Rare Community Profiles Rare Community Profiles is a new Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their…
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,…
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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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…
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…
La Jolla Pharmaceutical recently announced a new clinical trial targeting thalassemia. The trial, described as pivotal, investigates a drug referred to as LJPC-401. The treatment is intended to help patients…
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