Today, we’re pleased to share that we've received both orphan drug designation and rare pediatric disease designation from the FDA for ABS-0871, a TRPV4 inhibitor, for the treatment of Charcot-Marie-Tooth Disease 2C, or #CMT2C. Receiving these designations emphasizes the urgent need for treatments for CMT2C patients, who often live with debilitating symptoms like muscle weakness, vocal cord paresis and respiratory complications. We’re proud to be pursuing solutions for CMT2C with support from our STAR Alliance Partner, the Charcot-Marie-Tooth Association through their Patients as Partners platform. We will continue to advance this program as quickly as possible. Read more: https://lnkd.in/eaEKKVbV
Congratulations! What an amazing step forward!
Chief Executive Officer at Abner, Herrman & Brock LLC
1moGreat work!!! Congratulations!!!