dm+d

Unassigned

New Medicines

Mucopolysaccharidosis VII (MPS VII - Sly disease)

Information

New molecular entity
Ultragenyx
Ultragenyx

Development and Regulatory status

Phase II Clinical Trials
Phase II Clinical Trials
Phase III Clinical Trials

Category

Recombinant human beta-glucuronidase (rhGUS) enzyme replacement therapy. MPS VII is a genetic disorder characterised by a deficiency of beta-glucuronidase, leading to progressive accumulation of toxic glycolipids in various tissues and organs.
In the UK, Sly Disease is the rarest MPS with an average of only one baby every 10 years born with the condition. Worldwide it is estimated to have a frequency of less than 1:250,000 births. In its most extreme form, children are born with a condition called hydrops fetalis. Babies with hydrops fetalis rarely survive beyond a few weeks to a few months of age. Many individuals with Sly Disease are less severely affected and have clinical symptoms similar to other MPS [1].
Mucopolysaccharidosis VII (MPS VII - Sly disease)
Intravenous infusion