As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has launched an investigation into reports of serious adverse events, including one fatality, associated with Takeda’s enzyme replacement therapy…
Three-year-old Oliver Chu has stunned clinicians after becoming the first person with Hunter syndrome (MPS II) to receive an experimental gene therapy and showing striking early gains. Hunter syndrome is…
Editor's Note: Patient Worthy is pleased to share this article from our friends at Elephants & Tea, originally written by Cecily Liu. To see the article in its original format,…
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