Larimar Therapeutics, Inc. (NASDAQ:LRMR) released initial data from the ongoing long-term OLE study evaluating daily ...
It is a monogenic disease, that is to say, it is caused by a defect in only one gene. Those affected by this disorder have inherited an altered frataxin gene from both parents. The project aims to ...
The company’s alignment with the FDA on key regulatory endpoints for LX2006, such as a 40% frataxin expression and ... which is an AAVrh10-based gene therapy candidate for the treatment of ...
Reached alignment with FDA on key elements of registrational development plan for LX2006, including accelerated approval pathway with left-ventricular mass index (LVMI) and frataxin protein ...