"Complete Cure 'One-Shot Treatment' Available, but Prohibitively Priced at Hundreds of Millions Won"
[Asia Economy Reporter Kang Juhee] A mother of a 12-month-old child suffering from Spinal Muscular Atrophy (SMA) appealed to allow her child to receive a treatment that can cure the disease.
On the 7th, a petition titled "Please help babies with muscular dystrophy receive the world's only gene therapy" was posted on the Blue House National Petition Board.
The petitioner, identified as A, who said she is the mother of a girl with Spinal Muscular Atrophy, explained, "If treatment is not received in time, it is a fatal disease that leads to death before the age of two," adding, "Perhaps our desperate wish reached the heavens, as a treatment close to a cure has arrived in Korea." Spinal Muscular Atrophy is a rare incurable disease in which muscles gradually atrophy.
The treatment A mentioned is 'Zolgensma,' which was approved for use in Korea last May. Zolgensma can cure the rare disease with just one administration for a lifetime effect, but due to its price reaching 2.5 billion KRW, patients are reportedly unable to realistically afford the treatment.
An article titled "Please help babies with muscular dystrophy receive the world's only gene therapy" posted on the Blue House National Petition Board on the 7th./Photo by Blue House Board Capture
A said, "There are many children suffering from muscular dystrophy to the extent that the term 'rare disease' seems inappropriate. Our children, who have more days to live than days lived, are living in pain unable to move their own bodies freely," she lamented, "Although they cannot speak, it is probably our children who want this more desperately than us parents."
She continued, "Zolgensma is a gene replacement therapy that can treat the fundamental cause of SMA and is called a one-shot therapy because it requires only a single lifetime administration. Babies who could not even sit have stood and walked, and the drug is so effective that normal life can be expected," she said, appealing, "Please help ensure that insurance coverage is urgently expanded so that children who cannot receive it due to lack of money or age restrictions can have access."
As of 11:10 PM on the 14th, this petition had gained over 5,400 supporters.
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