Larimar Therapeutics' CTI-1601 offers hope for Friedreich's ataxia patients, with promising trial results. Check out why LRMR ...

Friedreich’s ataxia is a progressive neuromuscular disease that causes uncoordinated movement, muscle weakness, balance problems, impaired speech, and frequently, heart abnormalities.

Most people haven’t heard of the condition, but Friedreich’s ataxia is getting more attention because of a new treatment and ...

Panelists discuss how the differential diagnosis for Friedreich's Ataxia involves distinguishing it from other ataxias, the ...

Treatment success was measured as a change from baseline in the modified Friedreich’s Ataxia Rating Scale (mFARS) score. The mFARS is an assessment of neurological function and tests ...

A Friedreich ataxia specialist discusses how the typical clinical presentation of Friedreich's Ataxia includes early onset with symptoms such as ataxia and sensory loss, while also examining the ...

After getting a green light from the European Commission, Biogen’s Skyclarys is the first approved medicine for the inherited neurological disease Friedreich’s ataxia (FA) in the EU.

Papillon Therapeutics Inc.’s PPL-001 has been awarded orphan drug designation by the FDA for Friedreich’s ataxia. PPL-001 is an experimental gene-corrected CD34+ hematopoietic stem and progenitor cell ...

Friedreich's ataxia is a rare, debilitating and life-shortening neuromuscular disease caused by genetic factors 1 If approved, omaveloxolone would be the only disease-specific treatment option for ...

Stealth Biotherapeutics Inc. has been awarded the Kyle Bryant Translational Research Award from the Friedreich’s Ataxia Research Alliance (FARA) to evaluate mitochondria-targeted molecule SBT-589 in ...

The poster presentations are as follows: Effect of nomlabofusp administration on tissue frataxin levels, plasma lipid profiles, and gene expression in patients with Friedreich’s ataxia Oral ...