Results 181 to 190 of about 362,296 (275)

Euglycemic ketoacidosis in a non-diabetic patient with Duchenne muscular dystrophy on dapagliflozin. [PDF]

Intern Emerg Med
Gutiérrez-Baena AM, Tegido Mateu M, Álvarez Schlegel P, Clotet-Vidal S.   +3 more
europepmc   +1 more source

Progress toward Gene Therapy for Duchenne Muscular Dystrophy.

Molecular Therapy, 2017
J. Chamberlain, J. Chamberlain
semanticscholar   +1 more source

Isolated Transaminitis as a Sentinel Sign of Duchenne Muscular Dystrophy in an Infant: A Case Report. [PDF]

Cureus
Khalid A, Chaudhry T, Liaqat A, Tufts L.
europepmc   +1 more source

Defective membrane fusion and repair in Anoctamin5-deficient muscular dystrophy.

Human Molecular Genetics, 2016
D. Griffin, Ryan W. Johnson, Jarred M. Whitlock, E. Pozsgai, E. Pozsgai, K. Heller, W. Grose, W. Arnold, Z. Sahenk, H. Hartzell, L. Rodino-Klapac, L. Rodino-Klapac   +11 more
semanticscholar   +1 more source

A novel compound heterozygous variant in LAMA2 gene in a family with merosin-deficient congenital muscular dystrophy. [PDF]

BMC Med Genomics
Nejati P, Falsafi N, Alimoradi E, Khosravi T, Kamari M, Oladnabi M, Alibakhshi R.   +6 more
europepmc   +1 more source

Diagnosis and etiology of congenital muscular dystrophy: We are halfway there

Annals of Neurology, 2016
G. O'grady, M. Lek, S. Lamandé, L. Waddell, E. Oates, J. Punetha, R. Ghaoui, S. Sandaradura, H. Best, S. Kaur, M. Davis, N. Laing, F. Muntoni, E. Hoffman, D. MacArthur, N. Clarke, S. Cooper, K. North   +17 more
semanticscholar   +1 more source

When pullulanase needs a little push: MyoAAV capsids enhance gene therapy for GSD IIIa

Molecular Therapy: Methods & Clinical Development
Sree Venigalla, Christina A. Pacak
doaj   +1 more source

Synthetic bottlebrush block copolymer prevents disease onset in Duchenne muscular dystrophy. [PDF]

Proc Natl Acad Sci U S A
Cohen H, Angulski ABB, Quick JD, Kuebler TS, Thompson BR, Bauer J, Hahn D, Townsend D, Hassler JF, Hackel BJ, Lodge TP, Sham YY, Bates FS, Metzger JM.   +13 more
europepmc   +1 more source

Cancer and benign tumors in myotonic dystrophy, facioscapulohumeral muscular dystrophy, and oculopharyngeal muscular dystrophy: a 23-year, single-center, retrospective study. [PDF]

Acta Myol
Bareja N, Desai B, Vytopil M, Srinivasan J, Ghasemi M.   +4 more
europepmc   +1 more source

Long-Term Evaluation of Givinostat in Duchenne Muscular Dystrophy, and Natural History Comparisons. [PDF]

Ann Clin Transl Neurol
McDonald CM, Guglieri M, Vučinić D, Acsadi G, Brandsema JF, Bruno C, Finanger EL, Harper A, Lobato ML, Masson R, Muelas N, Munell F, Nevo Y, Péréon Y, Phan H, Sansone VA, Scoto M, Willis T, Finkel RS, Vandenborne K, Cazzaniga S, Montrasio S, Alessi F, Bettica P, Mercuri E, Givinostat Study 51 Investigators, Cooperative International Neuromuscular Research Group (CINRG) Duchenne Natural History Study (DNHS) Investigators, ImagingDMD Investigators.   +27 more
europepmc   +1 more source