Results 211 to 220 of about 48,181 (245)

Expression of full-length dystrophin reverses muscular dystrophy defects in young and old mdx4cv mice. [PDF]

J Clin Invest
Tasfaout H, McMillen TS, Reyes TR, Halbert CL, Tian R, Regnier M, Chamberlain JS.   +6 more
europepmc   +1 more source

miR-33 inhibition as a novel therapeutic approach for treating muscular dystrophy. [PDF]

EMBO Mol Med
Lopez MA, Alexander MS.
europepmc   +1 more source

Friend or Foe?: The Immune System's Dilemma With Dystrophin Replacement Therapy. [PDF]

JACC Basic Transl Sci
Townsend D.
europepmc   +1 more source

Enhanced therapeutic potential of a microdystrophin with an extended C-terminal domain. [PDF]

Mol Ther Methods Clin Dev
Foltz SJ, Qian R, Yang J, Elliott K, Heithoff BP, Jasick H, Smith JB, Karumuthil-Melethil S, Qiao C, Liu Y, Danos O.   +10 more
europepmc   +1 more source

Natural history of patients with nonsense mutation Duchenne muscular dystrophy treated with ataluren in Spain. [PDF]

Acta Myol
Armijo J, Nascimento A, Expósito J, Carrera L, Natera-de Benito D, Ortez C.   +5 more
europepmc   +1 more source

Editorial: Stem cell therapy for hereditary neuromuscular diseases. [PDF]

Front Cell Dev Biol
Goto K, Asakura A.
europepmc   +1 more source

A Sri Lankan boy with Emery-Dreifuss muscular dystrophy 5 presenting during infancy with persistent transaminitis. [PDF]

BMC Pediatr
Mettananda S, Vipulanayake T, Dineshika G, Suriapperuma T, Rajeindran M, Padeniya P.   +5 more
europepmc   +1 more source

Myonuclear domain-associated and central nucleation-dependent spatial restriction of dystrophin protein expression in a novel DMD mouse model

Chwalenia K, Feng V, Hemmer N, Hildyard JC, Roskrow LE, Piercy RJ, Wang ET, Aartsma-Rus A, van Putten M, Wood MJ, Roberts TC.   +10 more
europepmc   +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

AAV mini-dystrophin gene therapy for Duchenne muscular dystrophy: a phase 1b trial. [PDF]

Nat Med
Butterfield RJ, Shieh PB, Li H, Binks M, McDonnell TG, Ryan KA, Delnomdedieu M, Belluscio BA, Neelakantan S, Levy DI, Schwartz PF, Smith EC.   +11 more
europepmc   +1 more source