CraigMichelMcDonaldMD

PubMed

• Gait Event Detection and Travel Distance Using Waist-Worn Accelerometers across a Range of Speeds: Automated Approach.
  Ramli, A., Liu, X., Berndt, K., Chuah, C., Goude, E., Kaethler, L., Lopez, A., Nicorici, A., Owens, C., Rodriguez, D., Aranki, D., Henricson, E., Wang, J., McDonald, C.> ;Sensors. 2024 Feb 9
• 2 citations
  Gait Characterization in Duchenne Muscular Dystrophy (DMD) Using a Single-Sensor Accelerometer: Classical Machine Learning and Deep Learning Approaches.
  Ramli, A., Liu, X., Berndt, K., Goude, E., Hou, J., Kaethler, L., Liu, R., Lopez, A., Nicorici, A., Owens, C., Rodriguez, D., Zhang, H., Aranki, D., Henricson, E., Wan...> ;Sensors. 2024 Feb 8
• 1 citations
  Practical Considerations for Delandistrogene Moxeparvovec Gene Therapy in Patients With Duchenne Muscular Dystrophy.
  Mendell, J., Proud, C., Mason, S., Darton, E., Wang, S., Wandel, C., Murphy, A., Mercuri, E., Muntoni, F., Zaidman, C., McDonald, C.> ;Pediatric Neurology. 2024 Apr 1
• 269th ENMC international workshop: 10 years of clinical trials in Duchenne muscular dystrophy - What have we learned? 9-11 December 2022, Hoofddorp, The Netherlands.
  Karin J Naarding, Georgia Stimpson, Susan J Ward, Nathalie Goemans, Craig McDonald, Eugenio Mercuri, Francesco Muntoni> ;Neuromuscular Disorders. 2023 Nov 1
• Sleep Disordered Breathing in Children with Neuromuscular Disease.
  Ambika G Chidambaram, Sanjay Jhawar, Craig M McDonald, Kiran Nandalike> ;Children. 2023 Oct 11
• 5 citations
  Efficacy and Safety of Viltolarsen in Boys With Duchenne Muscular Dystrophy: Results From the Phase 2, Open-Label, 4-Year Extension Study.
  Paula R Clemens, Vamshi K Rao, Anne M Connolly, Amy D Harper, Jean K Mah, Craig M McDonald, Edward C Smith, Craig M Zaidman, Tomoyuki Nakagawa, , Eric P Hoffman> ;Journal of Neuromuscular Diseases. 2023 Jan 1
• 2 citations
  Disease progression rates in ambulatory Duchenne muscular dystrophy by steroid type, patient age and functional status.
  Craig M McDonald, Jessica R Marden, Perry B Shieh, Brenda L Wong, Henry Lane, Adina Zhang, Ha Nguyen, Molly Frean, Panayiota Trifillis, Karyn Koladicz, James Signorovitch> ;Journal of Comparative Effectiveness Research. 2023 Apr 1
• 6 citations
  Genotypes and Motor Function in Duchenne Muscular Dystrophy: A Multi-institution Meta-analysis With Implications for Clinical Trials.
  Francesco Muntoni, James Signorovitch, Gautam Sajeev, Henry Lane, Madeline Jenkins, Ibrahima Dieye, Susan J Ward, Craig McDonald, Nathalie Goemans, Erik H Niks, Brenda...> ;Neurology. 2023 Apr 11
• 3 citations
  Genetic modifiers of upper limb function in Duchenne muscular dystrophy.
  Daniele Sabbatini, Aurora Fusto, Sara Vianello, Matteo Villa, Joanna Janik, Grazia D'Angelo, Eleonora Diella, Francesca Magri, Giacomo P Comi, Chiara Panicucci, Claudi...> ;Journal of Neurology. 2022 Sep 1
• 7 citations
  Quantitative magnetic resonance imaging measures as biomarkers of disease progression in boys with Duchenne muscular dystrophy: a phase 2 trial of domagrozumab.
  Sarah P Sherlock, Jeffrey Palmer, Kathryn R Wagner, Hoda Z Abdel-Hamid, Enrico Bertini, Cuixia Tian, Jean K Mah, Anna Kostera-Pruszczyk, Francesco Muntoni, Michela Gug...> ;Journal of Neurology. 2022 Aug 1
• 3 citations
  Longitudinal changes in cardiac function in Duchenne muscular dystrophy population as measured by magnetic resonance imaging.
  Abhinandan Batra, Alison M Barnard, Donovan J Lott, Rebecca J Willcocks, Sean C Forbes, Saptarshi Chakraborty, Michael J Daniels, Jannik Arbogast, William Triplett, Er...> ;BMC Cardiovascular Disorders. 2022 Jun 9
• 18 citations
  Long-Term Functional Efficacy and Safety of Viltolarsen in Patients with Duchenne Muscular Dystrophy.
