According to a story from BioPharma Dive, the US Food and Drug Administration (FDA) has recently approved Beqvez, a gene therapy from pharmaceutical giant Pfizer intended to treat hemophilia B.…
I have lived with chronic pain from dystonia, a neurological movement disorder, for 23 years. The first 5 years my symptoms were so severe that I was barely able to…
Rare Community Profiles is a Patient Worthy article series of long-form interviews featuring various stakeholders in the rare disease community, such as patients, their families, advocates, scientists, and more.…
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