This report also provides a detailed analysis of the current mucopolysaccharidosis marketed drugs and late-stage pipeline ...

However, the market is constrained by challenges such as complex implementation and high costs. Nevertheless, there are ...

Ultragenyx's high burn rate, costly R&D, and recent setbacks increase dilution risk, impacting future profitability. Learn ...

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 ...

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

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 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.

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 ...