Five-Year Extension Results of the Phase 1 START Trial of Onasemnogene Abeparvovec in Spinal Muscular Atrophy

por Mendell, Jerry R., Al-Zaidy, Samiah A., Lehman, Kelly J., McColly, Markus, Lowes, Linda P., Alfano, Lindsay N., Reash, Natalie F., Iammarino, Megan A., Church, Kathleen R., Kleyn, Aaron, Meriggioli, Matthew N., Shell, Richard
Publicado 2021

Anti-HMGCR myopathy may resemble limb-girdle muscular dystrophy

por Mohassel, Payam, Landon-Cardinal, Océane, Foley, A. Reghan, Donkervoort, Sandra, Pak, Katherine S., Wahl, Colleen, Shebert, Robert T., Harper, Amy, Fequiere, Pierre, Meriggioli, Matthew, Toro, Camilo, Drachman, Daniel, Allenbach, Yves, Benveniste, Olivier, Béhin, Anthony, Eymard, Bruno, Lafôret, Pascal, Stojkovic, Tanya, Mammen, Andrew L., Bönnemann, Carsten G.
Publicado 2018