BOSTON, Sept. 30, 2024 -- Aktis Oncology, a clinical-stage biotechnology company pioneering the discovery and development of novel targeted alpha radiopharmaceuticals to treat a broad range of solid tumors, today...
Ultragenyx Initiates Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy
News | 07. 05. 2012
Ultragenyx Pharmaceutical
NOVATO, Calif., July 5, 2012 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc., a biotechnology company focused on developing treatments for rare and ultra-rare genetic disorders, today announced the dosing of the first two patients in a Phase 2 study of UX001 for hereditary inclusion body myopathy (HIBM). HIBM is a rare, severe, neuromuscular disease caused by sialic acid deficiency. UX001 is an extended-release oral tablet formulation of sialic acid (SA-ER) intended as a substrate replacement therapy for HIBM.
Read more here.
