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Stock impact report

Mirum Pharmaceuticals Announces First Patient Enrolled in the BLOOM Phase 2 Clinical Study Evaluating MRM-3379 in Fragile X Syndrome

Mirum Pharmaceuticals, Inc. - common stock (MIRM) 
Last mirum pharmaceuticals, inc. - common stock earnings: 3/12 04:02 pm Check Earnings Report
Company Research Source: Business Wire
Proof-of-concept study underway for investigational oral therapy in Fragile X syndrome; top-line data from Phase 2 expected in 2027 FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that the first participant has been enrolled in the BLOOM Phase 2 clinical study evaluating MRM-3379 in Fragile X syndrome (FXS).Caused by a mutation of the FMR1 gene, FXS is the most common inherited form of intellectual disability and autism spectrum disorder and affects an estimated 50,000 males in the U.S. and E.U. There are currently no approved therapies for the treatment of this serious, neurodevelopmental condition.The BLOOM Phase 2 Clinical Study will evaluate the safety, tolerability, and potential clinical benefit of MRM-3379 in male participants with a confirmed genetic diagnosis of FXS.“Despite its impact, Fragile X syndrome remains without an approved therapy,” said Joanne Quan, M.D., Chief Medical Officer at M Show less Read more
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