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Suhellen Oliveira Da Silva was six months pregnant when she learned that the child she was carrying had the same disease that had left her firstborn son paralyzed and nearly mute. But this time, there was a treatment available that could make a profound difference. This baby could live a normal life.
The problem was the price: The treatment cost the equivalent of $1.7 million, and the public health system in Brazil, where the family lives, was refusing to pay for it.
So Ms. Da Silva went to court — and won. A judge ruled that the government had to buy the therapy for her younger son, Levi.
Today, Levi, 2, chats and claps and crawls, all things his older brother Lorenzo, 10, has never been able to do.
The treatment, called Zolgensma, a one-time infusion, is among the first in a new class of cutting-edge gene therapies that offer enormous promise for people with fatal or debilitating conditions — at hugely expensive prices. Its maker, the pharmaceutical company Novartis, has negotiated deals with national health systems and insurers to...