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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PTC Therapeutics to Acquire Censa Pharmaceuticals, Developer of an Experimental Phenylketonuria Drug
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PTC Therapeutics to Acquire Censa Pharmaceuticals, Developer of an Experimental Phenylketonuria Drug

According to a story from Xconomy, the drug company PTC Therapeutics has come to an agreement that will allow it to acquire Censa Pharmaceuticals and its lead product candidate. This…

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Support “The Making of Justice” a Documentary About Seeking Treatment with a Rare Disease
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Support “The Making of Justice” a Documentary About Seeking Treatment with a Rare Disease

When Jennifer Payne was born in 1973, she was diagnosed with a rare disease: phenylketonuria (PKU). This genetic disorder is typically screened for at birth. This early screening process is…

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