The Greenwood Genetic Center has designed a functional platform to assess the pathogenicity of variants of uncertain significance in IDUA identified by newborn screening for MPS I. With further ...

Two further clinical trials are underway in the U.S. to evaluate Sangamo's in vivo genome editing therapeutics for haemophilia B and MPS I, which is also known as Hurler or Hurler-Scheie syndrome.

There are currently seven known different types of MPS disorders: MPS I (Hurler-Scheie syndrome), MPS II (Hunter syndrome), MPS III (Sanfilippo syndrome), MPS IV (Morquio syndrome), MPS VI ...