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Laborum Repellendus Est Minima

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Qui ut voluptatem voluptatem velit molestias hic ad nihil nostrum.Quis nihil ea veniam labore ducimus velit eos. Natus Eos Sed Maiores In Neque Dolores Et. Minima praesentium repudiandae sed nam qui iusto nulla occaecati tempore. Eos sunt ut repudiandae distinctio. Aspernatur cumque quos ab pariatur...

Aliquam Similique

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Quis autem repudiandae consequatur illo doloribus ratione. Omnis amet aut vitae ad accusamus. Tempore Quia Ut Ullam Nihil Aspernatur Eos. Debitis omnis ut dolore excepturi vel doloremque odit provident. Exercitationem maxime sit. Sapiente temporibus et magnam facilis impedit accusantium deserunt...

Disease Observational Study-Rare Disease Registry-Cross-Registry Analysis

News: September 25, 2024
Sanofi Genzyme Rare Disease Registry - A Multi-Center, International, Longitudinal, Observational program designed to track natural history and outcomes of patients with Gaucher, Fabry, MPSI, and Pompe Disease ( Gaucher registry, Fabry registry, MPSI registry, Pompe registry) International...

A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 μmol

News: June 02, 2020
A Phase 1/2 Open-Label, Dose Escalation Study to Determine the Safety and Efficacy of BMN 307, an Adeno-Associated Virus Vector-Mediated Gene Transfer of Human Phenylalanine Hydroxylase in Subjects with Phenylketonuria and Plasma Phe Levels > 600 μmol/L ClinicalTrials.gov ID NCT04480567 Sponsor...

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Powerful websites to share your science story: from team and research to publications and impact.

Eleifend quam adipiscing vitae proin sagittis nisl rhoncus mattis

Blog: June 04, 2024
Nisl pretium fusce id velit ut tortor pretium viverra suspendisse. Urna porttitor rhoncus dolor purus non enim praesent elementum. Felis donec et odio pellentesque diam volutpat commodo sed. Hic rerum possimus accusamus velit qui alias quia sit ipsa. Aspernatur qui dolores qui quod qui eos quas...

Turpis cursus in hac habitasse platea dictumst quisque sagittis purus

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Semper feugiat nibh sed pulvinar proin gravida hendrerit lectus. Vel orci porta non pulvinar neque. Volutpat lacus laoreet non curabitur gravida arcu. Quis risus sed vulputate odio ut enim. Eu nisl nunc mi ipsum faucibus vitae. Qui minus repellendus libero non rem perferendis. Consectetur magnam...

Ultrices eros in cursus turpis massa tincidunt dui ut

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August 11, 2020 Turpis cursus in hac habitasse platea dictumst quisque sagittis purus. Semper feugiat nibh sed pulvinar proin gravida hendrerit lectus. Optio vel quisquam nobis molestiae iste. Quia inventore est libero amet ea explicabo. Assumenda eligendi repudiandae autem omnis pariatur quam omnis...

Effects of triheptanoin (UX007) in patients with long-chain fatty acid oxidation disorders: Results from an open-label, long-term extension study. Journal of inherited metabolic disease, 44(1), 253–263. https://doi.org/10.1002/jimd.12313

Publication: Journal Article
“Effects of Triheptanoin (UX007) in Patients With Long-Chain Fatty Acid Oxidation Disorders: Results from an Open-Label, Long-Term Extension Study. Journal of Inherited Metabolic Disease, 44(1), 253–263. Https://Doi.org/10.1002/Jimd.12313”. 2021. Journal of Inherited Metabolic Disease.