Dr. Miller recently presented updated data for the Phase 3 Tofersen trial at the ENCALS Meeting in Edinburgh, Scotland. Slides from the presentation can be viewed here: https://investors.biogen.com/static-files/4e9393b8-0f67-47a3-a331-05ef3ac7fc63
Answer ALS is a biological and clinical resource of patient-derived, induced pluripotent stem (iPS) cell lines, multi-omic data
derived from iPS neurons and longitudinal clinical and smartphone data from over 1,000 patients with ALS. This resource provides population-level biological and clinical data that may be employed to identify clinical–molecular–biochemical subtypes of
amyotrophic lateral sclerosis (ALS). A unique smartphone-based system was employed to collect deep clinical data, including
fine motor activity, speech, breathing and linguistics/cognition. The iPS spinal neurons were blood derived from each patient
and these cells underwent multi-omic analytics including whole-genome sequencing, RNA transcriptomics, ATAC-sequencing
and proteomics. The intent of these data is for the generation of integrated clinical and biological signatures using bioinformatics, statistics and computational biology to establish patterns that may lead to a better understanding of the underlying
mechanisms of disease, including subgroup identification. A web portal for open-source sharing of all data was developed for
widespread community-based data analytics. Read the Publication (PDF)
At the American Neurological Association (ANA) Annual Meeting, Dr. Timothy Miller presented VALOR study of Tofersen Phase 3 Drug Results and Use with ALS. Tofersen is an antisense drug being evaluated for the potential treatment of SOD1-ALS. For more information and to learn about Tofersen Phase 3 Drug Results please see links below. Biogen News Release; […]
Check out Dr. Timothy Miller’s Featured Physicians interview, where WashU offers a more in-depth look at the people behind the white coats. Timothy Miller, MD, PhD – Washington University Physicians (wustl.edu)
How could a gene that causes one type of ALS be switched off? In episode 87, Tim Miller from the Washington University in St. Louis discusses his research into therapies that target the single strands of DNA or RNA which cause many cases of amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease. His article “Phase 1–2 […]
Two preliminary studies were published simultaneously in the New England Journal of Medicine, both aimed at reducing superoxide dismutase 1 (SOD1) protein levels in people who had amyotrophic lateral sclerosis (ALS) due to SOD1 mutations. In the first study, a phase I/II trial of intrathecal tofersen, Timothy Miller, MD, PhD, of Washington University in St. […]
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.
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.
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.
Phase 1/phase 2 trial shows evidence for safety, biological activity of tofersen; phase 3 trial underway