Why the study?
Does calibrated short-amplicon melt profiling accurately identify homozygous deletions of the SMN1 gene for newborn screening of spinal muscular atrophy?
Population
Approximately 1000 purified DNA samples, 100 self-created dried blood spots, and >1200 dried blood spots…
Design
Other
Authors
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Supports assay feasibility for SMA newborn screening; leaves open need for prospective validation before routine adoption.
Does calibrated short-amplicon melt profiling accurately identify homozygous deletions of the SMN1 gene for newborn screening of spinal muscular atrophy?
A newly developed DNA-based newborn screening assay using calibrated short-amplicon melt profiling can reliably identify presymptomatic spinal muscular atrophy patients.
Dobrowolski et al. (2012) studied this question.
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