Results 101 to 110 of about 248,256 (115)

33 Insights into current adult duchenne muscular dystrophy (DMD) care in wales

33 Insights into current adult duchenne muscular dystrophy (DMD) care in wales
Saw Aung, Salmon Rachel, Edwards Rhiannon, Fletcher Claire, Davies Elizabeth, Davis Hayley, Skone Kate, Roberts Veronica, Marsh Eleanor, Powell Robert, Heledd Tomos, Donna Richards, Fiona Davies, Kate Greenfield, Georgina Carey   +14 more
semanticscholar   +1 more source

Practical use of risdiplam (EVRYSDI▾) in Individuals with Spinal muscular atrophy

Practical use of risdiplam (EVRYSDI▾) in Individuals with Spinal muscular atrophy
Hewamadduma Channa, Baranello Giovanni, Marini-Bettolo Chiara, Borrelli Osvaldo, Hoyle Natasha, Mangera Altaf, Martinez Anna E, Raslan Zaher, Scoto Mariacristina   +8 more
semanticscholar   +1 more source

Safety update: risdiplam clinical trial program for spinal muscular atrophy

Safety update: risdiplam clinical trial program for spinal muscular atrophy
Yeung Wai Yin, Baranello Giovanni, Chiriboga Claudia A, Servais Laurent, Darras Basil T, Day John W, Mercuri Eugenio   +6 more
semanticscholar   +1 more source

Quality of life and fatigue in adults with spinal muscular atrophy

Quality of life and fatigue in adults with spinal muscular atrophy
Parker Joseph, Chaudhry Summer, Kelly Phillip, Rutherford Anna, Atherton Claire, Hewamadduma Channa, Lilleker James   +6 more
semanticscholar   +1 more source

Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy

Nature Biotechnology, 2018
Seuk-Min Ryu, T. Koo, Kyoungmi Kim, Kayeong Lim, Gayoung Baek, Sang-Tae Kim, H. Kim, Daesik Kim, Hyunji Lee, Eugene Chung, Jin-Soo Kim   +10 more
semanticscholar   +1 more source

Defective membrane repair in dysferlin-deficient muscular dystrophy

Nature, 2003
Steven S Vogel, Chien-Chang Chen, Kevin P Campbell   +2 more
exaly  

Dystrophin: The protein product of the duchenne muscular dystrophy locus

Cell, 1987
Eric P. Hoffman, Robert H. Brown, Louis M. Kunkel   +2 more
semanticscholar   +1 more source

Genetic and pharmacologic inhibition of mitochondrial-dependent necrosis attenuates muscular dystrophy

Nature Medicine, 2008
Douglas P Millay, Christopher P Baines, Elisabeth R Barton   +2 more
exaly  

Modular flexibility of dystrophin: Implications for gene therapy of Duchenne muscular dystrophy

Nature Medicine, 2002
Scott Q Harper, Dongsheng Duan, John F Engelhardt   +2 more
exaly  

Diagnosis and management of Duchenne muscular dystrophy, part 2: respiratory, cardiac, bone health, and orthopaedic management

Lancet Neurology, The, 2018
David J Birnkrant, Carla M Bann, Benjamin A Alman   +2 more
exaly