PrimeC, a new combination treatment by NeuroSense Therapeutics to slow down or halt the progression of amyotrophic lateral sclerosis (ALS), has received orphan drug status from the U.S. Food and Drug Administration (FDA), and is being investigated in two recently-initiated clinical trials. The decision to…

A new spinal cell delivery method may reduce the risks and boost the effectiveness of stem cell therapy designed to regenerate the nervous system in neurodegenerative diseases such as amyotrophic lateral sclerosis (ALS), an early study in rats has found. The study, “Spinal parenchymal occupation by…

Much work is ongoing in amyotrophic lateral sclerosis (ALS), as researchers look for better ways of treating people with a disease that progressively cripples and kills motor neurons — and possibly finding a first therapy addressing the root cause of ALS rather than its symptoms. “ALS is always…

A new tool to search for medicines that might treat neurodegenerative diseases linked to problems with mitochondria, including amyotrophic lateral sclerosis (ALS), has been developed by a team of scientists at the Scripps Research Institute, a study reports.  This tool…

At the time of his stunning 2015 amyotrophic lateral sclerosis (ALS) diagnosis, Ed Rapp was a veteran Caterpillar executive who had worked and lived around the world. Since being diagnosed, Rapp has directed his efforts toward ALS advocacy. Those efforts will be recognized on Feb. 22, when he…

A single protein called Gemin3 may be the bridge linking the varied proteins known to underlie amyotrophic lateral sclerosis (ALS), suggesting a common pathway in this disease, a study using a fruit fly model showed.  The discovery of this common…

The presence of autoantibodies against a calcium channel in people with amyotrophic lateral sclerosis (ALS) may explain the frequent development of type 2 diabetes in these patients, a preclinical study suggests. The data, which pinpoint the toxic effects of these autoantibodies in mouse insulin-secreting pancreatic cells, shed light on…

Kevin Schaefer hadn’t been in an airport since he was 4 years old, so he had been looking forward to flying from his home in Cary, North Carolina, to Anaheim, California, in June for the 2019 Cure SMA Conference. As it turned out, his experience didn’t go as expected.

A $1 million gift from the Manouk Djoukhadjian Family Foundation II will create Quebec’s first philanthropic research chair on amyotrophic lateral sclerosis (ALS). The donation to the Armand-Frappier Foundation will establish the Anna Sforza Djoukhadjian Philanthropic Research Chair to advance ALS research at the Institut national…

Stealth BioTherapeutics announced the initiation of a Phase 1 trial to assess the safety and tolerability of its investigational oral treatment, SBT-272, in healthy volunteers. SBT-272 is being developed to treat neurodegenerative diseases marked by problems in the workings of mitochondria (a cell’s powerhouse), including amyotrophic lateral sclerosis…