PTC Therapeutics Earns FDA Nod for SEPHIENCE™: A New Hope for Phenylketonuria (PKU) Patients

PTC Therapeutics has achieved a significant milestone with the U.S. Food and Drug Administration’s (FDA) approval of SEPHIENCE™ (sepiapterin), marking the first and only therapy approved for broad treatment of…

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Clinical Trial to Evaluate HMI-103 for PKU
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Clinical Trial to Evaluate HMI-103 for PKU

Previously, clinical-stage genetic medicines company Homology Medicines, Inc. ("Homology") submitted an Investigational New Drug (IND) application to the FDA for HMI-103, an investigational gene editing therapy designed to treat phenylketonuria…

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Data Collaboration Tool for Accelerating Rare Disease Research Celebrates Success in First Year

Rare Disease Cures Accelerator-Data and Analytics Platform (RDCA-DAP) The RDCA-DAP, funded by the FDA, was first launched in September of 2019. Its aim was to make data more accessible for…

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