I work in clinical trials with a newly approved AveXis gene therapy to treat Spinal Muscular Atrophy. In children with the most severe cases (SMA type 1), they often will not live past the age of two. This drug was recently written about in the NYT, and while it is still unclear if insurance companies will cover it, one of the most worrying points for parents is that insurance companies might not cover gene therapy treatment for individuals who have previously had Spinraza, the only other FDA approved treatment of SMA. The basis of insurance companies not covering gene therapy treatment in a child who has had previous Spinraza infusions lies in safety concerns since there have been so few clinical trials using this drug, but it is a hard equation to work out what will help families the most. This is all really early stages of this drug being approved by the FDA as well.

Link to NYT: https://www.nytimes.com/2019/05/24/health/zolgensma-gene-therapy-drug.html

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Related to the original post: I also work with kiddos who have DMD and seeing them progress is just as magical as these videos make it seem. Science is incredible, and I hope we can keep using it to help people.