  Paula R Clemens, Vamshi K Rao, Anne M Connolly, Amy D Harper, Jean K Mah, Craig M McDonald, Edward C Smith, Craig M Zaidman, Tomoyuki Nakagawa, , Eric P Hoffman> ;Journal of Neuromuscular Diseases. 2022 Jan 1
• 39 citations
  Effect of Different Corticosteroid Dosing Regimens on Clinical Outcomes in Boys With Duchenne Muscular Dystrophy: A Randomized Clinical Trial.
  Michela Guglieri, Kate Bushby, Michael P McDermott, Kimberly A Hart, Rabi Tawil, William B Martens, Barbara E Herr, Elaine McColl, Chris Speed, Jennifer Wilkinson, Jan...> ;JAMA. 2022 Apr 19
• 11 citations
  Real-world and natural history data for drug evaluation in Duchenne muscular dystrophy: suitability of the North Star Ambulatory Assessment for comparisons with extern...
  Francesco Muntoni, James Signorovitch, Gautam Sajeev, Nathalie Goemans, Brenda Wong, Cuixia Tian, Eugenio Mercuri, Nicolae Done, Hallee Wong, Jackson Moss, Zhiwen Yao,...> ;Neuromuscular Disorders. 2022 Apr 1
• 25 citations
  Repeated intravenous cardiosphere-derived cell therapy in late-stage Duchenne muscular dystrophy (HOPE-2): a multicentre, randomised, double-blind, placebo-controlled,...
  Craig M McDonald, Eduardo Marbán, Suzanne Hendrix, Nathaniel Hogan, Rachel Ruckdeschel Smith, Michelle Eagle, Richard S Finkel, Cuixia Tian, Joanne Janas, Matthew M Ha...> ;Lancet. 2022 Mar 12
• 3 citations
  Influence of βadrenergic receptor genotype on longitudinal measures of forced vital capacity in patients with Duchenne muscular dystrophy.
  Eli F Kelley, Troy J Cross, Craig M McDonald, Eric P Hoffman, Luca Bello> ;Neuromuscular Disorders. 2022 Feb 1
• 23 citations
  Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A 30-Month Nonrandomized Controlled Open-Label Extension Trial.
  Jean K Mah, Paula R Clemens, Michela Guglieri, Edward C Smith, Richard S Finkel, Mar Tulinius, Yoram Nevo, Monique M Ryan, Richard Webster, Diana Castro, Nancy L Kuntz...> ;JAMA Network Open. 2022 Jan 4
• 1 citations
  A Longitudinal Study of Quantitative Muscle Strength and Functional Motor Ability in Ambulatory Boys with Duchenne Muscular Dystrophy.
  Cathleen E Buckon, Susan E Sienko, Eileen G Fowler, Anita M Bagley, Loretta A Staudt, Mitell Sison-Williamson, Kent R Heberer, Craig M McDonald, Michael D Sussman> ;Journal of Neuromuscular Diseases. 2022 Jan 1
• 7 citations
  Comparing Deflazacort and Prednisone in Duchenne Muscular Dystrophy.
  W Douglas Biggar, Andrew Skalsky, Craig M McDonald> ;Journal of Neuromuscular Diseases. 2022 Jan 1
• 25 citations
  Ataluren delays loss of ambulation and respiratory decline in nonsense mutation Duchenne muscular dystrophy patients
  Craig M. McDonald, Francesco Muntoni, Vinay Penematsa, Joel Jiang, Allan Kristensen, Francesco Bibbiani, Elizabeth Goodwin, Heather Gordish-Dressman, Lauren P. Morgenr...> ;Journal of Comparative Effectiveness Research. 2021 Nov 18
• 3 citations
  Knee Strength and Ankle Range of Motion Impacts on Timed Function Tests in Duchenne Muscular Dystrophy: In the Era of Glucocorticoids.
  Tina Duong, Jennifer Canbek, Alicia Fernandez-Fernandez, Erik K Henricson, Marisa Birkmeier, Catherine Siener, Carolina Tesi Rocha, Craig M. McDonald, Heather Gordish-...> ;Journal of Neuromuscular Diseases. 2021 Oct 29
• 5 citations
  Meta-analyses of deflazacort versus prednisone/prednisolone in patients with nonsense mutation Duchenne muscular dystrophy.
  Perry B. Shieh, Gary Elfring, Panayiota Trifillis, Claudio Santos, Stuart W. Peltz, Julie A. Parsons, Susan D. Apkon, Basil T. Darras, Craig Campbell, Craig M. McDonal...> ;Journal of Comparative Effectiveness Research. 2021 Oct 25
• 1 citations
  Longitudinal changes in energy cost during walking in boys with Duchenne Muscular Dystrophy (DMD).
  Susan Sienko, Cathleen E. Buckon, Anita Bagley, Loretta A. Staudt, Eileen Fowler, Kent Heberer, Mitell Sison-Williamson, Craig M. McDonald, Michael D. Sussman> ;Gait & Posture. 2021 Oct 1
• 5 citations
  Evaluating longitudinal therapy effects via the North Star Ambulatory Assessment.
