Results 201 to 210 of about 8,752 (229)

Onasemnogene abeparvovec for presymptomatic infants with three copies of SMN2 at risk for spinal muscular atrophy: the Phase III SPR1NT trial

Nature Medicine, 2022
Kevin A Strauss, Michelle A Farrar Mbbs, Francesco Muntoni   +2 more
exaly  

TMEM43 mutations in emery‐dreifuss muscular dystrophy‐related myopathy

Annals of Neurology, 2011
W. Liang, H. Mitsuhashi, E. Keduka, I. Nonaka, S. Noguchi, I. Nishino, Y. Hayashi   +6 more
semanticscholar   +1 more source

Base editing rescue of spinal muscular atrophy in cells and in mice

Science, 2023
, Zaneta Matuszek
exaly  

Emery-Dreifuss Muscular Dystrophy

2015
Hacer Durmus, Piraye Serdaroglu-Oflazer, Feza Deymeer   +2 more
openaire   +1 more source

Emery-Dreifuss Muscular Dystrophies

2023
openaire   +1 more source

Wearable full-body motion tracking of activities of daily living predicts disease trajectory in Duchenne muscular dystrophy

Nature Medicine, 2023
Valeria Ricotti, Thomas Voit, Aldo A Faisal   +2 more
exaly  

Emery–Dreifuss Muscular Dystrophy

2017
Satish V. Khadilkar, Rakhil S. Yadav, Bhagyadhan A. Patel   +2 more
openaire   +1 more source

Emerin binding to Btf, a death-promoting transcriptional repressor, is disrupted by a missense mutation that causes Emery-Dreifuss muscular dystrophy.

European Journal of Biochemistry, 2004
T. Haraguchi, James M. Holaska, Miho Yamane, T. Koujin, Noriyo Hashiguchi, C. Mori, K. Wilson, Y. Hiraoka   +7 more
semanticscholar   +1 more source

Pharmacological strategies for muscular dystrophy

Nature Reviews Drug Discovery, 2003
Tejvir S Khurana, Kay E Davies
exaly  

Long-term effects of glucocorticoids on function, quality of life, and survival in patients with Duchenne muscular dystrophy: a prospective cohort study

Lancet, The, 2018
Craig M Mcdonald, Erik K Henricson, Paula R Clemens   +2 more
exaly