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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Investigational PKU Treatment BMN 307 Given Fast Track Designation

On October 2, 2020, biotechnology company BioMarin Pharmaceutical ("BioMarin") announced that its investigational gene therapy candidate BMN 307 received the Fast Track designation from the FDA. The therapy is designed…

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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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Gaucher Disease Patient and Activist Successfully Improves the Care for Other Rare Disease Patients in Her Country

This is the story of a brave journalist who was determined to improve care for rare disease patients in her country, North Macedonia. North Macedonia is a developing country located…

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