  Craig M. McDonald, Lee-Jen Wei, Kevin M. Flanigan, Gary Elfring, Panayiota Trifillis, Francesco Muntoni> ;Muscle & Nerve. 2021 Sep 2
• 27 citations
  First Regulatory Qualification of a Novel Digital Endpoint in Duchenne Muscular Dystrophy: A Multi-Stakeholder Perspective on the Impact for Patients and for Drug Deve...
  Laurent Servais, Eric Camino, Aude Clement, Craig M. McDonald, Jacek Lukawy, Linda Lowes, Damien Eggenspieler, Francesca Cerreta, Paul Strijbos> ;Digital Biomarkers. 2021 Aug 5
• Join now to see all

Journal Articles

• Development of a patient-reported outcome measure for upper limb function in Duchenne muscular dystrophy: DMD Upper Limb PROM  
  Klingels K, Mayhew AG, Mazzone ES, Duong T, Decostre V, Werlauff U, Vroom E, Mercuri E, Goemans NM; Upper Limb Clinical Outcome Group, Med Child Neurol, 1/26/2016
• Hip kinetics during gait are clinically meaningful outcomes in young boys with Duchenne muscular dystrophy  
  Heberer K, Fowler E, Staudt L, Sienko S, Buckon CE, Bagley A, Sison-Williamson M, McDonald CM, Sussman MD, Gait Posture, 1/24/2016
• Baseline results of the NeuroNEXT spinal muscular atrophy infant biomarker study  
  Kolb SJ, Coffey CS, Yankey JW, Krosschell K, Arnold WD, Rutkove SB, Swoboda KJ, Reyna SP, Sakonju A, Darras BT, Shell R, Kuntz N, Castro D, Iannaccone ST, Parsons J, C..., Ann Clin Transl Neurol, 1/21/2016
• Prednisone and Deflazacort in Duchenne Muscular Dystrophy: Do They Play a Different Role in Child Behavior and Perceived Quality of Life?  
  Sienko S, Buckon C, Fowler E, Bagley A, Staudt L, Sison-Williamson M, Zebracki K, McDonald CM, Sussman M, PLOS Currents Muscular Dystrophy, 1/17/2016
• Discovery of Metabolic Biomarkers for Duchenne Muscular Dystrophy within a Natural History Study  
  Boca SM, Nishida M, Harris M, Rao S, Cheema AK, Gill K, Seol H, Morgenroth LP, Henricson E, McDonald C, Mah JK, Clemens PR, Hoffman EP, Hathout Y, Madhavan S, PLoS One, 1/15/2016
• Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy  
  McDonald CM, Meier T, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, Rummey C, Leinonen M, Spagnolo P; and Buyse GM for the..., Neuromuscul Disord, 1/12/2016
• Association Study of Exon Variants in the NF-kB and TGFBeta Pathways Identifies CD40 as a Modifier of Duchenne Muscular Dystrophy  
  Bello L, Flanigan KM, Weiss RB; United Dystrophinopathy Project., Spitali P, Aartsma-Rus A, Muntoni F, Zaharieva I, Ferlini A, Mercuri E, Tuffery-Giraud S, Claustres M..., Am J Hum Genet, 1/12/2016
• Can Quantitative Muscle Strength and Functional Motor Ability Differentiate the Influence of Age and Corticosteroids in Ambulatory Boys with Duchenne Muscular Dystrophy?  
  Buckon C, Sienko S, Bagley A, Sison-Williamson M, Fowler E, Staudt L, Heberer K, McDonald CM, Sussman M, PLoS Curr, 1/8/2016
• Clinical trial Readiness in Non-Ambulatory Boys and Men with DMD: MDA-DMD Network Follow-up  
  Connolly A, Florence J, Zaidman C, Golumbek P, Mendell J, Flanigan K, Day J, McDonald C, Darras B, Kang P, Siener C, Gadeken R, Anand P, Schierbecker J, Malkus E, Lowe..., Muscle Nerve, 1/1/2016
• DMD genotypes and ambulation in the CINRG Duchenne Natural History Study: implications for clinical trials  
  Bello L, Morgenroth LP, Gordish-Dressman H, Hoffman EP, McDonald CM, Cirak S; on behalf of the CINRG investigators, Neurology, 1/1/2016
• Serum pharmacodynamics biomarkers for chronic corticosteroid treatment of children  
  Hathout Y, Conklin L, Seol H, Gordish-Dressman H, Brown KJ, Morgenroth LP1, Nagaraju K, Heier CR, Jesse M. Damsker JM, van den Anker JN, Henricson E, Clemens PR, Mah J..., Nature Scientific Reports, 1/1/2016
• How a patient advocacy group developed the first proposed draft guidance document for industry for submission to the US Food and Drug Administration  
