Results 111 to 120 of about 21,845 (197)

Givinostat enhances antisense oligonucleotide efficacy in the <i>mdx52</i> model of Duchenne muscular dystrophy. [PDF]

Mol Ther Nucleic Acids
Phongsavanh X, Gastaldi C, Mottais A, Doisy M, Le Coz O, de Haan S, Spitali P, Garcia L, Goyenvalle A.   +8 more
europepmc   +1 more source

Antisense oligonucleotide selection scheme for rare Duchenne muscular dystrophy mutations: Application to <i>DMD</i> exon 16 skipping. [PDF]

Mol Ther Nucleic Acids
Geoffroy-Guiraud M, Beley C, Campuzano S, Nguyen TH, Schmidt LM, Blaszykowski C, Krueger M, Garcia L, Guiraud S.   +8 more
europepmc   +1 more source

Timing matters: Exon skipping therapy is most effective when initiated early in a mouse model of Duchenne muscular dystrophy. [PDF]

Mol Ther Nucleic Acids
Stenler S, Huang J, van Westering TLE, Coenen-Stass AML, Jad Y, Krjutškov K, El Andaloussi S, Wood MJA, Roberts TC.   +8 more
europepmc   +1 more source

Exploring Dystrophin Expression and Mutations in the <i>DMD</i> and Dystrophin-Glycoprotein Complex Genes as Prognostic Factors in Leiomyosarcomas. [PDF]

Int J Mol Sci
Salazar J, Cerdà P, Artigas A, Orellana R, González A, Terés R, Arranz MJ, Bagué S, Fumagalli C, González JA, Peiró A, González-Quereda L, Rodríguez MJ, Gallardo E, Aguado M, Sebio A.   +15 more
europepmc   +1 more source

Microdystrophins partially rescue deficits of duchenne muscular dystrophy iPSC-cardiomyocytes. [PDF]

Mol Ther Adv
Keegan AR, Gonzalez AFGS, Zhang R, Doan H, Torres-Bigio SI, Lau F, Ly KN, Lai EC, Mamuyac LC, Bhimaraju S, Mishra A, Polen MK, Ortiz DS, Lam JH, Eguchi A.   +14 more
europepmc   +1 more source

Beyond the Diagnosis: A Comprehensive Case Report on Duchenne Muscular Dystrophy. [PDF]

Case Rep Crit Care
Shahin MAH, Asiri AA, Alotaibi AS, Majrashi H, Khubrani S, AlQarni SH, Alqurashi SS, Samti ME, AlQarni ZH.   +8 more
europepmc   +1 more source

Dystrophin-Deficient Muscular Dystrophy in a Jack Russell Terrier With a Large Deletion in the Canine DMD Gene. [PDF]

Vet Med Sci
Royaux E, Shelton GD, Jagannathan V, Leeb T, Drögemüller M.   +4 more
europepmc   +1 more source

CK-MM screening using neonatal dried blood spots may be inadequate at detecting all dystrophin mutation carriers. [PDF]

Transl Pediatr
Mehri S, Finsterer J.
europepmc   +1 more source

Novel DMD Frameshift Variant (p.Leu2017Profs*5) in Spectrin-Like Repeat 16 Expands the Mutational Spectrum of DMD. [PDF]

Mol Genet Genomic Med
Lin YC, Luxton GWG, Lee HJ, Lu YY, Yang HC, Hung KS, Lai MT, Hu CF.   +7 more
europepmc   +1 more source

FDA-approved antisense oligonucleotide therapies for duchenne muscular dystrophy: current status and future outlook. [PDF]

RNA Biol
Moriyama H, Moriyama S, Yokota T.
europepmc   +1 more source