After 10 Children with Rare Diseases died, Indian MP Varnum Gandhi Warns that more Children are in Danger
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After 10 Children with Rare Diseases died, Indian MP Varnum Gandhi Warns that more Children are in Danger

  New Delhi: Recent headlines in the Hindustan Times highlighted a letter written by the leader of the Bharatiya Janata Party, Varnum Gandhi, to Health Minister Mansukh Mandaviya urging him…

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Evaluating ST-920 for Fabry Disease: An Interview with Sangamo’s Bettina Cockroft, MD, MBA
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Evaluating ST-920 for Fabry Disease: An Interview with Sangamo’s Bettina Cockroft, MD, MBA

The European Society for Gene and Cell Therapy (ESGCT) recently held its Annual Congress from October 11-14, 2022. During the ESGCT Congress, various stakeholders discussed data, trends, and new research…

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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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