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Trial News & Events

First Participant Enrolled in NIH-Funded Access for All in ALS Consortium

The Access for All in ALS Consortium (ALL ALS) announced the successful enrollment of the first participant..

Established in the autumn of 2023 with funding from the National Institutes of Health (NIH), ALL ALS is a multi-institutional effort, and aims to disrupt the ALS clinical research landscape using open science methods to build broadly accessible resources to advance ALS research. The consortium brings together research scientists from across the country, combining their efforts to collect clinical and biomarker data from people with ALS symptoms, asymptomatic individuals at risk of developing inherited forms of ALS, and control participants. The ALL ALS clinical and genomic data and biofluid collections will help researchers investigate the antecedents of ALS, its onset, progression, prognostication, and response to effective therapies.

Transposon Announces Final Results from a Phase 2 Study of TPN-101 for the Treatment of C9orf72-Related Amyotrophic Lateral Sclerosis and/or Frontotemporal Dementia

Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, including Alzheimer’s disease, today announced final results from its Phase 2 study of TPN-101 in patients with amyotrophic lateral sclerosis (ALS) and/or frontotemporal dementia (FTD) related to hexanucleotide repeat expansion in the C9orf72 gene (C9orf72-related ALS/FTD).

Report Urges Immediate and Longer-Term Actions to Improve Quality of Health Care and Support Services for People with ALS, Speed Development of Therapies

A new congressionally mandated report from the National Academies of Sciences, Engineering, and Medicine recommends actions Congress, federal agencies, insurers, and others should take to strengthen health care and support services for people with amyotrophic lateral sclerosis (ALS) and accelerate research on therapies for the disease — with the goal of turning ALS into a livable disease within a decade.

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).

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