The Impact of COVID-19 on Patients with Thalassemia and Sickle Cell Disease: A Webinar Review
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The Impact of COVID-19 on Patients with Thalassemia and Sickle Cell Disease: A Webinar Review

  As COVID-19 continues to spread across the globe, more and more people are wondering how this virus might affect them. This is especially true for patients with rare diseases,…

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Review of Rare Movement Disorders in India and the United States

A recent article published in India’s Pharma Literati cites The World Health Organization as not having endorsed a definition for rare diseases. The European Union defines 'rare' as less than…

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An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit
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An In-Depth Look at the European Medicines Agency’s Orphan Drug Designation Post-Brexit

  On February 3, 2020, twenty-seven newly installed flags of the European Union stood in the gleaming lobby of the new European Medicines Agency (EMA) headquarters in Amsterdam. The Charcot-Marie-Tooth…

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Early Data Looks Encouraging for an Experimental Thalassemia Treatment
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Early Data Looks Encouraging for an Experimental Thalassemia Treatment

According to a story from globenewswire.com, the drug developer Agios Pharmaceuticals, Inc. has recently released a preliminary analysis of phase 2 trial findings that have served to establish proof-of-concept for…

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