Neurogene: Potential To Target Root Cause Of Rett Syndrome With NGN-401
NGNE to update on a potential registrational phase 3 study, using NGN-401, for the treatment of patients with Rett Syndrome.
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Autophagy malfunction study moves a step closer to understanding Rett syndrome
Rett syndrome is a rare genetic disease affecting 1 in 10,000 newborn girls, characterized by sudden regression around 1 year of age, with loss of acquired language and motor skills and leading to ...
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Minoryx doses first patient with leriglitazone in Rett syndrome study
Phase 2a TREE study assesses safety and efficacy of leriglitazone Minoryx Therapeutics has dosed the first patient in its phase 2a TREE study, which evaluates the safety and efficacy of leriglitazone ...
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Acadia Pharmaceuticals Announces Positive DAFFODIL Study Data For DAYBUE In Rett Syndrome
(RTTNews) - Acadia Pharmaceuticals Inc. (ACAD) announced the publication of clinical data from the open-label DAFFODIL study, which evaluated the long-term safety and efficacy of DAYBUE or ...