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Lipoprotein apheresis improves cardiovascular survival in HoFH

Portrait of little girl ready for blood testing

Patients with homozygous familial hypercholesterolaemia who initiate lipoprotein apheresis during childhood and adolescence have a reduced long-term risk for atherosclerotic cardiovascular disease and death, shows follow-up of two international registries.

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.