Biogen (MA, USA) has announced positive results from a Phase I/II study of tofersen – an antisense oligonucleotide – for the potential treatment of SOD1 amyotrophic lateral sclerosis (ALS). These results have been published in The New England Journal of Medicine.
Author: anna.ciskowski
Experimental drug shows promising results for rare form of ALS (Links to an external site)
Researchers led by a Washington University neurologist found promising results for an experimental drug to treat a rare, inherited form of amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.
Research in St. Louis leads to promise in the fight against ALS (Links to an external site)
ST. LOUIS – Research taking place at Washington University’s School of Medicine is giving hope to those affected by amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig’s disease.
Experimental drug shows early promise against inherited form of ALS, trial indicates (Links to an external site)
Phase 1/phase 2 trial shows evidence for safety, biological activity of tofersen; phase 3 trial underway
Healey ALS Platform Trial receives “May Proceed” notice for three drugs (Links to an external site)
The Sean M. Healey & AMG Center for ALS at Mass General has received approval from the U.S. Food and Drug Administration (FDA) to proceed with administering three proposed drug regimens in the HEALEY ALS Platform Trial – the first trial of its kind for amyotrophic lateral sclerosis (ALS).
Prospective natural history study of C9orf72ALS clinical characteristics and biomarkers
Prospective natural history study of C9orf72ALS clinical characteristics and biomarkers Our team’s hard work has been published in Neurology. This paper profiles the clinical features, such as age at disease onset, survival duration, and measures of disease progression, of ALS patients with mutations in the C9orf72 gene. By defining the natural history of this patient […]
Antisense Drugs for Huntington’s, ALS and Prion Diseases Could Meet the Dire Need for Brain Treatments (Links to an external site)
A genetic therapy that increases or lowers levels of a protein raises hopes for a treatment for neurological disorders.