News

Proof-of-concept trial for Rett treatment expected this summer

Unravel Biosciences is asking authorities in Colombia to allow the launch — which could be as early as this summer, if cleared — of proof-of-concept clinical trials in the country to test RVL001, its treatment candidate for Rett syndrome. The company has submitted an application seeking regulatory clearance…

NLX-101 aids breathing issues, cognitive deficits in Rett mice

NLX-101 improves breathing and cognitive function in a female mouse model of Rett syndrome, a study found. The potential therapy activates the receptors that bind serotonin, a signaling molecule found at low levels in the brains of Rett patients. “This work provides compelling evidence of the therapeutic potential of…

Computer modeling confirms Daybue dosing regimen

Computer modeling confirms the weight-based dosing strategy for Daybue (trofinetide) to treat people with Rett syndrome, ages 2 and older. That’s according to two, back-to-back studies published separately in Advances in Therapy. Daybue, from Acadia Pharmaceuticals, is the first therapy approved in the U.S. for people with…