Additional alignment with FDA on LX2006 planned pivotal study including protein expression co-primary endpoint: based on improvements in LVMI ...

At this point, there are several therapies which are coming along, a large trial called the ExRx trial, which included the nutraceutical, nicotinamide riboside has finished. The data are not yet ...

Per the company, SGT-212 is the only full-length frataxin replacement gene therapy candidate that targets the cardiac manifestations of FA. SGT-212 is being developed to treat the neurologic and ...

In this video David Lynch, MD, PhD, highlights one of the greatest unmet needs in Friedreich's ataxia management.