A messenger RNA (mRNA) molecule that provides the instructions for a protein involved in glutamate signaling is not edited as efficiently in amyotrophic lateral sclerosis (ALS) patients as in people without the neurological disorder, according to a new study suggesting its potential use as an ALS biomarker. Glutamate is…
Apellis Pharmaceuticals’ investigational therapy pegcetacoplan (APL-2) has failed to improve a combined assessment of function and survival in people with amyotrophic lateral sclerosis (ALS), according to top-line data from the MERIDIAN Phase 2 clinical trial. While the treatment was well tolerated, consistent with its established safety profile,…
A committee of the European Medicines Agency (EMA) is leaning toward not recommending a conditional approval of Amylyx Pharmaceuticals’ AMX0035 for treating amyotrophic lateral sclerosis (ALS) in the European Union. The drug is already marketed in the U.S. under the name Relyvrio (sodium phenylbutyrate and taurursodiol) and…
Allopurinol and carvedilol, two medications respectively used to manage gout and high blood pressure, significantly reduce the chances of developing amyotrophic lateral sclerosis (ALS), Alzheimer’s, or Parkinson’s disease, a new study suggests. “These findings suggest a possible new direction for repurposing or developing medications for neuroprotection,” Brad…
Locanabio‘s investigational therapy significantly reduced the buildup of toxic RNA molecules in amyotrophic lateral sclerosis (ALS) patient cells and mouse models associated with C9ORF72 mutations, according to new data. These RNA products — intermediate molecules that are produced from DNA as a template for protein production — are…
The data platform PatientsLikeMe (PLM)Â has made its de-identified amyotrophic lateral sclerosis (ALS) patient database available to scientists around the world to advance clinical research in the neurodegenerative disorder. Part of a collaboration between PLM and the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital (MGH), the…
WVE-004, Wave Life Sciences‘ investigational treatment for amyotrophic lateral sclerosis (ALS), significantly reduces toxic proteins associated with C9orf72 genetic mutations, but that doesn’t seem to translate into functional status gains in patients. Based on findings from the Phase 1b/2a FOCUS-C9 trial (NCT04931862), the company has decided to discontinue developing…
A sizable portion of people with sporadic amyotrophic lateral sclerosis (ALS) carry a genetic mutation known to cause other neurological disorders, a new study shows. “This suggests shared risk factors among these diseases, shared mechanisms that cause nerves to die – and perhaps shared therapeutic strategies in the future,”…
A Phase 2b trial of PrimeC for people with amyotrophic lateral sclerosis (ALS) has completed patient enrollment, according to NeuroSense Therapeutics, the therapy’s developer. Called PARADIGM (NCT05357950), the study enrolled 69 adults diagnosed with ALS, ages 18-75, at sites in Israel, Italy, and Canada. The company…
Jodi O’Donnell-Ames founded Hope Loves Company after losing her husband to ALS. (Courtesy of Jodi O’Donnell-Ames) This is Jodi O’Donnell-Ames’ story: The term amyotrophic lateral sclerosis (ALS) was not a part of my vocabulary until May 1995. That’s when my husband, Kevin O’Donnell, was diagnosed with…
