The study is being touted as a major breakthrough that has taken the leading genetic cause of infant death into something that will be screened for and can be treated.
- Additional research centers now enrolling patients in Australia and New Zealand - Company has achieved 50 percent of target enrollment in global Phase 3 trial LONDON
Newborn screening and early treatment for spinal muscular atrophy can save both lives and money
Gene therapy for spinal muscular atrophy might have a high up-front price tag. But by screening and treating infants early, the therapy can save both lives and money in the long term.
By the time an infant with SMA starts showing symptoms, they ve already lost 90 per cent of their nerves – which is why early detection and treatment is so important. Photo: Unsplash
Spinal muscular atrophy (SMA), a type of motor neurone disease, is one of the deadliest genetic illnesses an infant can be diagnosed with. Caused by a missing or faulty gene called ‘SMN1’, the disease causes muscles to shrink and become weaker, limiting a baby’s ability to sit, walk, crawl, or even hold their head up.
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Funding supports Australians living with rare disease
UNSW Sydney researchers have been awarded $1.9 million for a project that will develop and deliver rare disease resources, education and training.
UNSW has been awarded $1.9 million in federal government funding for the Rare Awareness, Education, Support and Training (RArEST) project. The project will develop and deliver rare disease awareness resources, education, support, and training with a focus on mental health, and social and emotional wellbeing.
The funding is part of the $3.3 million announced by federal Minister for Health and Aged Care, Greg Hunt, to support the approximate two million Australians living with one of 7000 rare diseases.
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