In India, Parents of Children with Rare Disease Plea for Help Online - The New York Times
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Gene therapy's promise and the high cost of hope - STAT
Obscure Model Puts a Price on Good Health—and Drives Down Drug Costs - WSJ
At $2.1 million, new gene therapy is the most expensive drug ever
Gene therapies must become miracles of medicine
Novartis' new $2.1 million drug has roots in Philadelphia. Here's how the city has impacted treatment of rare diseases.
Drug pricing legislation may not affect a new $2.1 million gene therapy drug - Roll Call