  Furlong P, Bridges JF, Charnas L, Fallon JR, Fischer R, Flanigan KM, Franson TR, Gulati N, McDonald C, Peay H, Sweeney HL, Orphanet J Rare Dis, 1/24/2015
• Prednisone/prednisolone and deflazacort regimens in the CINRG Duchenne Natural History Study  
  Bello L, Gordish-Dressman H, Morgenroth LP, Henricson EK, Duong T, Hoffman EP, Cnaan A, McDonald CM; CINRG Investigators, Neurology, 1/22/2015
• ICU-Acquired Weakness Is Associated With Differences in Clinical Outcomes in Critically Ill Children  
  Field-Ridley A, Dharmar M, Steinhorn D, McDonald C, Marcin JP, Pediatr Crit Care Med, 1/21/2015
• Large-scale serum protein biomarker discovery in Duchenne muscular dystrophy  
  Hathout Y, Brody E, Clemens PR, Cripe L, DeLisle RK, Furlong P, Gordish-Dressman H, Hache L, Henricson E, Hoffman EP, Kobayashi YM, Lorts A, Mah JK, McDonald C, Mehler..., Proc Natl Acad Sci U S A, 1/9/2015
• Reachable Workspace and Performance of Upper Limb (PUL) in Duchenne muscular dystrophy  
  Han JJ, de Bie E, Nicorici A, Abresch RT, Anthonisen C, Bajcsy R, Kurillo G, McDonald CM, Muscle Nerve, 1/5/2015
• Efficacy of idebenone on respiratory function in patients with Duchenne muscular dystrophy not using glucocorticoids (DELOS): a double-blind randomised placebo-control...  
  Buyse GM, Voit T, Schara U, Straathof CS, D'Angelo MG, Bernert G, Cuisset JM, Finkel RS, Goemans N, McDonald CM, Rummey C, Meier T; DELOS Study Group, Lancet, 1/2/2015
• Evaluation of Phrenic Nerve and Diaphragm Function with Peripheral Nerve Stimulation and M-Mode Ultrasonography in Potential Pediatric Phrenic Nerve or Diaphragm Pacin...  
  Skalsky AJ, Lesser DJ, McDonald CM, Phys Med Rehabil Clin N Am, 1/1/2015
• Clinical phenotypes as predictors of the outcome of skipping around DMD exon 45  
  Findlay AR, Wein N, Kaminoh Y, Taylor LE, Dunn DM, Mendell JR, King WM, Pestronk A, Florence JM, Mathews KD, Finkel RS, Swoboda KJ, Howard MT, Day JW, McDonald C, Nico..., Ann Neurol, 1/1/2015
• Genetic modifiers of ambulation in the Cooperative International Neuromuscular Research Group Duchenne Natural History Study  
  Bello L, Kesari A, Gordish-Dressman H, Cnaan A, Morgenroth LP, Punetha J, Duong T, Henricson EK, Pegoraro E, McDonald CM, Hoffman EP, on behalf of the CINRG Investigators, Ann Neurol, 1/1/2015
• Feasibility and Reproducibility of Echocardiographic Measures in Children with Muscular Dystrophies  
  Spurney CF, McCaffrey FM, Cnaan A, Morgenroth LP, Ghelani SJ, Gordish-Dressman H, Arrieta A, Connolly AM, Lotze TE, McDonald CM, Leshner RT, Clemens PR, J Am Soc Echocardiogr, 1/1/2015
• Idebenone as a Novel Therapeutic Approach for Duchenne Muscular Dystrophy  
  Buyse GM, Gueven N, McDonald CM, European Neurological Review, 1/1/2015
• Outcome reliability in non ambulatory boys/men with duchenne muscular dystrophy  
  Connolly AM, Malkus EC, Mendell JR, Flanigan KM, Miller JP, Schierbecker JR, Siener CA, Golumbek PT, Zaidman CM, McDonald CM, Johnson L, Nicorici A, Karachunski PI, Da..., Muscle Nerve, 1/24/2014
• Measuring clinical effectiveness of medicinal products for the treatment of Duchenne muscular dystrophy  
  Lynn S, Aartsma-Rus A, Bushby K, Furlong P, Goemans N, De Luca A, Mayhew A, McDonald C, Mercuri E, Muntoni F, Pohlschmidt M, Verschuuren J, Voit T, Vroom E, Wells DJ, ..., Neuromuscul Disord, 1/11/2014
• One year outcome of boys with Duchenne muscular dystrophy using the Bayley-III scales of infant and toddler development  
  Connolly AM, Florence JM, Cradock MM, Eagle M, Flanigan KM, McDonald CM, Karachunski PI, Darras BT, Bushby K, Malkus EC, Golumbek PT, Zaidman CM, Miller JP, Mendell JR..., Pediatr Neurol, 1/1/2014
• Join now to see all

Books/Book Chapters

• Pediatric Rehabilitation: Principles and Practice 
  Chapter: Electrodiagnostics in Pediatrics. 
