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Nearly 98% of patients experience quality of life impact as a result of tardive dyskinesia including social, psychological, physical and recreational aspects of daily living Data highlight for...
- In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose-Dependent Splicing Correction, Muscle Delivery and DMPK Knockdown - - All Evaluable Patients in the 3.4 mg/kg Cohort Treated with DYNE-101...
- In Phase 1/2 ACHIEVE Trial, DYNE-101 Demonstrated Dose-Dependent Splicing Correction, Muscle Delivery and DMPK Knockdown -
Dyne Therapeutics, Inc. announced positive initial clinical data from its ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 and its DELIVER trial of DYNE-251 in patients with...
Positive opinion from Committee for Medicinal Products for Human Use recommending approval of Bylvay® (odevixibat) based on Phase III ASSERT clinical-trial data in Alagille syndrome (ALGS) already received in July 2023Committee for Orphan Medicinal Products confirms a negative opinion of its review recommending not to maintain the orphan designation for Bylvay in ALGSIpsen plans to submit a new Marketing Authorisation Application for the treatment of ALGS by the end of 2023 under a new brand nam