Phase 2 Fabry Disease Clinical Trial Has Been Stopped Due to Negative Outcomes
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Phase 2 Fabry Disease Clinical Trial Has Been Stopped Due to Negative Outcomes

Avrobio has just announced that they will be stopping work on their investigative gene therapy for Fabry disease based on unexpected and disappointing results from a Phase 2 clinical trial.…

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14th ICIEM Conferences: Data to be Presented on Fabry Disease and Gaucher Disease Trials
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14th ICIEM Conferences: Data to be Presented on Fabry Disease and Gaucher Disease Trials

From November 21st until the 23rd, medical professionals and others involved in the metabolic field will come together for the 14th International Congress of Inborn Errors of Metabolism (ICIEM). It…

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EC Approves Galafold for Adolescent Fabry Disease with an Amenable Mutation
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EC Approves Galafold for Adolescent Fabry Disease with an Amenable Mutation

On August 2, 2021, biotechnology company Amicus Therapeutics ("Amicus") shared that its oral treatment Galafold (migalastat) was approved by the European Commission (EC) for the treatment of adolescent patients (ages…

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Rare Classroom: Fabry Disease
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Rare Classroom: Fabry Disease

Welcome to the Rare Classroom, a new series from Patient Worthy. Rare Classroom is designed for the curious reader who wants to get informed on some of the rarest, most…

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American Kidney Fund and Sanofi Genzyme Partner up for New Fabry Disease Campaign
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American Kidney Fund and Sanofi Genzyme Partner up for New Fabry Disease Campaign

According to a press release from the American Kidney Fund (AKF), the organization, which is dedicated to fighting kidney disease, has partnered with the biotech company Sanofi Genzyme in a…

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Experimental Treatment for Fabry Disease to be Made Available Under Expanded Access
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Experimental Treatment for Fabry Disease to be Made Available Under Expanded Access

According to a story from PR Newswire, Chiesi Global Rare Diseases and the biopharmaceutical company Protalix BioTherapeutics, Inc. have recently announced that they have launched an Expanded Access Program (EAP)…

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Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis
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Early Success in Trials for Experimental Gene Therapies for Fabry Disease and Cystinosis

As reported in Business Fortnight, the top level gene therapy company AVROBIO has announced successful clinical trials for investigational drugs for Fabry disease and cystinosis. The CEO of AVROBIO, Geoff…

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