  McDonald CMEditors: Alexander MA, and Mathews DJ, (Eds.), Murphy KP, Associate Editor.Demos Medical Publishing, New York, NYEdition 5th,2015
• Pediatric Rehabilitation: Principles and Practice 
  Chapter: Neuromuscular Diseases. 
  McDonald CMEditors: Alexander MA, and Mathews DJ, (Eds.), Murphy KP, Associate Editor.Demos Medical Publishing, New York, NYEdition 5th,2015
• Physical Medicine and Rehabilitation 
  Chapter: Myopathic Disorders. 
  McDonald CM, Han JJ, Carter GTEditors: Braddom R.Elsevier, Philadelphia2011
• Pediatric Rehabilitation Principles and Practice 
  Chapter: Electrodiagnosis in Pediatrics 
  Page 127 - 163McDonald CMEditors: Michael A. Alexander, Dennis J. Matthews.Demos Medical, New YorkEdition 4th,2009
• Pediatric Rehabilitation Principles and Practice 
  Chapter: Neuromuscular Diseases 
  Page 277 - 333McDonald CMEditors: Michael A. Alexander, Dennis J. Matthews.Demos Medical, New YorkEdition 4th,2009
• Physical Medicine and Rehabilitation 
  Chapter: Myopathic Disorders 
  Page 1099 - 1131McDonald CM, Han JJ, Abresch RT, Carter GTEditors: Braddom RL.Elsevier, Inc., PhiladelphiaEdition 3rd,2007
• Duchenne Muscular Dystrophy: Advances in Therapeutics 
  Chapter: Rehabilitation management of Duchenne muscular dystrophy. 
  Page 149 - 175McDonald CM, Carter, GT, Han JJ, Benditt JOEditors: Chamberlain JC, Rando TA.Taylor & Francis Group Publishing Co, New York2006
• Caring for the Child with Spina Bifida 
  Chapter: Mobility Issues and Prescribing Mobility Aids for Children with Myelomeningocele 
  Page 341 - 358McDonald CMEditors: Sarwark JF, Lubicky JP.American Association of Orthopaedic Surgeons, Rosemont, IL2001
• Pediatric Rehabilitation 
  Chapter: Electrodiagnosis in Pediatrics 
  Page 81 - 109McDonald CMEditors: Molnar GE, Alexander MA.Hanley and Belfus, PhiladelphiaEdition 3rd,1999
• Pediatric Rehabilitation 
  Chapter: Neuromuscular Diseases 
  Page 289 - 330McDonald CMEditors: Molnar GE, Alexander MA.Hanley and Belfus, PhiladelphiaEdition 3rd,1999
• Sports and Exercise for Children with Chronic Health Conditions 
  Chapter: Childhood Progressive Neuromuscular Disease 
  Page 109 - 121Kilmer DD, McDonald CMEditors: Barry Goldberg.Human Kinetics, Champaign, IL1995
• Myelodysplasias and Exstrophies 
  Page 313 - 356Shurtleff DB, Dudgeon B, McDonald CM, CarrSouza J: Mobility, Shurtleff DBGrune and Stratton, Orlando, FL1986
• Join now to see all

Abstracts/Posters

• Meta-analyses of ataluren in patients with nonsense mutation Duchenne muscular dystrophy.
  Campbell C, Muntoni C, Mercuri E, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel G, Peltz SW, McDonald C, 45th CNS Annual Meeting, Vancouver, BC, Canada, 1/26/2016
• Results of North Star Ambulatory Assessments in the Phase 3 Ataluren Confirmatory Trial in Patients with Nonsense Mutation Duchenne Muscular Dystrophy (ACT DMD).
  Muntoni F, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz S, McDonald C, 45th CNS Annual Meeting, Vancouver, BC, Canada, 1/26/2016
• Effect of Tadalafil, a Phosphodiesterase Type 5 Inhibitor, on Ambulatory Decline in Boys with Duchenne Muscular Dystrophy: Top-Line Results of a Phase III, Randomized,...
  Victor RG, Finkel R, Sweeney HL, McDonald CM, Byrne B, Vandenborne K, Miceli C, Eagle M, Goemans N, Furlong P, Landry J, Elashoff R, and Cox D, 2016 MDA Clinic Directors Meeting, Denver, CO, 1/20/2016
• Effect of Tadalafil, a Phosphodiesterase Type 5 Inhibitor, on Ambulatory Decline in Boys with Duchenne Muscular Dystrophy: Top-Line Results of a Phase III Randomized C...
  Victor RG, Finkel R, Sweeney HL, McDonald CM, Byrne B, Vandenborne K, Miceli C, Eagle M, Goemans N, Furlong P, Landry J, Elashoff R, and Cox D, 2016 MDA Clinic Directors Meeting, Denver, CO, 1/20/2016
• Ataluren: An overview of clinical trial results in nonsense mutation Duchenne muscular dystrophy.
