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We live in exciting times. Molecular therapies for previously untreatable genetic disorders are gradually being introduced. Currently, there are ≈300 gene or molecular therapy efforts, and a few of them, such as those in spinal muscular atrophy (SMA),1,2 have already been approved by national health authorities. They are called disease-modifying agents because there is prolongation of life without cure. Some of the trials have demonstrated better clinical response when patients are treated early in the course of the disease.3 Experts endorse their use.4 These therapies are very expensive, currently costing millions of dollars per individual, leading to escalating concerns by the scientific community.5,6 The medical community does not want to refrain from treating these children and adults with SMA, so the question is, how will health care systems or individual families pay for this?
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