‘Maisie’s Army’

August 20, 2019

How a grassroots group is mobilizing to help toddlers access a lifesaving drug

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But a curable disease? If this controversial idea gains acceptance, it could radically change the way we treat getting old

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As someone who has lived with spinal muscular atrophy for all 30 years of my life, I was perplexed and disappointed that the recent approval of Novartis’ gene therapy Zolgensma was immediately overshadowed by outrage over the drug’s price: $2.125 million

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Trials of vaccines and treatments have not included enough female participants. Now that scientists are exploring possible cures, the need to enroll women is greater than ever

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Novartis AG is expected to begin selling Zolgensma, a possible cure for spinal muscular atrophy, after the FDA approves it this month. Novartis executives say Zolgensma’s ability to curb SMA—a muscle-wasting disease that often kills babies before age 2—explains the eye-popping price

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Scientists have long tried to duplicate the procedure that led to the first long-term remission 12 years ago. With the so-called London patient, they seem to have succeeded

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Success against sickle-cell would be “the first genetic cure of a common genetic disease” and could free tens of thousands of Americans from agonizing pain

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A bone-marrow transplant treated a patient’s leukemia — and his delusions, too. Some doctors think they know why

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