Two toddlers ‘miraculously’ regain abilities to stand and sit up after new therapy to treat most deadly genetic disease in infants

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Both of Danyelle Sun’s children suffer from a genetic disorder that has robbed them of the physical strength to even stand on their own.

Ruby, age six, and Landon, age three, have spinal muscular atrophy (SMA), which kills more infants than any other genetic disorder. 

But since they began getting treatments of the new drug, Spinraza, in July, Ruby and Landon have already begun to make remarkable progress.

This week, the New England Journal of Medicine (NEJM) published promising results from studies on the same drug Ruby and Landon are taking and a new gene therapy that could infants that often die of SMA within the first years of life.

Ruby and Landon will have to have Spinraza injections for the rest of their lives, but a gene therapy in clinical trials could stop the disease in its tracks with just one shot.

8bb9ed52254dc66920100f7a0aacd7ec Two toddlers 'miraculously' regain abilities to stand and sit up after new therapy to treat most deadly genetic disease in infants

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