-
Featured
Innovations in ALS Clinical Trials
Merit Cudkowicz, MD, MSc, chief of Neurology at Massachusetts General Hospital, discusses innovations in ALS clinical trials since she started in the field in 1994.
-
Featured
Advancements in the Treatment of Neurodegenerative Disease
In this video, Ghazaleh Sadri-Vakili, PhD, director of the NeuroEpigenetics Laboratory and the MassGeneral Institute of Neurodegenerative Disease, discusses her genetic research, specifically concerning neurodegenerative diseases like ALS and X-linked dystonia parkinsonism.
-
Featured
Identifying New Potential Therapeutic Targets for ALS FTD
Transcripts of repeat expansions in the C90RF72 gene undergo a repeat-associated non-AUG (RAN) translation pathway that generates multiple aberrant proteins. Researchers at Mass General Hospital identified new potential therapeutic targets for ALS FTD.
-
Tofersen Lowers Disease Markers, Slowing Progression for SOD1-Mutated ALS in Phase 3 Trial
Merit E. Cudkowicz, MD, MSc, and colleagues report that in a phase 3 randomized trial, patients with faster-progressing SOD1-mutated amyotrophic lateral sclerosis who received tofersen showed greater reductions in SOD1 than the placebo group, but the primary endpoint of change in ALS-related function was not met.
-
Endoplasmic Reticulum Proteins Regulate Organelle Distribution; Change in Function May Treat Neurologic Disease
Craig Blackstone, MD, PhD, and colleagues discovered interaction between the endoplasmic reticulum (ER) and microtubules modified by ER proteins plays a key role in the positioning of cellular organelles. Changing ER function by "fine-tuning" molecular interactions may have applications in treating neurologic diseases.
-
Healey Platform ALS Trial Expected to Expedite U.S. Approval of New Treatments
The Healey Platform ALS Trial, headquartered at Massachusetts General Hospital, launched in July 2020 and is expected to bring new treatments to patients with amyotrophic lateral sclerosis more quickly and at lower cost than randomized, controlled trials of single agents.
ALS Contributors
-
Clotilde Lagier-Torurenne, MD, PhD
Assistant Professor of Neurology, Harvard Medical School, Assistant in Neuroscience, Massachusetts General Hospital
Recent Article
Researchers Identify a New Therapeutic Target for TDP-43-mediated Neurodegenerative Diseases
-
Ghazaleh Sadri-Vakili, MS, PhD
Assistant in Neuroscience, Massachusetts General Hospital, Assistant Professor of Neurology, Harvard Medical School
Recent Article
Novel Target Identified for Treatment of ALS
-
James D. Berry, MD, MPH
Co-director, Multidisciplinary Amyotrophic Lateral Sclerosis (ALS) Clinic
Recent Article
Healey Platform ALS Trial Expected to Expedite U.S. Approval of New Treatments
-
Merit Ester Cudkowicz, MD, MSc
Chief, Neurology Service, Director of the Sean M. Healey & AMG Center for ALS at Mass General, Director of Neurological Clinical Research Institute and the Jilieane Dorn Professor of Neurology, Harvard Medical School
Recent Article
Tofersen Lowers Disease Markers, Slowing Progression for SOD1-Mutated ALS in Phase 3 Trial
-
Nazem Atassi, MD
Associate Director, Neurological Clinical Research Institute (NCRI), Mass General Hospital, Associate Professor of Neurology, Harvard Medical School
Recent Article
High-Dose Tamoxifen "Wins" Selection Trial in ALS
Neuroscience Care at Mass General
Through a combination of unmatched expertise, creativity and compassion, Mass General Neuroscience relentlessly advances the fields of Neurosurgery, Neurology and Psychiatry to unleash our patients’ potential.