Opinion: The FDA’s approval of a new gene therapy for Duchenne muscular dystrophy won’t help me — but it gives me hope
This is not the final victory for people with Duchenne muscular dystrophy. It is only the beginning.
When I was diagnosed with Duchenne muscular dystrophy 20 years ago, there was no hope. The guidance the diagnosing doctor gave my parents was simple: Love your child as much as you can now because he won’t be here very long.
That was the inspiration that my mom needed to start CureDuchenne. Her intention — our whole family’s intention — was to cure this disease so no other parents would have to go through the same traumatic prognosis.
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