  McDonald CM, Bushby K, Tulinius M, Finkel R, Topaloglu H, Day J, Flanigan K, Lowes L, Eagle M, Luo X, Elfring G, Kroger H, Ong T, Spiegel R, Peltz SW, Kirschner J, for..., 2016 MDA Clinic Directors Meeting, Denver, CO, 1/20/2016
• Effect of ataluren on timed function tests in nonsense mutation Duchenne muscular dystrophy.
  Goemans N, Campbell C, McDonald CM, Voit T, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz SW, Bushby K, for the Ataluren DMD Study Steering Committee..., 2016 MDA Clinic Directors Meeting, Denver, CO, 1/20/2016
• Use of the six-minute walk distance (6MWD) across Duchenne muscular dystrophy (DMD) studies.
  McDonald CM, Sweeney HL, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz SW, and Mercuri E for the Ataluren DMD Study Steering Committee, 2016 MDA Clinic Directors Meeting, Denver, CO, 1/20/2016
• Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy.
  Buyse GM, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset J-M, Finkel R, Goemans N, Rummey C, Leinonen M, Mayer OH, Spagnolo P, Meier T, McDonald CM, ..., 2016 MDA Clinic Directors Meeting, Denver, CO, 1/20/2016
• Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy.
  McDonald CM, Meier T, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset J-M, Finkel R, Goemans N, Rummey C, Leinonen M, Spagnolo P, Buyse GM, on behalf ..., 2016 MDA Clinic Directors Meeting, Denver, CO, 1/20/2016
• Use of the Six-Minute Walk Distance (6MWD) Across Duchenne Muscular Dystrophy (DMD) Studies.
  McDonald C, Sweeney HL, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz SW, and Mercuri E for the Ataluren DMD Study Steering Committee, 68th American Academy of Neurology Annual Meeting, Vancouver, BC, Canada, 1/15/2016
• Ataluren: An Overview of Clinical Trial Results in Nonsense Mutation Duchenne Muscular Dystrophy.
  McDonald C, Bushby K, Tulinius M, Finkel R, Topaloglu H, Day J, Flanigan K, Lowes L, Eagle M, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz S, Kirsch..., 68th American Academy of Neurology Annual Meeting, Vancouver, BC, Canada, 1/15/2016
• ACTDMD: Effect of Ataluren on Timed Function Tests (TFTs) in Nonsense Mutation Duchenne Muscular Dystrophy.
  Goemans N, Campbell C, McDonald C, Voit T, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz S, Bushby K, 68th American Academy of Neurology Annual Meeting, Vancouver, BC, Canada, 1/15/2016
• Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy.
  Buyse GM, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset J-M, Finkel R, Goemans N, Rummey C, Leinonen M, Mayer OH, Spagnolo P, Meier T, McDonald CM, ..., 5th International Congress of Myology, Lyon, France, 1/14/2016
• Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy.
  McDonald CM, Meier T, Voit T, Schara U, Straathof CSM, D'Angelo MG, Bernert G, Cuisset J-M, Finkel R, Goemans N, Rummey C, Leinonen M, Spagnolo P, Buyse GM, on behalf ..., 5th International Congress of Myology, Lyon, France, 1/14/2016
• Use of the Six-Minute Walk Distance (6MWD) Across Duchenne Muscular Dystrophy (DMD) Studies.
  McDonald C, Sweeney HL, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz SW, and Mercuri E for the Ataluren DMD Study Steering Committee, 5th International Congress of Myology, Lyon, France, 1/14/2016
• Ataluren: An overview of clinical trial results in nonsense mutation Duchenne muscular dystrophy.
  McDonald CM, Bushby K, Tulinius M, Finkel R, Topaloglu H, Day J, Flanigan K, Lowes L, Eagle M, Luo X, Elfring G, Kroger H, Ong T, Spiegel R, Peltz SW, Kirschner J, for..., 5th International Congress of Myology, Lyon, France, 1/14/2016
• Effect of ataluren on timed function tests in nonsense mutation Duchenne muscular dystrophy.
  Goemans N, Campbell C, McDonald CM, Voit T, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz SW, Bushby K, for the Ataluren DMD Study Steering Committee..., 5th International Congress of Myology, Lyon, France, 1/14/2016
• Comparing clinical phenotype of patients with Duchenne muscular dystrophy with deletions amenable to skipping exons 44, 45, and 53.
  Wang S, Wong B, McDonald C, Yang Y, Goemans N, 21st International Annual Congress of the World Muscle Society, Granada, Spain, 1/4/2016
• DMD genotypes and loss of ambulation in the CINRG Duchenne natural history study.
  Bello L, Morgenroth L, Gordish-Dressman H, Hoffman E, McDonald C, Cirak S, 21st International Annual Congress of the World Muscle Society, Granada, Spain, 1/4/2016
• Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy.
  McDonald C, Meier T, Voit T, Schara U, Straathof C, D'Angelo M, Bernert G, Cuisset J, Finkel R, Goemans N, Rummey C, Leinonen M, Spagnolo P, Buyse G on behalf of DELOS..., 21st International Annual Congress of the World Muscle Society, Granada, Spain, 1/4/2016
• Treatment effect of idebenone on inspiratory function in patients with Duchenne muscular dystrophy.
