DDx
ALS, DMD and Adapting Treatment Mechanisms for Genetic Variations
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Description
Understand the different mechanisms by which gene therapy can potentially treat specific genetic diseases – such as amyotrophic lateral sclerosis, or ALS, and Duchenne muscular dystrophy, or DMD.
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After a lifetime of treating her rare bone disease, X-linked hypophosphatemia, a patient is at the end of her rope. She’s in pain, frustrated, and nothing seems to help. So when a clinical trial presents itself, her doctor isn’t sure she’ll want to participate. While the decision to participate...
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DDx
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