News

Nine patients with an ultra-rare form of amyotrophic lateral sclerosis (ALS) caused by mutations in the CHCHD10 gene were treated with an experimental antisense oligonucleotide (ASO) that’s designed to silence the disease-causing mutations. The treatment was developed as part of Silence ALS, an initiative that designs ASOs, that is,…

The U.S. Food and Drug Administration (FDA) has authorized Sineugene Therapeutics to begin a Phase 1/2a trial of its experimental gene therapy, SNUG01, in people with amyotrophic lateral sclerosis (ALS). The global trial will assess the treatment’s safety, tolerability, and preliminary efficacy through a dose escalation and expansion…

People living in California can help fund amyotrophic lateral sclerosis (ALS) research when filing their state taxes. The California ALS Research Network Voluntary Tax Contribution Fund allows Californians to contribute to research projects selected by the ALS Network’s scientific advisory committee. At least $250,000 must be raised…

NUZ-001, which was previously known as monepantel, slowed lung function declines by nearly 50% in people with amyotrophic lateral sclerosis (ALS), according to new analyses from a Phase 1 trial. This slowing of respiratory declines was strongly correlated with slower declines in overall function that were observed in…

Certain subsets of natural killer (NK) cells, a part of the immune system, were elevated in the blood of people with amyotrophic lateral sclerosis (ALS), where they showed altered gene activity and signaling patterns, a study found. One subset was associated with altered immune signaling, while another was linked…

Scientists have developed a novel method to efficiently grow motor neurons from skin cells, which may serve as a platform for creating cell therapies to treat diseases like amyotrophic lateral sclerosis (ALS). The new method was described in two papers — one titled “Compact transcription factor cassettes…

Health Canada has invited Neurosense Therapeutics to discuss the potential for giving conditional approval to PrimeC in treating amyotrophic lateral sclerosis (ALS), according to a company filing with the U.S. Securities and Exchange Commission. The upcoming meeting provides an opportunity to consider if the drug is eligible…

Signaling at the low-density lipoprotein receptor, known as LDLR, may be an important driver of neurodegeneration in sporadic amyotrophic lateral sclerosis (ALS), according to new preclinical research by U.S. scientists. In earlier work, the scientists discovered that mice injected with cerebrospinal fluid, or CSF — the fluid that…

A single dose of Insmed’s investigational gene therapy INS1202 preserved motor function and prolonged survival in a mouse model of amyotrophic lateral sclerosis (ALS) associated with SOD1 gene mutations. The therapy was also found to protect against nerve cell loss and had other beneficial effects on disease-related biomarkers.

An experimental oral small molecule being developed by Quralis for amyotrophic lateral sclerosis (ALS) reduced motor neuron excitability as intended in healthy volunteers. That’s according to top-line data from a Phase 1 clinical study (NCT06681441) that tested two doses of QRL-101 against a placebo in healthy adults.