Friedreich’s ataxia

Lexeo Therapeutics’ investigational gene therapy reduces left ventricular volume and wall thickness in patients with Friedreich’s ataxia, according to a small study.

Phase II data indicate Larimar Therapeutics’ injectable therapy nomlabofusp could go head-to-head in the market with Biogen’s Skyclarys.

Despite recent investment and breakthroughs for rare diseases such as Friedreich’s ataxia and progeria, experts say helping patients with rare diseases will require cross-sector support.

Patient advocacy groups aided in the development of the very first marketed drugs for certain rare diseases, including progeria and Friedreich’s ataxia.