On July 18, 2025, the U.S. Food and Drug Administration (FDA) took decisive action against Sarepta Therapeutics following the deaths of three individuals who had received the company’s gene therapy…
Patient Worthy is honored to share Oliver's story on behalf of SynGAP Research Fund. SRF is a global group of families committed to accelerating the science to cure SYNGAP1 &…
One death is too many - Especially when it can be prevented. The new MGB policy will benefit patients who are at risk of deadly toxicities from a widely prescribed…
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