One year of treatment with the experimental oral therapy PrimeC led to improved iron accumulation in people with amyotrophic lateral sclerosis (ALS), according to new clinical trial data announced by PrimeC’s developer, Neurosense Therapeutics. Previous results showed that people who received PrimeC for one year experienced significantly slower…
Diamir Biosciences has received a patent in the European Union that covers the use of microRNAs (miRNAs), that is, small noncoding RNA molecules that modulate gene activity, as biomarkers for diagnosing amyotrophic lateral sclerosis (ALS). The patent, “Methods of using miRNAs from bodily fluids for detection and…
Black people with amyotrophic lateral sclerosis (ALS) tend to have a different disease course than white patients; they are diagnosed at a younger age, more commonly have muscle weakness that starts in the hands, and have pneumonia more frequently, a study from the Centers for Disease Control and Prevention…
Engensis (VM202) is an investigational gene therapy being developed by Helixmith, formerly known as ViroMed, for the treatment of amyotrophic lateral sclerosis (ALS). How Engensis works ALS is characterized by the progressive loss of motor neurons, the specialized nerve cells that control voluntary muscles, resulting in muscle weakness and…
A mutation in the IGFBP7 gene is associated with a very rare phenomenon in which people with amyotrophic lateral sclerosis (ALS) have a reversal in disease progression and experience partial or full recovery, a new study reports. Because the mutation results in less production of the resulting protein IGFBP7,…
A decade later, the family of Pete Frates, the amyotrophic lateral sclerosis (ALS) patient who helped start the Ice Bucket Challenge, is relaunching the campaign that went viral on social media, raising awareness and money to support ALS research. In an event organized by the Peter Frates…
A couple years after my husband, Todd, was diagnosed with ALS, we moved to the Keweenaw Peninsula of Michigan to be near my parents. We had two small children at the time, so we’d explore the shores of Lake Superior for family outings. We spent time at McLain State…
The investigational therapy BIIB078 was generally well tolerated in a Phase 1 clinical trial, but did not show evidence of slowing disease progression in people with amyotrophic lateral sclerosis (ALS) caused by mutations in the C9orf72Â gene, according to recently published data from the trial. Because the trial failed to…
Treatment with Qalsody (tofersen) substantially slowed disease progression and reduced markers of nerve damage in people with amyotrophic lateral sclerosis (ALS) associated with SOD1 mutations (SOD1-ALS) who took part in an expanded access program in Germany. Patient-reported outcome measures also suggested a favorable perception of Qalsody, with most…
Nearly a year of treatment with TPN-101, an investigational oral molecule from Transposon Therapeutics, safely slowed disease progression and lung function decline in people with amyotrophic lateral sclerosis (ALS) related to C9orf72 mutations. That’s according to final data from a Phase 2a study (NCT04993755) that tested TPN-101…
