RAG-17 safe, shows early signs of efficacy as SOD1-ALS treatment

RAG-17, an investigational therapy from Ractigen Therapeutics, was safe and well tolerated across all tested doses in people with amyotrophic lateral sclerosis (ALS) carrying mutations in the SOD1 gene — cases in which the disease is known as SOD1-ALS. Those are the findings of an investigator-initiated trial (NCT05903690)…

Quralis has completed dosing in the first group of healthy adults who volunteered for its Phase 1 clinical trial of QRL-101, an experimental oral small molecule being developed for amyotrophic lateral sclerosis (ALS). QRL-101-03 (NCT06532396) is expected to enroll about 60 volunteers who’ll be divided into five…

NRG Therapeutics has received a grant from the nonprofit Target ALS to advance preclinical studies of its new class of oral small molecules targeting mitochondria, the energy producers of cells, as a potential treatment for amyotrophic lateral sclerosis (ALS). The 2024 In Vivo Target Validation grant program, an…

A friend from a lifetime ago rolled down an accessible path leading to Lake Superior in my husband’s backup power wheelchair, and I experienced a sense of déjà vu after we turned down a boardwalk trail through the woods. My husband, Todd, was able to traverse this trail in McLain…

The U.S. Food and Drug Administration (FDA) has given the green light to Dr.Noah Biotech to initiate a Phase 1 clinical trial of NDC-011, a combination therapy for amyotrophic lateral sclerosis (ALS), in healthy volunteers. The trial, planned for the first half of 2025 at a single site…

AcuraStem has received $4 million in funding from the California Institute for Regenerative Medicine (CIRM) to advance the development of AS-241, an investigational treatment strategy for amyotrophic lateral sclerosis (ALS) and related diseases. AS-241 is an antisense oligonucleotide (ASO), a type of small molecule that binds to RNA…

“Keep on moving!” That’s something I tell myself every day. Since I live with ALS, however, keeping myself moving is easier said than done — especially in the afternoon, when my energy drops and I slump deeper into my chair. Oh, my, it’d be so easy to just stay…

Measuring the levels of a novel protein biomarker called C-X-C motif chemokine ligand 12 — CXCL12 for short — in the spinal fluid may improve the diagnosis of amyotrophic lateral sclerosis (ALS) by better differentiating it from similar conditions, according to a new study. In particular, the…

A strategy that simultaneously targets motor neurons, astrocytes, and microglia — three cell types involved in the development and progression of amyotrophic lateral sclerosis (ALS) — was shown to further extend survival in a mouse model of the disease. Specifically, targeting the three cell types at the same time…

When I met my late husband, Jeff, we lived exactly 7 miles apart by car, our respective homes separated by the Potomac River just south of Washington, D.C. The bridge that crossed it nearest to our places featured a wide and safe bike lane that separated cyclists and pedestrians from…