The findings in Alzheimer disease further elucidated the safety profile of AMX0035, as treatment emergent adverse events in the PEGASUS trial appeared to be largely disease driven. New data from… More >>

Reductions in poly(GP), a key disease biomarker indicating target engagement, observed across all treatment groups after single doses Extending dose observation period from three months (day 85) to six months… More >>

Dear ALS Community, We are writing to let you know that Biogen and Ionis Pharmaceuticals announced topline results from the Phase 1 study of BIIB078/Ionis-C9RX. This asset was an investigational… More >>

Biogen and Ionis Pharmaceuticals today announced topline results from the Phase 1 study of BIIB078 (IONIS-C9Rx), an investigational antisense oligonucleotide (ASO) for people with C9orf72-associated amyotrophic lateral sclerosis (ALS). In… More >>

TRICALS (the Treatment Research Initiative to Cure ALS), in collaboration with ALS associations and funding agencies from Europe and Australia, has launched its clinical platform trial for amyotrophic lateral sclerosis… More >>

The independent Data and Safety Monitoring Board (DSMB) recommended stopping the zilucoplan regimen in the HEALEY ALS Platform Trial after a pre-scheduled interim analysis demonstrated futility. The Healey ALS Platform… More >>

Amylyx Pharmaceuticals announced that its Marketing Authorisation Application (MAA) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for AMX0035 (sodium phenylbutyrate [PB] and ursodoxicoltaurine [TURSO;… More >>

CAMBRIDGE, Mass. – Amylyx Pharmaceuticals, Inc. today announced it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP)… More >>