JCR Pharmaceuticals said on February 12 that it has earned orphan drug designation for JR-171, a blood-brain-barrier (BBB)-penetrating α-L-iduronidase, for the treatment of mucopolysaccharidosis type 1 (Hurler, Hurler-Scheie, and Scheie syndrome).The company is running a PI/II trial globally for JR-171,…
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