News

The experimental treatment SBT-272 delayed the onset of neurological symptoms, lowered levels of a biomarker of nerve damage, and prolonged the life of male mice in a model of amyotrophic lateral sclerosis (ALS). Stealth BioTherapeutics, the company developing SBT-272, also announced plans to open a Phase 1…

The final amyotrophic lateral sclerosis (ALS) patient treated with oral AMX0035 in the CENTAUR study has completed all scheduled follow-up visits and researchers will now evaluate data collected on the potential treatment’s safety and efficacy, Amylyx Pharmaceuticals, its developer, announced. Final results of the Phase 2 study (NCT03127514), which…

Use of reldesemtiv in a clinical trial in amyotrophic lateral sclerosis (ALS) patients led to a slower decline in muscle control needed for speech relative to placebo, a study reports. The trial used Aural Analytics’ proprietary speech analytics platform technology to evaluate this motor function in a large group of…

Experimental treatment reldesemtiv appears to reduce functional deterioration in patients with fast progressing amyotrophic lateral sclerosis (ALS), a new analysis of the FORTITUDE-ALS trial shows. The additional analyses were presented by the trial’s principal investigator, Jeremy M. Shefner, MD, PhD, a professor at the Barrow Neurological Institute…

The U.S. Department of Defense (DoD) has issued a two-year research grant totaling $758,121 to support a group of scientists from the University of Arizona Health Sciences Center for Innovation in Brain Science (CIBS) working on the development of RASRx1902, a potential treatment for amyotrophic lateral sclerosis…

The Chan Zuckerberg Initiative (CZI) has awarded I AM ALS a $453,000 grant to develop ways to connect amyotrophic lateral sclerosis (ALS) stakeholders and the public in the fight against ALS and other rare disorders. As part of CZI’s wider effort to accelerate investigations into rare diseases,…

Mutations in the annexin A11 gene (ANXA11), seen in familial amyotrophic lateral sclerosis (ALS) and frontotemporal dementia, disrupt the transport of key RNA molecules inside neurons, recent research shows. The annexin A11 protein is a molecular adaptor required for RNA molecules, essential for neurons’ function, to hitchhike on lysosomes…

In contrast to what is often assumed, new research suggests that clumps of the protein TDP-43 — a hallmark of amyotrophic lateral sclerosis (ALS) — actually protect nerve cells rather than harm them. If this finding confirmed in future studies, it could hugely impact how ALS and other neurodegenerative diseases…

The release of fragmented or dysfunctional mitochondria — a cell’s powerhouse — by immune and structural cells inside the central nervous system is a critical step that triggers neuron death and the progression of human neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), a study shows.

Next month’s annual conference of the National Organization for Rare Disorders (NORD) in Washington, D.C., couldn’t come at a better time, says Marshall Summar, MD, chairman of NORD’s board of directors. “The pace of discovery in rare diseases has gone from brisk to hypersonic,” Summar told Bionews Services, publisher…