Neuromuscular diseases: Progress in gene discovery drives diagnostics and therapeutics

The Lancet Neurology

Volumn 14, Issue 1, 2015, Pages 13-14

  Fratta, Pietro ^a   Hanna, Michael G ^a  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

GENETICS; HUMAN; NEUROMUSCULAR DISEASES;

HUMANS; NEUROMUSCULAR DISEASES;

EID: 84918568123     PISSN: 14744422     EISSN: 14744465     Source Type: Journal    
DOI: 10.1016/S1474-4422(14)70239-X     Document Type: Note

References (6)

1. Voit T, Topaloglu H, Straub V, et al. Safety and efficacy of drisapersen for the treatment of Duchenne muscular dystrophy (DEMAND II): an exploratory, randomised, placebo-controlled phase 2 study. Lancet Neurol 2014, 13:987-996.
2. Bushby K, Finkel R, Wong B, et al. Ataluren treatment of patients with nonsense mutation dystrophinopathy. Muscle Nerv 2014, 5:47-87.
3. Rohrer J, Isaacs A, Mizlienska S, Mead S, Lashley T, Wray S. C9orf72 expansions in frontotemporal dementia and amyotrophic lateral sclerosis. Lancet Neurol (in press).
4. Naryshkin NA, Weetall M, Dakka A, et al. Motor neuron disease. SMN2 splicing modifiers improve motor function and longevity in mice with spinal muscular atrophy. Science 2014, 345:688-693.
5. Bryson JB, Machado CB, Crossley M, et al. Optical control of muscle function by transplantation of stem cell-derived motor neurons in mice. Science 2014, 344:94-97.
6. Périé S, Trollet C, Mouly V, et al. Autologous myoblast transplantation for oculopharyngeal muscular dystrophy: a phase I/IIa clinical study. Mol Ther J Am Soc Gene Ther 2014, 22:219-225.