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A new RNA datatool aims to help patients with genetic diseases to receive a quicker diagnosis and improve their access to therapies.

The development of a world-first nasal spray vaccine could be a turning point in protection against whooping cough, helping stop the spread of the respiratory infection to vulnerable babies.

New research has shown newborn bloodspot screening is more effective than conventional clinical diagnosis pathways for improving health outcomes in infants with SMA.

Experts are calling for research to look at how gene therapies can be adapted and scaled to become standard practices for people right around Australia.

In a new clinical trial being led by The Children’s Hospital at Westmead, doctors are testing whether drug used to help adult Alzheimer’s patients can help improve some of the devastating features of Rett syndrome.