Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study

por Bello, Luca, Kesari, Akanchha, Gordish-Dressman, Heather, Cnaan, Avital, Morgenroth, Lauren P, Punetha, Jaya, Duong, Tina, Henricson, Erik K, Pegoraro, Elena, McDonald, Craig M, Hoffman, Eric P
Publicado 2015

Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study

por Boca, Simina M., Nishida, Maki, Harris, Michael, Rao, Shruti, Cheema, Amrita K., Gill, Kirandeep, Seol, Haeri, Morgenroth, Lauren P., Henricson, Erik, McDonald, Craig, Mah, Jean K., Clemens, Paula R., Hoffman, Eric P., Hathout, Yetrib, Madhavan, Subha
Publicado 2016

Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial

por Clemens, Paula R., Rao, Vamshi K., Connolly, Anne M., Harper, Amy D., Mah, Jean K., Smith, Edward C., McDonald, Craig M., Zaidman, Craig M., Morgenroth, Lauren P., Osaki, Hironori, Satou, Youhei, Yamashita, Taishi, Hoffman, Eric P.
Publicado 2020

Safety and effectiveness of ataluren: comparison of results from the STRIDE Registry and CINRG DMD Natural History Study

por Mercuri, Eugenio, Muntoni, Francesco, Osorio, Andrés Nascimento, Tulinius, Már, Buccella, Filippo, Morgenroth, Lauren P, Gordish-Dressman, Heather, Jiang, Joel, Trifillis, Panayiota, Zhu, Jin, Kristensen, Allan, Santos, Claudio L, Henricson, Erik K, McDonald, Craig M, Desguerre, Isabelle
Publicado 2020

Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022): 2022 interim...

por Mercuri, Eugenio, Osorio, Andrés Nascimento, Muntoni, Francesco, Buccella, Filippo, Desguerre, Isabelle, Kirschner, Janbernd, Tulinius, Már, de Resende, Maria Bernadete Dutra, Morgenroth, Lauren P., Gordish-Dressman, Heather, Johnson, Shelley, Kristensen, Allan, Werner, Christian, Trifillis, Panayiota, Henricson, Erik K., McDonald, Craig M.
Publicado 2023

Correction to: Safety and effectiveness of ataluren in patients with nonsense mutation DMD in the STRIDE Registry compared with the CINRG Duchenne Natural History Study (2015–2022)...

por Mercuri, Eugenio, Osorio, Andrés Nascimento, Muntoni, Francesco, Buccella, Filippo, Desguerre, Isabelle, Kirschner, Janbernd, Tulinius, Már, de Resende, Maria Bernadete Dutra, Morgenroth, Lauren P., Gordish-Dressman, Heather, Johnson, Shelley, Kristensen, Allan, Werner, Christian, Trifillis, Panayiota, Henricson, Erik K., McDonald, Craig M.
Publicado 2023

Correction: Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study

por Boca, Simina M., Nishida, Maki, Harris, Michael, Rao, Shruti, Cheema, Amrita K., Gill, Kirandeep, Wang, Difei, An, Lin, Gauba, Robinder, Seol, Haeri, Morgenroth, Lauren P., Henricson, Erik, McDonald, Craig, Mah, Jean K., Clemens, Paula R., Hoffman, Eric P., Hathout, Yetrib, Madhavan, Subha
Publicado 2016

Serum pharmacodynamic biomarkers for chronic corticosteroid treatment of children

por Hathout, Yetrib, Conklin, Laurie S., Seol, Haeri, Gordish-Dressman, Heather, Brown, Kristy J., Morgenroth, Lauren P., Nagaraju, Kanneboyina, Heier, Christopher R., Damsker, Jesse M., van den Anker, John N., Henricson, Erik, Clemens, Paula R., Mah, Jean K., McDonald, Craig, Hoffman, Eric P.
Publicado 2016

Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study

por Smith, Edward C., Conklin, Laurie S., Hoffman, Eric P., Clemens, Paula R., Mah, Jean K., Finkel, Richard S., Guglieri, Michela, Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Castro, Diana, Kuntz, Nancy L., Kerchner, Laurie, Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Jaros, Mark, Shale, Phil, Gordish-Dressman, Heather, Hagerty, Laura, Dang, Utkarsh J., Damsker, Jesse M., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., McDonald, Craig M.
Publicado 2020

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function

por Hoffman, Eric P., Schwartz, Benjamin D., Mengle-Gaw, Laurel J., Smith, Edward C., Castro, Diana, Mah, Jean K., McDonald, Craig M., Kuntz, Nancy L., Finkel, Richard S., Guglieri, Michela, Bushby, Katharine, Tulinius, Mar, Nevo, Yoram, Ryan, Monique M., Webster, Richard, Smith, Andrea L., Morgenroth, Lauren P., Arrieta, Adrienne, Shimony, Maya, Siener, Catherine, Jaros, Mark, Shale, Phil, McCall, John M., Nagaraju, Kanneboyina, van den Anker, John, Conklin, Laurie S., Cnaan, Avital, Gordish-Dressman, Heather, Damsker, Jesse M., Clemens, Paula R.
Publicado 2019