PLYMOUTH MEETING, Pa., September 24, 2025--(BUSINESS WIRE)--Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced topline results from its Phase 3 registrational clinical trial (the ...

Harmony also touts late-stage asset EPX-100 (clemizole hydrochloride), which the biotech is running through a pair of phase 3 trials in Dravet syndrome and Lennox-Gastaut Syndrome— both rare forms of ...

University of Utah and Primary Children’s Hospital have created a FXS clinic to care for patients and families living with Fragile X. Our clinic serves the children of Utah and surrounding states ...

Harmony Biosciences’ $60 million bet on a once-failed Fragile X candidate has gone off the rails. Two years after picking up ZYN002 despite a midphase flop, the biotech has reported that a ...

For 22 years, Jason Mazzola's life was defined by Fragile X, a genetic condition that often causes autism and intellectual disability. Jason, who is 24 now, needed constant supervision. He had ...

Harmony Biosciences has paused a mid-stage trial of ZYN002 in 22q11.2 deletion syndrome after the THC-free cannabinoid drug ...

Fragile X syndrome, the most common form of inherited intellectual disability, may be unfolding in brain cells even before birth, despite typically going undiagnosed until age 3 or later. A new study ...

Joel Richter receives funding from NIH and FRAXA. Sneha Shah receives funding from the FRAXA Research Foundation. Fragile X syndrome is a genetic disorder caused by a mutation in a gene that lies at ...