ReviewConcerns about the design of clinical trials for spinal muscular atrophy
References (27)
- et al.
The neurobiology of childhood spinal muscular atrophy
Neurobiol Dis
(1996) - et al.
Quantitative analyses of SMN1 and SMN2 based on real-time lightCycler PCR: fast and highly reliable carrier testing and prediction of severity of spinal muscular atrophy
Am J Hum Genet
(2002) Small clinical trials
- et al.
The SMN complex, an assemblyosome of ribonucleoproteins
Curr Opin Cell Biol
(2002) - et al.
Synaptic alterations of anterior horn cells in Werdnig-Hoffmann disease
Pediatr Neurol
(1996) - et al.
A placebo-controlled trial of gabapentin in spinal muscular atrophy
J Neurol Sci
(2001) - et al.
Pilot trial of phenylbutyrate in spinal muscular atrophy
Neuromuscul Dis
(2004) - et al.
Motor unit number estimation in infants and children with spinal muscular atrophy
Muscle Nerve
(2002) Infantile muscular atrophy: a prospective study with particular reference to a slowly progressive variety
Brain
(1964)- et al.
Prospective analysis of strength in spinal muscular atrophy
J Child Neurol
(2000)
90th ENMC International Workshop: European spinal muscular atrophy randomised trial (EuroSMART) 9–10 February 2001, Naarden, The Netherlands
Neuromuscul Dis
(2002)
Profiles of neuromuscular diseases. Spinal muscular atrophy
Am J Phys Med Rehabil
(1995)
Pilot trial of albuterol in spinal muscular atrophy
Neurology
(2002)
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