Post by CLIRNET

The U.S. FDA has approved the 𝐟𝐢𝐫𝐬𝐭-𝐞𝐯𝐞𝐫 𝐭𝐡𝐞𝐫𝐚𝐩𝐲 for 𝐩𝐞𝐝𝐢𝐚𝐭𝐫𝐢𝐜 𝐌𝐞𝐧𝐤𝐞𝐬 𝐝𝐢𝐬𝐞𝐚𝐬𝐞—a rare genetic disorder that impairs copper absorption.⁣ ⁣ This treatment delivers bioavailable copper and can 𝐫𝐞𝐝𝐮𝐜𝐞 𝐦𝐨𝐫𝐭𝐚𝐥𝐢𝐭𝐲 𝐫𝐢𝐬𝐤 by 𝟕𝟖%, especially when started early. Some patients have survived beyond a decade.⁣ ⁣ A major breakthrough—bringing long-awaited hope to affected children and their families.⁣ ⁣ #FDA #MenkesDisease #RareDisease #PediatricCare #PediatricNeurology #MetabolicDisorders #ChildHealth