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  • Chris McMillan

New hope offered to rare disease patients


21st December 2021

New hope offered to rare disease patients

China Daily Editor:Mo Hong'e

Early this month, the world's first therapy to treat spinal muscular atrophy, or SMA, a rare genetic disorder that affects muscle control, was added to the National Reimbursement Drug List in China.

It was the first time that a highly expensive drug to treat a rare condition had been included on the list.

This means that most children who have SMA, which results in death in 95 percent of patients up to 18 months of age, can be saved by an injection at a far more affordable price than previously.

Patients who have reached adulthood can also benefit from the therapy.

The treatment was priced at 700,000 yuan ($109,800) per dose when it was launched in China in April 2019. Official data showed that only 50 patients in the country had used it, paying the full price before the therapy was included on the national list. There are estimated to be 20,000 to 30,000 SMA patients in China.

Parents whose children have SMA said they had waited years for affordable medication, adding that it was unbelievable that their dreams had eventually come true.

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