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Meeting report: the 2020 FSHD International Research Congress
Kyba M, Bloch RJ, Dumonceaux J, et al.
Skeletal Muscle, 2020, 10, 1, p 36
Randomized phase 2 trial and open-label extension of domagrozumab in Duchenne muscular dystrophy
Wagner KR, Abdel-Hamid HZ, Mah JK, et al.
Neuromuscular disorders : NMD, 2020, 30, 6, p 492
Longitudinal functional and imaging outcome measures in FKRP limb-girdle muscular dystrophy
Leung DG, Bocchieri AE, Ahlawat S, et al.
BMC neurology, 2020, 20, 1, p 196
The care of patients with Duchenne, Becker, and other muscular dystrophies in the COVID-19 pandemic
Veerapandiyan A, Wagner KR, Apkon S, et al.
Muscle & Nerve, 2020
Applying genome-wide CRISPR-Cas9 screens for therapeutic discovery in facioscapulohumeral muscular dystrophy
Lek A, Zhang Y, Woodman KG, et al.
Science translational medicine, 2020, 12, 536
Improving translatability of preclinical studies for neuromuscular disorders: lessons from the TREAT-NMD Advisory Committee for Therapeutics (TACT)
Willmann R, Lee J, Turner C, et al.
Disease models & mechanisms, 2020, 13, 2
A decade of optimizing drug development for rare neuromuscular disorders through TACT
Wagner KR, de Luca A, Caizergues D, et al.
Nature reviews. Drug discovery, 2020, 19, 1, p 1
Facioscapulohumeral Muscular Dystrophies
Wagner KR
Continuum (Minneapolis, Minn.), 2019, 25, 6, p 1662
Diabetes, Metformin, and Cancer Risk in Myotonic Dystrophy Type I
Alsaggaf R, Pfeiffer RM, Wang Y, et al.
International journal of cancer, 2019
Stem cell-based therapies for Duchenne muscular dystrophy.
Sun C, Serra C, Lee G, et al.
Experimental neurology, 2019, 323
AAV9 Edits Muscle Stem Cells in Normal and Dystrophic Adult Mice.
Nance ME, Shi R, Hakim CH, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2019, 27, 9, p. 1568-1585
Benign tumors in myotonic dystrophy type I target disease-related cancer sites
Alsaggaf R, St George DMM, Zhan M, et al.
Annals of clinical and translational neurology, 2019, 6, 8, p 1510
The NIH Toolbox for cognitive surveillance in Duchenne muscular dystrophy
Thangarajh M, Kaat AJ, Bibat G, et al.
Annals of clinical and translational neurology, 2019, 6, 9, p 1696
Cancer Risk in Myotonic Dystrophy Type I: Evidence of a Role for Disease Severity
Alsaggaf R, Alsaggaf R, St George DMM, et al.
JNCI cancer spectrum, 2018, 2, 4
Cardiac Management of the Patient With Duchenne Muscular Dystrophy
Buddhe S, Buddhe S, Cripe L, et al.
Pediatrics, 2018, 142, S2, p S72
Neurology Care, Diagnostics, and Emerging Therapies of the Patient With Duchenne Muscular Dystrophy
Leigh F, Leigh F, Ferlini A, et al.
Pediatrics, 2018, 142, S2, p S5
A Transition Toolkit for Duchenne Muscular Dystrophy
Trout CJ, Trout CJ, Case LE, et al.
Pediatrics, 2018, 142, S2, p S110
Diagnosis and management of Duchenne muscular dystrophy, part 1: diagnosis, and neuromuscular, rehabilitation, endocrine, and gastrointestinal and nutritional management
Birnkrant DJ, Bushby K, Bann CM, et al.
The Lancet. Neurology, 2018, 17, 3, p 251
Morpholino-mediated Knockdown of DUX4 Toward Facioscapulohumeral Muscular Dystrophy Therapeutics
Chen JC, King OD, Zhang Y, et al.
Molecular therapy : the journal of the American Society of Gene Therapy, 2016, 24, 8, p 1405
Clinical practice considerations in facioscapulohumeral muscular dystrophy Sydney, Australia 21 September 2015
Tawil R, Mah JK, Baker S, et al.
Neuromuscular disorders : NMD, 2016, 26, 7, p 462
Dystrophin-deficient dogs with reduced myostatin have unequal muscle growth and greater joint contractures
Kornegay JN, Bogan DJ, Bogan JR, et al.
Skeletal Muscle, 2016, 6, 14, p. 1-17
Individual epigenetic status of the pathogenic D4Z4 macrosatellite correlates with disease in facioscapulohumeral muscular dystrophy
Jones TI, King OD, Himeda CL, et al.
Clinical Epigenetics, 2015, 7
Results of a Two-Year Pilot Study of Clinical Outcome Measures in Collagen VI- And Laminin alpha2-related Congenital Muscular Dystrophies
Meilleur KG, Jain MS, Hynan LS, et al.
Neuromuscular disorders : NMD, 2015, 25, 1, p 43
The TREAT-NMD advisory committee for therapeutics (TACT): an innovative de-risking model to foster orphan drug development
Heslop E, Csimma C, Straub V, et al.
Orphanet journal of rare diseases, 2015, 10
Sildenafil does not improve cardiomyopathy in Duchenne/Becker muscular dystrophy
Leung DG, Herzka DA, Thompson WR, et al.
Annals of neurology, 2014, 76, 4, p 541
