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Cambridge, UK – 06 September 2023, 12:00 BST. Healx, the AI-powered, patient-inspired, techbio company pioneering the next generation of drug discovery has received Orphan Drug Designation from the US Food and Drug Administration (FDA) for its AI-discovered treatment of Neurofibromatosis Type 1 (NF1). Their team of expert scientists and technologists discovered HLX-1502 as a potential treatment … Continued

Five new Wikipedia articles were created on the day in the following rare diseases: SLC6A1 epileptic encephalopathy, Spastic paraplegia 15, Arboleda-Tham syndrome (KAT6A syndrome), KCNQ2 developmental and epileptic encephalopathy and HNRNPH2-related disorders. Two more draft pages were created for SATB2-associated syndrome and SLC13A5 citrate transporter disorder. Dr Clara Tang, Alliance Strategy Manager, who led the … Continued