MDA 2023: Designer DNA drug for ALS shows promise in mouse studies

How I raise ALS awareness, even at my ALS clinic

“So tell us, Dagmar, what’s your latest project?” The question came from my neurologist as we chatted during my recent ALS clinic visit. She asked not only for herself, but for the medical student who was also in the room. As I prattled on, part of my brain reflected…

FDA panel votes in favor of tofersen biomarker data

Changes in neurofilament light chain (NfL) levels — a biomarker of nerve cell damage — were deemed by an advisory committee reasonably likely to predict clinical efficacy from tofersen in people with amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations. The unanimous vote from the Peripheral and Central Nervous…

MDA 2023: COYA 302 therapy slows ALS progression in early trial

Four people with amyotrophic lateral sclerosis (ALS) each experienced a marked slowing in disease progression with COYA 302, an experimental immune-modulating therapy tested in a small proof-of-concept clinical trial, according to Coya Therapeutics, its developer. Based on these findings, Coya is planning to start work on further trials to…

MDA 2023: Walking, other functions retained longer on Radicava

Amyotrophic lateral sclerosis (ALS) patients treated with Radicava (edaravone) tend to go longer before reaching disease milestones such as needing a walking aid or breathing support, according to an analysis of insurance data. The findings were presented at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference in…