SINGAPORE--March 31, 2023--Horizon Quantum Computing, a Singapore-based company building software development tools to unlock the potential of quantum computing hardware, announced today that it has raised USD 18.1 million Series A...
Ultragenyx Initiates Phase 2 Study of UX001 in Hereditary Inclusion Body Myopathy
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.
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