Page 2 - Inborn Errors News Today

- 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...

JNT-517 is a Potential First-in-Class Oral Treatment for PKU; Phase 1b Study Follows Positive Clinical Proof of Mechanism Data in Healthy Individuals

Continued progress on 5x25 strategy to advance five AAV Therapeutics from REGENXBIOs internal pipeline and licensed programs into pivotal-stage or...

Cash, cash equivalents and current financial assets totaled €140.2 million1 as of December 31, 2022, expected to fund operations through third quarter 2024Transformative milestone expected towards end

4Q2022 Total Revenues of $50 Million, a 90% Y-o-Y Increase 4Q2022 Gross Profit of $14 Million, an $11 Million Y-o-Y Increase FY 2023 Total Revenue Expected to Exceed $210 Million ... | March 23, 2023

Scientists at the Icahn School of Medicine at Mount Sinai in New York have identified which parts of the immune system go awry and contribute to autoimmune diseases in individuals with Down syndrome.