell (Treg) therapy designed to halt amyotrophic lateral sclerosis (ALS) progression, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). Furthermore, a Phase 2a trial (NCT04055623) testing ALS001 versus a placebo in 12 ALS patients…

Alector and GlaxoSmithKline (GSK) have established a partnership to develop two investigational antibodies — called AL001 and AL101 — to boost the production of the progranulin protein as a potential treatment for neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS). Low levels of progranulin, known as PGRN, which is a…

Clene and its subsidiary Clene Nanomedicine are negotiating to increase their manufacturing capacity as they prepare to release findings — expected early next year — from a pivotal and enrolling Phase 3 trial of CNM-Au8 as an oral therapy aiming to slow amyotrophic lateral sclerosis (ALS) progression. Negotiations include the…

An investigational RNA molecule called an RNA aptamer halted the death of motor neurons and helped preserve motor function in a mouse model of amyotrophic lateral sclerosis (ALS). Further research is ongoing to confirm the findings in additional ALS models before testing it in humans, “but I’m optimistic that this…

The U.S. Food and Drug Administration (FDA) has awarded orphan drug designation to Spinogenix’s investigational oral molecule SPG302 for treating amyotrophic lateral sclerosis (ALS). Orphan drug status is granted by the FDA to encourage the development of therapies for conditions that affect fewer than 200,000 people in the…

Ahead of this year’s Rare Disease Week on Capitol Hill, held virtually July 14–22, the EveryLife Foundation will award grants to top advocates of rare disease organizations who participate in the week’s pre-events. The top 50 point-earners will be eligible to win $1,000 to $5,000 in grants, totaling…

Clearance was given to open a Phase 1/2 trial evaluating APB-102, a potential gene therapy for amyotrophic lateral sclerosis (ALS) patients who carry mutations in the SOD1 gene, its developer Apic Bio announced. The trial is scheduled to begin later this year or early next year, following U.S. Food and Drug Administration…