Qalsody can slow progression of SOD1-ALS, aid function: Study

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…

When my late husband, Jeff, was diagnosed with ALS in 2018, I remember reading that it could be an isolating disease. I didn’t understand that at the time; our days were filled with testing appointments as we went from electromyography to blood work, swallow study to spinal tap. Our…

A computer analysis using real-world data from an amyotrophic lateral sclerosis (ALS) clinic identified four groups of patients with distinct rates of disease progression, a new study by researchers in Portugal reported. The findings continue to demonstrate the differences in ALS presentation and progression seen among people with the…

Twelve years ago, my husband, Todd, and I installed bamboo flooring in three bedrooms and the living room of our new accessible home. Todd had been diagnosed with ALS two years before, and his arms had become quite weak. Contractors built most of the house, and a few volunteers…

Treatment with a high dose of ILB, an experimental injection therapy being developed by Tikomed, was well tolerated and appeared to slow the progression of amyotrophic lateral sclerosis (ALS) in a small clinical trial in the U.K. That’s according to data from the exploratory Phase 2 study…

A new genetic mutation that may cause amyotrophic lateral sclerosis (ALS) has been discovered in families living in La Rioja, a region in Spain, according to a study. The gene mutation, found in 10 ALS patients from seven different families, affected the ARPP21 gene, which encodes a protein that…

Imagine you’re all alone, stranded on a remote island and surrounded by miles and miles of uncharted ocean. Here’s my question: How much time would you let pass before giving up hope of ever being rescued? One year? How about five years? Or are you an “I’ll never give up…

Io Therapeutics is planning to launch a Phase 2 trial into its investigational oral therapy IRX4204 in people with amyotrophic lateral sclerosis (ALS). The decision follows recent tests showing that the candidate treatment dampened neuroinflammation in an animal model of neuronal autoimmunity, or diseases marked by self-directed…

Kadimastem has partnered with Pluri to produce its investigational cell-based therapy AstroRx for an upcoming Phase 2a clinical trial in amyotrophic lateral sclerosis (ALS). Under the agreement, Pluri will manufacture AstroRx at its 47,000-square-foot cell-therapy production facility under current good manufacturing practices (GMP), a set of…