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- Total of six poster presentations analyze the effects of treatment with enzyme replacement therapies in Fabry disease and alpha-mannosidosis -- ...
JNT-517 demonstrated sustained, dose-dependent pharmacodynamic effects and was safe and well tolerated in healthy volunteers Phase 1b study is ongoing in individuals with PKU BOSTON, Aug. 31, 2023 (GLOBE NEWSWIRE) --
Additional biomarker data up to two years of treatment continue to demonstrate rapid and sustained normalization of CSF heparan sulfate to normal healthy levels and improvement in lysosomal function...
- Results from the APHENITY trial in PKU and long-term extension study including Phe tolerance to be highlighted -
SAN DIEGO, Aug. 24, 2023 -- Travere Therapeutics, Inc. today announced that the Company will present clinical data from the Phase 1/2 COMPOSE Study of pegtibatinase, a novel investigational enzyme...