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SMA newborn screening improves functional outcomes

Close up details as a family doctor carries out a heel prick blood test during newborn baby health screening, squeezing drops from foot for analysis

Children diagnosed with spinal muscular atrophy through a newborn screening initiative have better functional outcomes than their peers diagnosed after the development of clinical symptoms, the SMARTCARE study findings show.

Cerliponase alfa treatment shows extended efficacy for CLN2 disease

Cerliponase alfa treatment slows the progression of neuronal ceroid lipofuscinosis type 2 disease for at least 5 years, shows an open-label extension study in The Lancet Neurology.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.

Zonulin shows biomarker potential for celiac disease in at-risk children

Serum levels of the protein zonulin, high levels of which are associated with increased intestinal permeability, may be a useful biomarker for the development of celiac disease autoimmunity in children at genetic risk of the condition, suggest findings from the CD-GEMMA study.

This independent news story was supported by an educational grant from L’Institut Servier, Suresnes, France.