Trial News & Events
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Rapa Therapeutics have announced the first participant enrolled in the Expanded Access to RAPA-501 Protocol (EAP)
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Rapa Therapeutics have announced the first participant enrolled in the Expanded Access to RAPA-501 Protocol (EAP). This EAP is sponsored by the National Institutes of Health (NIH) - Neurological Disorders and Stroke (NINDS), as part of the U.S. government’s landmark Accelerating Access to Critical Therapies for ALS Act (ACT for ALS).
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Rapa Therapeutics have announced the first participant enrolled in the Expanded Access to RAPA-501 Protocol (EAP
The Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital and Rapa Therapeutics have announced the first participant enrolled in the Expanded Access to RAPA-501 Protocol (EAP). This EAP is sponsored by the National Institutes of Health (NIH) - Neurological Disorders and Stroke (NINDS), as part of the U.S. government’s landmark Accelerating Access to Critical Therapies for ALS Act (ACT for ALS).
Clene Reports Significant Survival Benefit With CNM-Au8® Treatment in ALS EAP Compassionate Use Programs
Clene Nanomedicine, Inc. reported new, significant survival results from two independent analyses of the pooled data from the intermediate-size EAPs supported by Clene.
Transposon Announces Interim Results from a Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related ALS
TPN-101 showed a clinical effect on Vital Capacity, an objective respiratory measure that correlates with mortality in patients with ALS
National Institutes of Health (NIH) funds New Multi-Institutional ALS Clinical Research Consortium
The NIH has established a transformative national ALS clinical research consortium called Access for All in ALS (ALL ALS). This new consortium is a multi-institutional effort and aims to disrupt the ALS clinical research landscape in the U.S.
"FDA Grants Accelerated Approval for QALSODY™ (tofersen) for SOD1-ALS, a Major Scientific Advancement as the First Treatment to Target a Genetic Cause of ALS," published by Biogen
QALSODY is the first approved treatment to target a genetic cause of ALS.
"Biogen Provides Update on FDA Advisory Committee Meeting on Tofersen for SOD1-ALS," published by Biogen
FDA Advisory Committees provide non-binding recommendations for consideration by the FDA. The New Drug Application for tofersen for the treatment of SOD1-ALS was submitted to the FDA for consideration under accelerated approval. The FDA is continuing its review of tofersen with a Prescription Drug User Fee Act action date of April 25, 2023.
