Reply to F. Muntoni et al.: “In response to P.R. Clemens et al., Efficacy and Safety of Viltolarsen in Boys with Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label,...

por Clemens, Paula R., Hoffman, Eric P.
Publicado 2023

Concerns Regarding Therapeutic Implications of Very Low‐Level Dystrophin

por Hoffman, Eric P., Clemens, Paula R.
Publicado 2021

Exon-Skipping in Duchenne Muscular Dystrophy

por Takeda, Shin’ichi, Clemens, Paula R., Hoffman, Eric P.
Publicado 2021

Effect of rapamycin on immunity induced by vector-mediated dystrophin expression in mdx skeletal muscle

por Eghtesad, Saman, Jhunjhunwala, Siddharth, Little, Steven R., Clemens, Paula R.
Publicado 2012

Effects of irradiation of adult mdx mice prior to full-length dystrophin cDNA transfer

por Eghtesad, Saman, Zheng, Heng, Nakai, Hiroyuki, Epperly, Michael W., Clemens, Paula R.
Publicado 2010

Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys

por Dang, Utkarsh J, Ziemba, Michael, Clemens, Paula R, Hathout, Yetrib, Conklin, Laurie S, Hoffman, Eric P
Publicado 2020

Modeling Early Heterogeneous Rates of Progression in Boys with Duchenne Muscular Dystrophy

por Fang, Yuan, McDonald, Craig M., Clemens, Paula R., Gordish, Heather-Dressman, Illei, Kate, Hoffman, Eric P., Dang, Utkarsh J.
Publicado 2023

Improvement of the mdx mouse dystrophic phenotype by systemic in utero AAV8 delivery of a minidystrophin gene

por Koppanati, Bhanu Munil, Li, Juan, Reay, Dan P., Wang, Bing, Daood, Molly, Zheng, Heng, Xiao, Xiao, Watchko, Jon F., Clemens, Paula R.
Publicado 2010

Neurodevelopmental Needs in Young Boys with Duchenne Muscular Dystrophy (DMD): Observations from the Cooperative International Neuromuscular Research Group (CINRG) DMD Natural Hist...

por Thangarajh, Mathula, Spurney, Christopher F., Gordish-Dressman, Heather, Clemens, Paula R., Hoffman, Eric P., McDonald, Craig M., Henricson, Erik K., Investigators, CINRG
Publicado 2018

Antigen-driven T cell-macrophage interactions mediate the interface between innate and adaptive immunity in histidyl-tRNA synthetase-induced myositis

por Reay, Daniel P., Tabib, Tracy, Wang, Ying, Oriss, Timothy B., Young, Nicholas A., Lafyatis, Robert A., Jarjour, Wael N., Clemens, Paula R., Ascherman, Dana P.
Publicado 2023

Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study

por Clemens, Paula R., Rao, Vamshi K., Connolly, Anne M., Harper, Amy D., Mah, Jean K., McDonald, Craig M., Smith, Edward C., Zaidman, Craig M., Nakagawa, Tomoyuki, Hoffman, Eric P.
Publicado 2023

Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy

por Clemens, Paula R., Rao, Vamshi K., Connolly, Anne M., Harper, Amy D., Mah, Jean K., McDonald, Craig M., Smith, Edward C., Zaidman, Craig M., Nakagawa, Tomoyuki, Hoffman, Eric P.
Publicado 2022

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

Disease-specific and glucocorticoid-responsive serum biomarkers for Duchenne Muscular Dystrophy

por Hathout, Yetrib, Liang, Chen, Ogundele, Michael, Xu, Ganggang, Tawalbeh, Shefa M., Dang, Utkarsh J, Hoffman, Eric P., Gordish-Dressman, Heather, Conklin, Laurie S., van den Anker, John N., Clemens, Paula R., Mah, Jean K., Henricson, Erik, McDonald, Craig
Publicado 2019

GAA variants and phenotypes among 1,079 patients with Pompe disease: Data from the Pompe Registry

por Reuser, Arnold J. J., van der Ploeg, Ans T., Chien, Yin‐Hsiu, Llerena, Juan, Abbott, Mary‐Alice, Clemens, Paula R., Kimonis, Virginia E., Leslie, Nancy, Maruti, Sonia S., Sanson, Bernd‐Jan, Araujo, Roberto, Periquet, Magali, Toscano, Antonio, Kishnani, Priya S., on behalf of the Pompe Registry Sites
Publicado 2019

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

Development of novel NEMO-binding domain mimetics for inhibiting IKK/NF-κB activation

por Zhao, Jing, Zhang, Lei, Mu, Xiaodong, Doebelin, Christelle, Nguyen, William, Wallace, Callen, Reay, Daniel P., McGowan, Sara J., Corbo, Lana, Clemens, Paula R., Wilson, Gabriela Mustata, Watkins, Simon C., Solt, Laura A., Cameron, Michael D., Huard, Johnny, Niedernhofer, Laura J., Kamenecka, Theodore M., Robbins, Paul D.
Publicado 2018

Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy

por Hathout, Yetrib, Brody, Edward, Clemens, Paula R., Cripe, Linda, DeLisle, Robert Kirk, Furlong, Pat, Gordish-Dressman, Heather, Hache, Lauren, Henricson, Erik, Hoffman, Eric P., Kobayashi, Yvonne Monique, Lorts, Angela, Mah, Jean K., McDonald, Craig, Mehler, Bob, Nelson, Sally, Nikrad, Malti, Singer, Britta, Steele, Fintan, Sterling, David, Sweeney, H. Lee, Williams, Steve, Gold, Larry
Publicado 2015