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Étude de phase 1/2 et d’extension en ouvert du Givosiran, un agent thérapeutique ARNi expérimental chez des patients atteints de porphyrie aiguë intermittente

Year: 2018

Type: article

Study design: Clinical Trial

Source: La Revue de Médecine Interne

DOI: 10.1016/j.revmed.2018.10.164

Authors: R. Desnick, E. Sardh, P. Harper, M. Balwani, P. Stein, D. Rees, J. Bloomer, D. Bissel, C. Park, J. Phillips, H. Bonkovsky, K. Anderson

Institutions: Mount Sinai Beth Israel, Karolinska Institutet, King's College London, University of Alabama at Birmingham Hospital, University of Alabama at Birmingham, UCSF Benioff Children's Hospital, Atrium Health Wake Forest Baptist, The University of Texas Medical Branch at Galveston

Language: French

Topic: Porphyrin Metabolism and Disorders

Subfield: Molecular Biology

Field: Biochemistry, Genetics and Molecular Biology

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: closed

OpenAlex ID: W2902541618

FWCI:

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