Promising Interim Data in Phase 1/2 Hunter Syndrome Trial
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Promising Interim Data in Phase 1/2 Hunter Syndrome Trial

Professionals from all corners of the world convene at SSIEM each year to discuss the latest groundbreaking discoveries. According to a report in BioSpace, one such breakthrough was presented by…

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RDLA Webinar: Updates on the VALID Act, RUSP Alignment, Newborn Screening, and Precision Medicine for Kids
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RDLA Webinar: Updates on the VALID Act, RUSP Alignment, Newborn Screening, and Precision Medicine for Kids

On September 28, 2022, the Rare Disease Legislative Advocates (RDLA) hosted their monthly webinar. These webinar help provide updates to the rare disease community on legislation and other policy initiatives…

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Experimental Gene Therapy Program for Hunter Syndrome (MPS II) Begins Expanding
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Experimental Gene Therapy Program for Hunter Syndrome (MPS II) Begins Expanding

According to a story from PR Newswire, the biotechnology company REGENXBIO Inc. has recently announced plans to expand its RGX-121 developmental program. RGX-121 is being developed as a potential one-time…

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Kate Middleton Visits Boys with Hunter Syndrome and Dystonic Cerebral Palsy
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Kate Middleton Visits Boys with Hunter Syndrome and Dystonic Cerebral Palsy

  East Anglia's Children's Hospices in Norfolk, U.K., has recently opened up its new facilities after renovations. To celebrate this opening, Kate Middleton, the Duchess of Cambridge, visited and spoke…

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