The FDA cited manufacturing issues but did not flag problems with Ultragenyx’s data package for UX111, with the biotech ...

Explore the competitive landscape of Mucopolysaccharidosis I (Hurler syndrome) Therapeutics. In 2024, over 1,740 cases were diagnosed across 16 countries. With one FDA-approved drug, the pipeline ...

Explore the competitive landscape of Mucopolysaccharidosis I (Hurler syndrome) Therapeutics. In 2024, over 1,740 cases were diagnosed across 16 countries. With one FDA-approved drug, the pipeline ...

JCR and MEDIPAL announced that the EC has granted ODD to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB.

Explore the competitive landscape of Mucopolysaccharidosis I (Hurler syndrome) Therapeutics. In 2024, over 1,740 cases were diagnosed across 16 count ...

Mucopolysaccharidosis type II (MPS II, or Hunter syndrome) is an X-linked recessive lysosomal storage disorder caused by a deficiency of iduronate-2-sulfatase, an enzyme that breaks down complex ...

Mucopolysaccharidosis type IIIB, or Sanfilippo syndrome type B, is an autosomal recessive disease caused by pathogenic mutations in the NAGLU gene, encoding a lysosomal enzyme involved in the ...

HYOGO, Japan, June 18, 2025--JCR announced the launch of a film featured in The Next Frontier, a series presented by BIO and produced by BBC StoryWorks Commercial Productions.

TOKYO& HYOGO, Japan---- MEDIPAL HOLDINGS CORPORATION and JCR Pharmaceuticals Co., Ltd. today announced that the European Commission has granted orphan drug designation to JR-446, an ...

JCR Pharmaceuticals Co, a global specialty biopharmaceutical company dedicated to developing therapies for rare and genetic diseases, announced the launch of a film featured in The Next Frontier, a ...

Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial ...