FDA approves tofersen, now Qalsody, as treatment for SOD1-ALS
The U.S. Food and Drug Administration (FDA) has granted conditional approval to Biogen’s tofersen, now named Qalsody, for the…
Articles by Inês Martins, PhD
AC Immune Working to Move TDP-43 Antibody Into Clinical Testing for ALS
AC Immune is planning to advance its investigational anti-TDP-43 antibody into clinical testing for neurodegenerative diseases, like amyotrophic…
First Patients Enrolled in Pivotal HEALEY ALS Trial of Multiple Therapies
The first platform trial for amyotrophic lateral sclerosis (ALS), designed to assess multiple treatment candidates simultaneously with a goal…
AstroRx Slows ALS Progression at All Doses Tested, Trial Shows
Kadimastem’s cell therapy candidate, AstroRx, significantly lowers the rate of disease progression in…
ALS Researchers Probe Cerebrospinal Fluid’s Role in Motor Neurons
The clear, colorless liquid that fills and surrounds the brain and spinal cord in people with amyotrophic lateral sclerosis…
New FUS Mutation Linked to Aggressive ALS in Teenage Girl
A new mutation in the FUS gene, which results in a shorter version of the FUS protein that…
Tofersen Safe, Appears to Slow Disease Progression in SOD1 ALS Patients, Phase 1/2 Trial Shows
Tofersen, Biogen’s investigational therapy for people with familial amyotrophic lateral sclerosis (ALS) caused by …
Lifelong Exercise May Limit Brain’s Ability to Adapt in ALS, Study Suggests
Amyotrophic lateral sclerosis (ALS) patients who never regularly engaged in sports or physical activity have more extensive metabolic changes…
Researchers Identify New Genetic Defect Linked to ALS
Mutations in the UBQLN2 gene, known to cause amyotrophic lateral sclerosis (ALS), promote the buildup of toxic waste…
Studies of DNL747 Paused, Work Shifts to Possibly Better Treatment
Denali Therapeutics and Sanofi announced a pause in clinical testing of their small molecule inhibitor DNL747 in favor…