Search Results - "McDonald, Craig"

Timed function tests have withstood the test of time as clinically meaningful and responsive endpoints in duchenne muscular dystrophy by McDonald, Craig M.

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

“…An unmet need remains for safe and efficacious treatments for Duchenne muscular dystrophy (DMD). To date, there are limited agents available that address the…”
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Evidence-based care in Duchenne muscular dystrophy by McDonald, Craig M, Mercuri, Eugenio

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Functional trajectories before and after loss of ambulation in Duchenne muscular dystrophy and implications for clinical trials by McDonald, Craig M, Signorovitch, James, Mercuri, Eugenio, Niks, Erik H, Wong, Brenda, Fillbrunn, Mirko, Sajeev, Gautam, Yim, Erica, Dieye, Ibrahima, Miller, Debra, Ward, Susan J, Goemans, Nathalie

“…This study examined functional trajectories of subjects during the transition phase between ambulatory and non-ambulatory Duchenne muscular dystrophy (DMD) to…”
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Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study by Bello, Luca, Gordish-Dressman, Heather, Morgenroth, Lauren P, Henricson, Erik K, Duong, Tina, Hoffman, Eric P, Cnaan, Avital, McDonald, Craig M

“…OBJECTIVE:We aimed to perform an observational study of age at loss of independent ambulation (LoA) and side-effect profiles associated with different…”
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Characterization of patients with Duchenne muscular dystrophy across previously developed health states by Muntoni, Francesco, Goemans, Nathalie, Posner, Nate, Signorovitch, James, Johnson, Michaela, He, Chujun, Dorling, Patricia, Beaverson, Katherine, Alvir, Jose, Mahn, Matthias, Ward, Susan J, McDonald, Craig M, Mercuri, Eugenio

“…Project HERCULES has developed a natural history model (NHM) of disease progression in Duchenne muscular dystrophy (DMD) that comprises eight ordered health…”
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Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and pharmacological and psychosocial management by Bushby, Katharine, MD, Finkel, Richard, MD, Birnkrant, David J, MD, Case, Laura E, DPT, Clemens, Paula R, MD, Cripe, Linda, MD, Kaul, Ajay, MD, Kinnett, Kathi, MSN, McDonald, Craig, MD, Pandya, Shree, PT, Poysky, James, PhD, Shapiro, Frederic, MD, Tomezsko, Jean, PhD, Constantin, Carolyn, PhD

“…Summary Duchenne muscular dystrophy (DMD) is a severe, progressive disease that affects 1 in 3600–6000 live male births. Although guidelines are available for…”
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THE 6-minute walk test and other endpoints in Duchenne muscular dystrophy: Longitudinal natural history observations over 48 weeks from a multicenter study by Mcdonald, Craig M., Henricson, Erik K., Abresch, R. Ted, Florence, Julaine M., Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M., Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart

“…ABSTRACT Introduction: Duchenne muscular dystrophy (DMD) subjects ≥5 years with nonsense mutations were followed for 48 weeks in a multicenter, randomized,…”
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Novel approaches to analysis of the North Star Ambulatory Assessment (NSAA) in Duchenne muscular dystrophy (DMD): Observations from a phase 2 trial by Muntoni, Francesco, Guglieri, Michela, Mah, Jean K., Wagner, Kathryn R., Brandsema, John F., Butterfield, Russell J., McDonald, Craig M., Mayhew, Anna G., Palmer, Jeffrey P., Marraffino, Shannon, Charnas, Lawrence, Mercuri, Eugenio

“…Introduction The North Star Ambulatory Assessment (NSAA) tool is a key instrument for measuring clinical outcomes in patients with Duchenne muscular dystrophy…”
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The 6-minute walk test and other clinical endpoints in duchenne muscular dystrophy: Reliability, concurrent validity, and minimal clinically important differences from a multicenter study by McDonald, Craig M., Henricson, Erik K., Abresch, R. Ted, Florence, Julaine, Eagle, Michelle, Gappmaier, Eduard, Glanzman, Allan M., Spiegel, Robert, Barth, Jay, Elfring, Gary, Reha, Allen, Peltz, Stuart W.

“…ABSTRACT Introduction: An international clinical trial enrolled 174 ambulatory males ≥5 years old with nonsense mutation Duchenne muscular dystrophy (nmDMD)…”
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A Myoelectric Control Interface for Upper-Limb Robotic Rehabilitation Following Spinal Cord Injury by McDonald, Craig G., Sullivan, Jennifer L., Dennis, Troy A., O'Malley, Marcia K.