  Buyse G, Voit T, Schara U, Straathof C, D'Angelo M, Bernert G, Cuisset J, Finkel R., Goemans N, Rummey C, Leinonen M, Mayer O, Spagnolo P, Meier T, McDonald C, on beha..., 21st International Annual Congress of the World Muscle Society, Granada, Spain, 1/4/2016
• Results of North Star ambulatory assessments in the phase 3 ataluren confirmatory trial in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD).
  Muntoni F, Luo X, Elfring G, Kroger H, Riebling P, Ong T, Spiegel R, Peltz S, McDonald C, 21st International Annual Congress of the World Muscle Society, Granada, Spain, 1/4/2016
• Comparing clinical phenotype of patients with Duchenne muscular dystrophy with deletions amenable to skipping exons 44, 45, and 53.
  Wang S, Wong B, McDonald C, Yang Y, Goemans N, Neuromuscular Disorders, 1/1/2016
• DMD genotypes and loss of ambulation in the CINRG Duchenne natural history study.
  Bello L, Morgenroth L, Gordish-Dressman H, Hoffman E, McDonald C, Cirak S, Neuromuscular Disorders, 1/1/2016
• Idebenone reduces respiratory complications in patients with Duchenne muscular dystrophy.
  McDonald C, Meier T, Voit T, Schara U, Straathof C, D'Angelo M, Bernert G, Cuisset J, Finkel R, Goemans N, Rummey C, Leinonen M, Spagnolo P, Buyse G on behalf of DELOS..., Neuromuscular Disorders, 1/1/2016
• Join now to see all

Lectures

• 6-Minute Walk Test (6MWT) in Duchenne Muscular Dystrophy. 
  Taormina, Italy
• The Impact of Steroids for Young Children with Duchenne muscular dystrophy. 
  Orlando, FL - 1/28/2016
• Epicatechin for Treatment of Dystrophinopathy. 
  Orlando, FL - 1/27/2016
• Reachable Workspace in DMD using Kinect. Presented as part of the session on Novel Upper Extermity Outcomes Measures in Duchenne muscular dystrophy. 
  Orlando, FL - 1/26/2016
• Challenges in Duchenne Muscular Dystrophy Clinical Trial Design and the Impact of Deflazacort on the Natural History of the Disease. 
  Washington, DC - 1/21/2016
• Translarna (ataluren) for the treatment of nonsense mutation DMD in ambulatory patients 5 years and older EMEA/H/C/002720. 
  London, United Kingdom - 1/21/2016
• Development of Novel Upper Extremity Outcome Measure: Reachable Workspace using 3D sensor Technology. 
  San Rafael, CA - 1/19/2016
• Pulmonary Function Testing in Duchenne Muscular Dystrophy. 
  San Rafael, CA - 1/19/2016
• "Developing a State-of-the-Art and Sustainable Clinical Trials Infrastructure in an Academic PM&R Department: Meeting the Research, Education and Clinical Missions." 
  Sacramento, CA - 1/18/2016
• Duchenne Muscular Dystrophy: Unmet Medical Need, Natural History and Clinical Trial Challenges: Implications for Ataluren Drug Development. 
  South Plainfield, NJ - 1/18/2016
• Epicatechin enhances mitochondrial biogenesis, increases dystrophin and utrophin, increases follistatin while decreasing myostatin, and improves skeletal muscle exerci... 
  Sausalito, CA - 1/17/2016
• Meeting the Strategic Initiatives of a Health System: Interface of PM&R Clinical trials with Research Projects focusing on Physiatric Therapeutics. 
  1/15/2016
• Clinical Trial Endpoints in DMD: A Disease Milestone Perspective. 
  Washington, DC - 1/14/2016
• Pulmonary Endpoints in DMD: the CINRG Duchenne Natural History Study data. 
  Washington, DC - 1/14/2016
• Reliability of Pulmonary Endpoints in Duchenne muscular dystrophy. 
  Washington, DC - 1/14/2016
• Pulmonary data in the context of a DMD disease progression model: Case example of eteplirsen for exon 51 skipping amenable DMD & effect on pulmonary function. 
  Washington, DC - 1/14/2016
• Historical Imperatives and Lessons Learned from Human Clinical Trials in Duchenne muscular dystrophy. 
  Dallas, TX - 1/9/2016
• Pulmonary Endpoints in Duchenne muscular dystrophy, and Clinical Meaningfulness of the DELOS Phase 3 Trial Results. 
  Washington, DC - 1/30/2015
• Natural history studies and development of clinical endpoints in Duchenne muscular dystrophy to guide therapeutic development. 
  Honolulu, HI - 1/29/2015
• The Cooperative International Neuromuscular Research Group (CINRG) University of California, Davis/CINRG Duchenne Natural History Study (DNHS) Bridge Funding and Acade... 
