共 110 条
[1]
Kolb SJ(2015)Spinal Muscular Atrophy Neurol Clin 33 831-846
[2]
Kissel JT(2015)Spinal muscular atrophies Pediatr Clin North Am 62 743-766
[3]
Darras BT(1995)Identification and characterization of a spinal muscular atrophy-determining gene Cell 80 155-165
[4]
Lefebvre S(2011)Spinal muscular atrophy Orphanet J Rare Dis 6 71-1049
[5]
Burglen L(2007)Consensus statement for standard of care in spinal muscular atrophy J Child Neurol 22 1027-812
[6]
Reboullet S(2014)Comparative cost of illness analysis and assessment of health care burden of Duchenne and Becker muscular dystrophies in Germany Orphanet J Rare Dis 9 210-109
[7]
Clermont O(2001)PedsQL 4.0: reliability and validity of the Pediatric Quality of Life Inventory version 4.0 generic core scales in healthy and patient populations Med Care 39 800-1450
[8]
Burlet P(2010)The PedsQL in pediatric patients with Duchenne muscular dystrophy: feasibility, reliability, and validity of the Pediatric Quality of Life Inventory Neuromuscular Module and Generic Core Scales J Clin Neuromuscul Dis 11 97-1136
[9]
Viollet L(2002)American Spinal Muscular Atrophy Randomized Trials G. Outcome measures for pediatric spinal muscular atrophy Arch Neurocir 59 1445-812
[10]
D’Amico A(2003)American Spinal Muscular Atrophy Randomized Trials G. Reliability of 4 outcome measures in pediatric spinal muscular atrophy Arch Neurocir 60 1130-61