Asya’s mother, Güler Polatlı, emphasised, however, that this was preliminary treatment only, and funds are still required to provide the one-off gene therapy treatment which costs 2.4 million dollars.
The gene therapy treatment named Zolgensma*, will slow down and potentially halt the muscle-wasting disease.
* The main study showed that Zolgensma reduces the need for artificial ventilation in babies with spinal muscular atrophy. In this study, 20 out of the 22 babies given Zolgensma were alive and breathing without a permanent ventilator after 14 months, when normally only a quarter of untreated patients would survive without needing a ventilator.
The study also showed that Zolgensma can help babies sit unaided for at least 30 seconds. 14 out of the 22 babies given Zolgensma were able to do so after 18 months, a milestone that is never achieved in untreated babies with severe forms of the disease.
Asya’s mother said, “There is still a need for money for gene therapy, it is important for us to continue the campaign“.
“The campaign continues to collect gene therapy money. The muscle therapy applied now only saves us time until the other treatment, but there is still a need for another drug for gene therapy that will provide the definitive solution. Please let’s continue the campaign“, Güler Polatlı said.
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