This event is in the past.It took place on March 27, 2026 at Amphithéâtre Marie Curie.
Educational TalkEducational TalkFrom a single patient to a US FDA approved drug: the story of a drug repositioning for a rare disease
5th Arr.
Hear how one doctor turned a single patient case into an FDA-approved treatment for a rare genetic disorder. This talk traces the science behind repositioning an existing drug to treat CLOVES syndrome, complete with clinical results that changed nineteen lives.
Scientific lecture
Rare disease focus
Clinical breakthrough story
Research institute
Tips
🧬CLOVES syndrome involves PIK3CA gene mutations
💊BYL719 is the repurposed PIK3CA inhibitor
🏛️at Institut Curie's historic Marie Curie amphitheater
From a single patient to a US FDA approved drug: the story of a drug repositioning for a rare disease
5th Arr.
Educational TalkEducational TalkHear how one doctor turned a single patient case into an FDA-approved treatment for a rare genetic disorder. This talk traces the science behind repositioning an existing drug to treat CLOVES syndrome, complete with clinical results that changed nineteen lives.
Scientific lecture
Rare disease focus
Clinical breakthrough story
Research institute
Tips
🧬CLOVES syndrome involves PIK3CA gene mutations
💊BYL719 is the repurposed PIK3CA inhibitor
🏛️at Institut Curie's historic Marie Curie amphitheater
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