“…Spinal cord injury (SCI) is a widespread, life-altering injury leading to impairment of sensorimotor function that, while once thought to be permanent, is now…”
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Clinical Approach to the Diagnostic Evaluation of Hereditary and Acquired Neuromuscular Diseases by McDonald, Craig M., MD

“…For diagnostic evaluation of a neuromuscular disease, the clinician must be able to obtain a relevant patient and family history and perform focused general,…”
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Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-controlled phase 3 trial by Buyse, Gunnar M, Prof, Voit, Thomas, Prof, Schara, Ulrike, Prof, Straathof, Chiara S M, MD, D'Angelo, M Grazia, MD, Bernert, Günther, MD, Cuisset, Jean-Marie, MD, Finkel, Richard S, Prof, Goemans, Nathalie, MD, McDonald, Craig M, Prof, Rummey, Christian, PhD, Meier, Thomas, PhD

“…Summary Background Cardiorespiratory failure is the leading cause of death in Duchenne muscular dystrophy. Based on preclinical and phase 2 evidence, we…”
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Survival among patients receiving eteplirsen for up to 8 years for the treatment of Duchenne muscular dystrophy and contextualization with natural history controls by Iff, Joel, Done, Nicolae, Tuttle, Edward, Zhong, Yi, Wei, Fangzhou, Darras, Basil T., McDonald, Craig M., Mercuri, Eugenio, Muntoni, Francesco

“…Introduction/Aims Eteplirsen, approved in the US for patients with Duchenne muscular dystrophy (DMD) with exon 51 skip‐amenable variants, is associated with…”
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Delays in pulmonary decline in eteplirsen‐treated patients with Duchenne muscular dystrophy by Iff, Joel, Gerrits, Charles, Zhong, Yi, Tuttle, Edward, Birk, Erica, Zheng, Yeya, Paul, Xander, Henricson, Erik K., McDonald, Craig M.

“…Introduction/Aims Pulmonary decline is a major issue in patients with Duchenne muscular dystrophy (DMD). Eteplirsen is a United States–approved treatment for…”
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Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study by McDonald, Craig M, Henricson, Erik K, Abresch, Richard T, Duong, Tina, Joyce, Nanette C, Hu, Fengming, Clemens, Paula R, Hoffman, Eric P, Cnaan, Avital, Gordish-Dressman, Heather, Vishwanathan, Vijay, Chidambaranathan, S, Biggar, W. Douglas, McAdam, Laura C., Mah, Jean K., Tulinius, Mar, Cnaan, Avital, Morgenroth, Lauren P., Leshner, Robert, Tesi-Rocha, Carolina, Thangarajh, Mathula, Duong, Tina, Kornberg, Andrew, Ryan, Monique, Nevo, Yoram, Dubrovsky, Alberto, Clemens, Paula R., Abdel-Hamid, Hoda, Connolly, Anne M., Pestronk, Alan, Teasley, Jean, Bertorin, Tulio E., Webster, Richard, Kolski, Hanna, Kuntz, Nancy, Driscoll, Sherilyn, Bodensteiner, John B., Carlo, Jose, Gorni, Ksenija, Lotze, Timothy, Day, John W., Karachunski, Peter, Henricson, Erik K., Abresch, Richard T., Joyce, Nanette C., McDonald, Craig M.

“…Glucocorticoid treatment is recommended as a standard of care in Duchenne muscular dystrophy; however, few studies have assessed the long-term benefits of this…”
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Genetic modifiers of ambulation in the cooperative international Neuromuscular research group Duchenne natural history study by 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.

“…Objective We studied the effects of LTBP4 and SPP1 polymorphisms on age at loss of ambulation (LoA) in a multiethnic Duchenne muscular dystrophy (DMD) cohort…”
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Efficacy and safety of vamorolone in Duchenne muscular dystrophy: An 18-month interim analysis of a non-randomized open-label extension study by 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.

“…The differential mechanism of action of vamorolone compared to traditional corticosteroid anti-inflammatory drugs is attributed to the loss of gene…”
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Suitability of external controls for drug evaluation in Duchenne muscular dystrophy by Goemans, Nathalie, Signorovitch, James, Sajeev, Gautam, Yao, Zhiwen, Gordish-Dressman, Heather, McDonald, Craig M., Vandenborne, Krista, Miller, Debra, Ward, Susan J., Mercuri, Eugenio

“…OBJECTIVETo evaluate the suitability of real-world data (RWD) and natural history data (NHD) for use as external controls in drug evaluations for ambulatory…”
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Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy by 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

“…Significance Duchenne muscular dystrophy (DMD) is a rare and devastating muscle disease caused by mutations in the X-linked DMD gene (which encodes the…”
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