BioMarin, a San Francisco area rare disease biopharma, reported China's NMPA approved Vimizim® (elosulfase alfa) to treat patients with mucopolysaccharidosis type IVA (MPS IVA), a rare disease also known as Morquio A syndrome. In 2018, China issued its first national list of rare diseases that have no treatment in China (including MPS), promising expedited review of any drugs that addressed these rare conditions. Vimizim is one of the first rare disease drugs from the list to be accepted for China use. More details....
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Original Article: BioMarin's Very-Rare Disease Treatment Approved in China