Paediatrics and Child Health (East Africa)
Emerging genetic therapies for rare disorders at high cost, cannot realistically address the global burden of disease. Stakeholders must develop new pathways to ensure safe, fair and sustainable provision of such therapies.
Journal of the International Child Neurology Association
Triki, C. C.,
Wilmshurst, J. M.,
(2019). Position Statement:Emerging genetic therapies for rare disorders. Journal of the International Child Neurology Association, 1(1).
Available at: https://ecommons.aku.edu/eastafrica_fhs_mc_paediatr_child_health/175
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