According to a recent article, the family of a young boy from Australia who is diagnosed with the rare genetic disease mucopolysacchariodosis type 1 (MPS 1) is sharing his story to…
According to a story from sectorpublishingintelligence.co.uk, the biopharmaceutical company Orchard Therapeutics recently announced the release of some data from an ongoing proof-of-concept study. This special trial is testing the company's…
The Virginia Newborn Screening Advisory Committee met late last week to determine the fate of Pompe Disease and MPS-1 screening in VA. Before the vote took place, some families spoke…
The fate of newborn screening for Pompe disease and MPS-1 throughout the state of VA will be determined next week. The VA Newborn Screening Advisory Committee meeting will take place…
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