Opinion: Limb girdle muscular dystrophy patients face a maddening reality
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When I was first diagnosed with limb girdle muscular dystrophy (LGMD), I was devastated to learn there wasn’t any treatment or cure. For those of us living with progressive diseases, time is not neutral. Every month a promising therapy is delayed, we will have irreversible loss of muscle function.
Rare disease drug development is in the midst of an extraordinary era of scientific innovation. Researchers are advancing gene therapies and targeted treatments unimaginable just a decade ago
Rare disease drug development is in the midst of an extraordinary era of scientific innovation. Researchers are advancing gene therapies and targeted treatments unimaginable just a decade ago
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