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FDA has placed clinical holds on its RGX-111 and RGX-121 gene therapy programs for MPS I and MPS II after a preliminary analysis identified a single case of an intraventricular CNS tumor in a child treated with RGX-111 four years earlier

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This start low, go slow approach is essential because MCAS patients are notoriously sensitive to medications

PlexusDx also accepts HSA/FSA funds and serves all 50 states without requiring insurance or membership fees

Author disclosures Mohit Kehar MD is a consultant for Mirum

However, animal studies done with Wegovy suggest that there may be risks to using it