  Washington, DC - 1/26/2015
• Duchenne Muscular Dystrophy Natural History: Unmet Medical Need. 
  Washington, DC - 1/24/2015
• Risk-Benefit of drisapersen in Duchenne muscular dystrophy. 
  Washington, DC - 1/24/2015
• An Overview of Duchenne Muscular Dystrophy: Natural History and Clinical End Points. 
  New York, NY - 1/22/2015
• Use of StepWatchTM stride activity patterns as an outcome measure in Duchenne muscular dystrophy. 
  Washington, DC - 1/21/2015
• Different Together: Explaining Phenotype Variability in Duchenne and its Implications in Clinical Trials 
  Washington, DC - 1/20/2015
• Join now to see all

Other

• FDA Advisory Committee on Eteplirsen as an exon skipping treatment for for exon 51 skipping amenable patients with in Duchenne muscular dystrophy. 
  McDonald CM, Expert Testimony for Sarepta Therapeutics
  Washington, DC - 1/26/2016
• Translarna (ataluren) for the treatment of nonsense mutation DMD. 
  McDonald CM, Parent Project Muscular Dystrophy (PPMD) National Webinar
  1/1/2016
• Understanding clinical endpoints in Duchenne Muscular Dystrophy. 
  McDonald CM, PTC webinar "ACT DMD Clinical Trial Results."
  1/15/2015
• Review results from Study DMD114673: Open Label Extension of Drisapersen in Duchenne muscular dystrophy-177 Weeks. 
  McDonald CM, United Parent Project Muscular Dystrophy (UPPMD) Webinar for the Patient Community
  Leiden, The Netherlands - 1/25/2014
• 48-week results of US phase II study (DEMAND V) of Drisapersen in Duchenne muscular dystrophy. 
  McDonald CM, United Parent Project Muscular Dystrophy (UPPMD) Webinar for the Patient Community
  Leiden, The Netherlands - 1/25/2014
• User manual for the Quality of Life in Neurological Disorders (Neuro-QOL) measures, version 2.0 
  National Institute of Neurological Disorders and Stroke (NINDS), Cella, D. and the Neuro-QOL Investigators
  1/1/2014
• Natural history in DMD. What is Clinically Meaningful? 
  McDonald CM, Parent Project Muscular Dystrophy (PPMD) Webinar
  1/1/2013
• Slowly progressive myopathies in Adults. 
  McDonald CM, Comprehensive Review in Physical Medicine and Rehabilitation Video Series
  1/1/2011
• Childhood-onset neuromuscular diseases. 
  McDonald CM, Comprehensive Review in Physical Medicine and Rehabilitation Video Series
  1/1/2011
• Neuro-QOL technical report: Item bank development and item response theory statistics. 
  Cella, D. and the Neuro-QOL Investigators
  http://www.neuroqol.org
  1/28/2010
• Measuring Quality of Life in Neurological Disorders-Final report of the Neuro-QOL study. 
  Cella, D and the Neuro-QOL Investigators
  http://www.neuroqol.org
  1/1/2010
• Boy with weakness. (Neuromuscular Update II presented at the 2009 Course F of the AANEM 56th Annual Meeting, San Diego, CA) 
  Han JJ, McDonald CM, Abresch RT, Carter GT, American Association of Neuromuscular and Electrodiagnostic Medicine
  Rochester, MN - 1/1/2009
• The effect of exercise training in dystrinopathies. (Neuromuscular Update II presented at the 2009 Course F of the AANEM 56th Annual Meeting, San Diego, CA) 
  Han JJ, McDonald CM, Abresch RT, Carter GT, American Association of Neuromuscular and Electrodiagnostic Medicine
  Rochester, MN - 1/1/2009
• Spinal muscular atrophy. (Neuromuscular Update I presented at the 2008 Course C of the AANEM 55th Annual Meeting, Providence, Rhode Island) 
  Leshner RT, McDonald CM, American Association of Neuromuscular and Electrodiagnostic Medicine
  Rochester, MN - 1/1/2008
• Clinical findings, measurement of impairment, and natural history of Duchenne's muscular dystrophy and spinal muscular atrophy. 
  McDonald CM, American Association of Electrodiagnostic Medicine
  1/1/1998
• New advances in the rehabilitation of neuromuscular diseases. 
  McDonald CM, American Association of Electrodiagnostic Medicine
  1/1/1998
• Spine Sonography in the follow-up of post-repair myelomeningocele. 
  Gersovich EO, Maslen L, Cronan MS, Poirier V, Anderson MW, McDonald C, Boggan JE, Ivanovic M, Proceedings of the 4th Congress of the International Society for Musculoskeletal Sonography (ISMUS),
  1/1/1998
• Predominantly proximal autosomal recessive spinal muscular atrophy. 
  McDonald CM, American Association of Electrodiagnostic Medicine
  1/1/1996
• Join now to see all