Note: These quotes and reflections are from members of the cystinosis community who were in attendance at the 2023 Cystinosis Research Network Family Conference which took place in Nashville, TN…
In 2020, the U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy designation to magrolimab for the treatment of newly diagnosed myelodysplastic syndromes (MDS). This designation is designed to expedite…
According to a story from Market Screener, the drug company AbbVie recently announced that its investigational therapy epcoritamab has received a positive opinion from the European Union's Committee for Medicinal…
There are an estimated 10,000 rare diseases. A majority of rare diseases are genetically oriented. Because of this, we learn more about health and disease as we learn more…
According to a story from WGN-TV Chicago, treatment for esophageal cancer can be grueling on patients, even if successful. Surgery is often a critical aspect of the process and can…
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…
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