News

‘Silence ALS’ Aiming for Personalized Therapy for Rare-mutation Patients

Silence ALS, a new initiative supported by Target ALS, set as its goals discovering and developing personalized antisense oligonucleotide (ASO) treatments for amyotrophic lateral sclerosis (ALS) patients with rare disease-causing mutations, while advancing understanding of the disease’s overall biology. The initiative, a collaboration between the n-Lorem Foundation, which…

Lead Antibody Targeting Toxic TDP-43 Protein to Advance, ProMIS Says

ProMIS Neurosciences announced plans to advance a monoclonal antibody against toxic TDP-43 protein clumps as a potential treatment for amyotrophic lateral sclerosis (ALS). The candidate antibody therapy, called PMN267, has shown promise in preclinical experiments conducted at independent institutions, the company also reported in a press release. Nearly all cases…

AMX0035 Extended Patients’ Survival By More Than 10 Months

Treatment with AMX0035 significantly extended the median survival of amyotrophic lateral sclerosis (ALS) patients by more than 10 months compared with a placebo, a new analysis of the Phase 2/3 CENTAUR clinical trial indicates. That survival difference was even greater in patients who continued to receive AMX0035 in the open-label…

Wave Life’s WVE-004 Shows Proof of Concept in Cell, Mouse Models

WVE-004, Wave Life Sciences‘ experimental therapy for people with amyotrophic lateral sclerosis (ALS) associated with mutations in the C9ORF72 gene, has demonstrated proof-of-concept efficacy in cell and animal models, a new study shows. The nucleic acid-based therapy was able to “potently” reduce the toxic RNA molecules and small proteins…

#AAN2022 – How Environmental Exposure Affects ALS Risk Is Studied

Researchers at the University of Michigan are focused on establishing cause and effect relationships between environmental and occupational exposures with amyotrophic lateral sclerosis (ALS). They hope this information will shed light on the mechanisms behind the disease and identify modifiable risk factors, which may have implications in preventing ALS.