P.341A phase 2 randomized placebo-control trial of domagrozumab in boys with Duchenne muscular dystrophy (DMD) von Marraffino, S., Binks, M., Palmer, J., Sherlock, S., Purohit, V., Charnas, L.

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Safety and disease monitoring biomarkers in Duchenne muscular dystrophy: results from a Phase II trial von Wagner, KR, Guglieri, M, Ramaiah, SK, Charnas, L, Marraffino, S, Binks, M, Vaidya, VS, Palmer, J, Goldstein, R, Muntoni, F

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Randomized, Double-Blind, Placebo-Controlled, Parallel-Group, Adult Laboratory Classroom Study of the Efficacy and Safety of PRC-063 (Extended-Release Methylphenidate) for the Trea... von Childress, Ann, Cutler, Andrew J., Marraffino, Andrea H., Bhaskar, Sailaja, Donnelly, Graeme

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Time to Failure After Rotator Cuff Repair: A Prospective Imaging Study von Iannotti, Joseph P., Deutsch, Allen, Green, Andrew, Rudicel, Sally, Christensen, Jared, Marraffino, Shannon, Rodeo, Scott

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Dual-energy X-ray absorptiometry measures of lean body mass as a biomarker for progression in boys with Duchenne muscular dystrophy von Sherlock, Sarah P., Palmer, Jeffrey, Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Tian, Cuixia, Mah, Jean K., Muntoni, Francesco, Guglieri, Michela, Butterfield, Russell J., Charnas, Lawrence, Marraffino, Shannon

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Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy von Wagner, Kathryn R., Abdel-Hamid, Hoda Z., Mah, Jean K., Campbell, Craig, Guglieri, Michela, Muntoni, Francesco, Takeshima, Yasuhiro, McDonald, Craig M., Kostera-Pruszczyk, Anna, Karachunski, Peter, Butterfield, Russell J., Mercuri, Eugenio, Fiorillo, Chiara, Bertini, Enrico S., Tian, Cuixia, Statland, Jeffery, Sadosky, Alesia B., Purohit, Vivek S., Sherlock, Sarah P., Palmer, Jeffrey P., Binks, Michael, Charnas, Lawrence, Marraffino, Shannon, Wong, Brenda L.

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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 von 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

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A phase Ib/IIa, open‐label, multiple ascending‐dose trial of domagrozumab in fukutin‐related protein limb‐girdle muscular dystrophy von Leung, Doris G., Bocchieri, Alex E., Ahlawat, Shivani, Jacobs, Michael A., Parekh, Vishwa S., Braverman, Vladimir, Summerton, Katherine, Mansour, Jennifer, Stinson, Nikia, Bibat, Genila, Morris, Carl, Marraffino, Shannon, Wagner, Kathryn R.

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Adapting training in real time: An empirical test of adaptive difficulty schedules von Marraffino, Matthew D., Schroeder, Bradford L., Fraulini, Nicholas W., Van Buskirk, Wendi L., Johnson, Cheryl I.

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5.10 EFFICACY AND SAFETY OF AN EXTENDED-RELEASE, ORALLY DISINTEGRATING METHYLPHENIDATE TABLET IN CHILDREN 6-12 YEARS OF AGE BASED ON ADHD RATING SCALE-IV SCORE AT BASELINE von Ann Catherine Childress, Kollins, Scott H., Cutler, Andrew J., Marraffino, Andrea, Sikes, Carolyn

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Quantitative muscle MRI biomarkers in Duchenne muscular dystrophy: cross-sectional correlations with age and functional tests von Sherlock, Sarah P, Zhang, Yao, Binks, Michael, Marraffino, Shannon

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Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Domagrozumab (PF‐06252616), an Antimyostatin Monoclonal Antibody, in Healthy Subjects von Bhattacharya, Indranil, Pawlak, Sylvester, Marraffino, Shannon, Christensen, Jared, Sherlock, Sarah P., Alvey, Christine, Morris, Carl, Arkin, Steven, Binks, Michael

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Progress: Responsiveness Validation of an Electronic Patient Reported Outcome (ePRO) in Adults with Sickle Cell Disease (NCT05407805) von Minniti, Caterina P, Bronte-Hall, Lanetta, Marraffino, Shannon, Healy, Jason, Rybin, Denis, Beidler, David, Huang, Lori, Shelton, Dana, Taylor, Steven, Goossens, Colet, Arkin, Steven

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Longitudinal functional and imaging outcome measures in FKRP limb-girdle muscular dystrophy von Leung, Doris G, Bocchieri, Alex E, Ahlawat, Shivani, Jacobs, Michael A, Parekh, Vishwa S, Braverman, Vladimir, Summerton, Katherine, Mansour, Jennifer, Bibat, Genila, Morris, Carl, Marraffino, Shannon, Wagner, Kathryn R

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6.66 A PHASE 4, OPEN-LABEL, SINGLE-DOSE PHARMACOKINETIC STUDY OF A NOVEL METHYLPHENIDATE EXTENDED-RELEASE ORALLY DISINTEGRATING TABLET FORMULATION IN PRESCHOOL-AGED CHILDREN von Sikes, Carolyn, Laage, Thomas, Volosov, Andrew, Hart, Alison, Engelking, Dorothy, Marraffino, Andrea

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5.21 POST HOC META-ANALYSIS OF MORNING AND EVENING BEHAVIORS IN CHILDREN WITH ADHD TREATED WITH DELAYED-RELEASE AND EXTENDED-RELEASE METHYLPHENIDATE FROM TWO PHASE 3 STUDIES von Pliszka, Steven R., Childress, Ann Catherine, Cutler, Andrew J., Marraffino, Andrea, DeSousa, Norberto J., Sallee, Floyd R., Incledon, Bev

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Serdexmethylphenidate/dexmethylphenidate capsules for children and adolescents with attention-deficit/hyperactivity disorder (ADHD): a plain language summary von Kollins, Scott H., Marraffino, Andrea, Cutler, Andrew J., Oh, Charles, Brams, Matthew N., Braeckman, Rene, Childress, Ann C.

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A Randomized, Controlled Laboratory Classroom Study of Serdexmethylphenidate and d-Methylphenidate Capsules in Children with Attention-Deficit/Hyperactivity Disorder von Kollins, Scott H, Braeckman, Rene, Guenther, Sven, Barrett, Andrew C, Mickle, Travis C, Oh, Charles, Marraffino, Andrea, Cutler, Andrew J, Brams, Matthew N

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Novel approaches to analysis of the North Star Ambulatory Assessment von 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

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Novel approaches to analysis of the North Star Ambulatory Assessment : Observations from a phase 2 trial von 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

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