CNM-Au8, an investigational disease-modifying therapy for people with early amyotrophic lateral sclerosis (ALS), showed consistent clinical benefits in patients with both limb and bulbar onset disease, according to new analyses of RESCUE-ALS Phase 2 clinical trial data. In particular, the treatment significantly extended the time to disease progression —…

When Leah Stavenhagen was 27, she was living in Paris and traveling through Europe. But an amyotrophic lateral sclerosis (ALS) diagnosis forced her to give up her job as a consultant with Ernst & Young and dashed her dreams of continuing to work abroad. At the time, she seemed…

Repeat injections of mesenchymal stem cells (MSCs) into the spinal canal were safe and well tolerated in patients with amyotrophic lateral sclerosis (ALS), and showed potential to significantly slow the rate of disease progression, a small Phase 2 clinical trial found. “Larger studies are warranted to confirm our observations…

Brainstorm Cell Therapeutics‘ investigational cell-based therapy, NurOwn, might be able to slow disease progression and be of “meaningful” benefit to  people with less severe amyotrophic lateral sclerosis (ALS), according to new analyses of Phase 3 trial data. The company announced last year that its Phase 3 trial…

A combined gene therapy that delivered two nerve growth factors — NRG1-I and NRG1-III — to muscle and nerve cells improved motor function and delayed disease onset in a mouse model of amyotrophic lateral sclerosis (ALS), a study demonstrated. However, the combo therapy did not show a synergistic effect,…

The U.S. Food and Drug Administration (FDA) has cleared AB Science to resume patient enrollment in its confirmatory Phase 3 trial investigating masitinib as an add-on treatment in people with amyotrophic lateral sclerosis (ALS). AB Science put a temporary hold on all its masitinib clinical trials due…

The loss of chemical modifications in the regulatory sequence of the TARDBP gene, called DNA methylation, seems to contribute to increased levels and clumping of the TDP-43 protein in the motor cortex of people with amyotrophic lateral sclerosis (ALS), a study found. As methylation in this gene — which…

A Phase 2 clinical trial evaluating AL-S Pharma AG’s experimental therapy AP-101 in people with amyotrophic lateral sclerosis (ALS) has enrolled its first patient. The study (NCT05039099) already is recruiting up to 63 adults with sporadic and SOD1-related familial ALS at one of its Canadian sites.

An abnormal version of tau, a protein associated with Alzheimer’s disease, may contribute to the progression of amyotrophic lateral sclerosis (ALS) by damaging mitochondria in neurons, according to a new study. The findings suggest that decreasing tau levels might be a new therapeutic strategy to lessen mitochondrial dysfunction in…

Prilenia Therapeutics has raised $43 million to support potential regulatory submissions and marketing of its lead candidate, pridopidine, for the treatment of amyotrophic lateral sclerosis (ALS). The funding, obtained through a Series B financing round, also will be used to support the potential marketing of the candidate therapy for…