This chapter reviews existing functional motor and respiratory outcome measures in spinal muscular atrophy discussing how each of them fulfills a number of requirements including not only clinical usefulness but also statistical robustness, clinical meaningfulness, and other criteria regarding their suitability for clinical trials recently discussed with regulatory authorities.
Spinal Muscular Atrophy Motor Functional Scales and Measures of Pulmonary Function
MESSINA, Sonia;
2017-01-01
Abstract
This chapter reviews existing functional motor and respiratory outcome measures in spinal muscular atrophy discussing how each of them fulfills a number of requirements including not only clinical usefulness but also statistical robustness, clinical meaningfulness, and other criteria regarding their suitability for clinical trials recently discussed with regulatory authorities.File in questo prodotto:
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