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

Desnick, R. J.; Sardh, Eliane; Harper, Pauline; Balwani, M.; Stein, P.; Rees, David A.; Bloomer, J.; Bissel, D.; Park, C.; Phillips, J.; Bonkovsky, Herbert L.; Anderson, Kathryn T.

doi:10.1016/j.revmed.2018.10.164

La Revue de Médecine Interne

2018

DOI: 10.1016/j.revmed.2018.10.164

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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

R. J. Desnick

Eliane Sardh

Pauline Harper

et al.

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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

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