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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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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Company Gears Up to Test a Gene Therapy for Phenylketonuria
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Company Gears Up to Test a Gene Therapy for Phenylketonuria

According to a story from BioSpace, the biotechnology company BioMarin Pharmaceutical Inc. has recently announced its submission of a clinical trial application with the UK's Medicines and Healthcare Products Regulatory…

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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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Phase 1/2 Trial for an Experimental Phenylketonuria Gene Therapy Has Begun Enrollment

According to a story from globenewswire.com, the genetic medicines company Homology Medicines, Inc. has recently announced that it has opened enrollment for its phase 1/2 clinical trial. This clinical trial…

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Phenylketonuria Patient Didn’t Receive Life-Altering Treatment for 10 Years, Alludes to Bigger Problem

Within the pharmaceutical industry, we need 1) better policies for patients and 2) improved communication/education of the policies that do exist for patients and the rights that they have. Ultimately,…

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This Phenylketonuria Patient Helped Test a Breakthrough Therapy Only to Lose Access Once the Trial Ended
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This Phenylketonuria Patient Helped Test a Breakthrough Therapy Only to Lose Access Once the Trial Ended

According to a story from BBC, Louise Moorhouse gets the nutrition that she needs through two sources: pills and foul tasting nutrient shakes. This is because she was born with…

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FDA Gives Go Ahead for Clinical Trial of Phenylketonuria Gene Therapy
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FDA Gives Go Ahead for Clinical Trial of Phenylketonuria Gene Therapy

According to a story from Market Screener, the genetic medicines company Homology Medicines, Inc. recently announced that the US Food and Drug Administration (FDA) has approved the company's application to…

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Committee Calls for the Approval of a New Treatment for Phenylketonuria in Europe
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Committee Calls for the Approval of a New Treatment for Phenylketonuria in Europe

The European Medicines Agency (EMA) recently published a press release in which the agency's human medicines committee, known as CHMP, as recommended the authorization of a new treatment for a…

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