We are proud to announce the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Cure Rare Disease's investigational anti-sense Oligonucleotide therapeutic for the treatment of Spinocerebellar Ataxia (SCA), including Spinocerebellar Ataxia Type 3 (SCA3). As noted by CRD founder and CEO, Rich Horgan, “receiving Orphan Drug Designation for our SCA3 program is an important milestone and a validation of our mission to deliver life-saving therapies to patients with ultra-rare and rare diseases. This designation not only provides key development incentives, including potential market exclusivity upon approval, but also accelerates our efforts to bring hope to families battling this devastating condition.” Learn more: https://www.cureraredisease.org/blog-posts/cure-rare-disease-receives-orphan-drug-designation-targeting-spinocerebellar-ataxia-type-3-sca3 (fb)

Our patient families and their communities continue to amaze us! The Falger-Jackson family and their crew are embarking on a 157-mile rowing journey to benefit CRD and our DMD research efforts. To support this incredible journey, visit www.wewillforwilliam.org/row for sponsorship and donation opportunities. (fb)