As reported on Drugs.com, REGENXBIO has announced that the U.S. Food and Drug Administration (FDA) has halted clinical testing of its investigational gene therapies RGX‑111 and RGX‑121, two programs targeting…
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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