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8 th September SCMP
excerpted from:
//Josy Chow Pui-shan, who was born with spinal muscular atrophy (SMA), a genetic disorder that gradually destroys physical abilities such as walking and breathing, urged the government to introduce nusinersen, marketed as Spinraza, the first approved drug for SMA by the US Food and Drug Administration.//“It is really tiring to write with two fingers only ... but I don’t want other children [in a similar condition] to experience the hardship I have had growing up,” Chow said.//
#SMA #DrugApproval #Spinraza #SpinalMuscularAtrophy #HKARD #HongKongAllianceForRareDiseases
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