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).

The Sean M. Healey & AMG Center for ALS and Prilenia Therapeutics Announce First Participant Enrolled in NIH-Sponsored ACT for ALS Expanded Access Protocol for Pridopidine

Amylyx Pharmaceuticals Announces Formal Intention to Remove RELYVRIO®/ALBRIOZA™ from the Market; Provides Updates on Access to Therapy, Pipeline, Corporate Restructuring, and Strategy

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.

The ALS Association Webinar: Tofersen and the FDA Approval Process

Click here to watch a recording of The ALS Association webinar on Tofersen and the FDA Approval Process.