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 ...

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 ...

Analysts provide deeper insights through their assessments of 12-month price targets, revealing an average target of $64.75, a high estimate of $65.00, and a low estimate of $64.00. This current ...

First rare disease candidates to benefit from the FDA START programme are from Denali Therapeutics, Neurogene, Larimar Therapeutics, and Grace Science ...

Background Mucopolysaccharidoses (MPS) are monogenic metabolic disorders that significantly affect the skeleton. Eleven enzyme defects in the lysosomal degradation of glycosaminoglycans (GAGs) have ...

Morquio Syndrome, or Mucopolysaccharidosis Type IV (MPS IV), is a rare genetic disorder causing glycosaminoglycans (GAGs) buildup due to enzyme deficiencies, leading to severe skeletal and organ ...

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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 ...

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