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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The Early Results of This Gene Therapy Trial for Phenylketonuria Look Encouraging
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The Early Results of This Gene Therapy Trial for Phenylketonuria Look Encouraging

According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc., has recently released promising early data from its phase 1/2 clinical trial. This clinical trial is testing…

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Preclinical Findings for Phenylketonuria and Metachromatic Leukodystrophy Gene Therapies Look Promising

According to a story from BioSpace, the genetic medicines company Homology Medicines, Inc., has recently released preclinical data supporting the development of two experimental gene therapies. One, called HMI-202, is…

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