Friedreich ataxia, an autosomal recessive neurodegenerative disease, is the most common of the inherited ataxias. The recent discovery of the gene that is mutated in this condition,FRDA, has led to ...
Larimar’s lead compound, nomlabofusp, is being developed as a potential treatment for Friedreich's ataxia. Larimar also plans to use its intracellular delivery platform to design other fusion ...
Only dual route gene transfer therapy in development to treat Friedreich’s ataxia with FDA IND clearance and Fast Track designation - CHARLESTOWN, Mass., Jan. 21, 2025 (GLOBE NEWSWIRE ...
Fast-track status will allow Solid Biosciences to have more frequent meetings with the FDA throughout the treatment-development process and, potentially, get the product to patients faster than would ...
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.
Larimar doses adolescents in nomlabofusp paediatric pharmacokinetic run-in study for patients with Friedreich’s ataxia: Bala Cynwyd, Pennsylvania Saturday, January 25, 2025, 15: ...