Neuren Pharmaceuticals Ltd.
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Latest From Neuren Pharmaceuticals Ltd.
Acadia’s Daybue Gets First Approval In Rett Syndrome
Daybue is the first drug approved to treat the rare neurodevelopmental disorder Rett syndrome, despite high clinical trial incidence of diarrhea. Product labeling includes clinician guidance for the side effect.
With Hopeful Interim Data, Ultragenyx Bets On GeneTx, Angelman Syndrome Candidate
Ultragenyx is buying its Angelman syndrome partner GeneTx for $75m on the heels of an interim Phase I/II readout that shows signs of clinical activity and appears to resolve earlier safety worries.
Acadia’s Trofinetide Shows Efficacy In Rett Syndrome, But Safety Looks Shaky
Trofinetide could be the first approved drug for Rett patients after hitting both co-primary endpoints in a Phase III trial, but high rates of diarrhea and vomiting raise questions.
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