orphan disease Latest News

Jasper Against Batten at Cures Within Reach, a 501(c)3 not-for-profit located in IL, is collaborating with an int'l coalition to fund “Global Gene Transfer for Batten Disease” research at the UNC Gene Therapy Center, announced Dr. Bruce Bloom.

STATegics, Inc. awarded a new grant from the Friedreich’s Ataxia Research Alliance (FARA) to advance erythropoietin receptor agonist compounds for the treatment of Friedreich’s ataxia (FRDA).

Fighting Back, the podcast that tells inspirational stories about people fighting serious or incurable diseases, announces that the latest episode features an interview with the retiring president of the National Organization for Rare Disorders.