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

Jobsite Fan Market projected to reach USD 1.43 billion by 2035, driven by demand for workplace safety, energy efficiency, and smart ventilation solutions. ROCKVILLE, MD , MD, UNITED STATES, July 15, ...

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

During the two decades that lapsed since “ironome” was coined for the ensemble of proteins associated with iron metabolism, a surge of iron-driven biological processes have been recognized as ...

Following our analysis of the options activities associated with Ultragenyx Pharmaceutical, we pivot to a closer look at the company's own performance. With a volume of 9,702,498, the price of RARE is ...

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

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

MEDIPAL HOLDINGS CORPORATION (TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd. (TSE 4552, JCR) today announced that the European Commission (EC) has granted orphan drug designation (ODD) to JR-446 ...

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 ...

Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by European Commission (EC) ...

Reference: Press release on the orphan drug designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA (May 7, 2025). Based on data from JCR’s own investigations, referring to the ...

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 ...