Posted by Michael Wonder on 03 May 2018
Treatment for families battling a devastating rare disease
3 May 2018 - New Zealand infants suffering from a rare neuromuscular condition may soon be eligible for compassionate access to a life-changing new medicine.
Spinal muscular atrophy (SMA) is a rare genetic disease that causes muscle atrophy, weakness and shortened life expectancy. There are four types of SMA which vary in age of onset and severity, but 95% of children with SMA Type 1 will die within 18 months.
In recognition of the urgent need for treatment, pharmaceutical company Biogen will provide Spinraza, approved by the FDA since December 2016, free to New Zealand infants with SMA Type 1, the most severe form of the condition. The drug will be administered through regular spinal injections at Starship Children’s Hospital and has been shown to significantly improve motor function enabling affected infants to achieve milestones.
