The Sanofi Biopharmaceutical Company and the Into-Sana medical center network (Kyiv, Odesa) signed a Memorandum of ...

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

Sly syndrome, or Mucopolysaccharidosis Type VII (MPS VII), is a rare genetic disorder caused by a deficiency in the enzyme β-glucuronidase, leading to glycosaminoglycan accumulation in tissues and ...

Orchard Therapeutics, a Kyowa Kirin company, today announced the last patient has been treated in a registrational trial evaluating the efficacy and safety of OTL-203, an investigational hematopoietic ...

Mucopolysaccharidosis IV-A (MPS IV-A) is an autosomal recessive genetic disorder caused by a deficiency in the enzyme N-acetylgalactosamine-6-sulfatase, leading to the accumulation of ...

The Mucopolysaccharidosis I treatment market is experiencing significant expansion, driven by increasing disease awareness, rising prevalence, and b ...

Medipal Holdings-JCR Pharmaceuticals’ JR-446 for mucopolysaccharidosis type IIIB recevies EC orphan drug designation: Tokyo Thursday, June 26, 2025, 15:00 Hrs [IST] Medipal Hold ...

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

TOKYO & HYOGO, Japan, June 24, 2025--JCR and MEDIPAL announced that the EC has granted ODD to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB.

Children with mucopolysaccharidosis who receive adenotonsillectomy are at higher risk for respiratory complications and perioperative bleeding, have longer lengths of stay, and have higher healthcare ...