The US Food and Drug Administration approved a Duchenne muscular dystrophy drug yesterday (December 13) that it had originally rejected in August, reports STAT. The
A clinical trial of a gene therapy for Duchenne muscular dystrophy has been halted after a patient suffered serious side effects following treatment, Reuters reports
Researchers have been studying the CRISPR-Cas9 gene-editing system as a potential therapeutic tool to modify or delete pathogenic sequences within the human genome. Now, using
Last year, Elizabeth Parrish, the CEO of Seattle-based biotech firm BioViva, hopped a plane to Colombia, where she received multiple injections of two experimental gene
An advisory panel to the US Food and Drug Administration (FDA) convened Tuesday (November 24) to evaluate the strength of evidence for drisapersen, a drug
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