News

Effort will make Friedreich’s ataxia data more accessible for research

The nonprofit Critical Path Institute (C-Path) and the Friedreich’s Ataxia Research Alliance (FARA) are partnering to bolster the library of data from people with Friedreich’s ataxia (FA) and make it more accessible for researchers to develop new treatments. The effort will involve integrating additional FA datasets into C-Path’s…

FA treatment nomlabofusp picked for FDA’s START pilot program

The U.S. Food and Drug Administration (FDA) has selected nomlabofusp, an investigational treatment for Friedreich’s ataxia (FA), to participate in the Support for Clinical Trials Advancing Rare Disease Therapeutics (START) pilot program. Launched by the FDA in September 2023, the milestone-driven program seeks to accelerate the development of…

Potential therapy ATH434 may target toxic iron buildup in cells

An experimental compound called ATH434, currently being evaluated in clinical trials for a neurological disorder called multiple system atrophy, also may have the potential to treat Friedreich’s ataxia (FA). Data specifically indicate that ATH434 could help to reduce the toxic buildup of iron in cells that is characteristic…

Friedreich’s Ataxia Awareness Month begins with conversations

May will be a busy month for patients, doctors, researchers, and advocates in the Friedreich’s ataxia (FA) community as they participate in FA Awareness Month, an initiative for sharing information, personal stories, and support for those living with the rare neuromuscular disease. Named after Nikolaus Friedreich, the German physician…