Table 1. Compares curative treatment, current gene therapy, and standard of care. Abbreviations: EHL-enhanced half-life; TMA-thrombotic microangiopathy; DRG-dorsal root ganglion *Access may be limited in various parts of the world, but it is available and is approved for use in patients with mild, moderate, and severe hemophilia with or without an inhibitor. $ other than patients who become alloimmunized. & not reported in clinical trials for hemophilia or spinal muscular atrophy (SMA) but has been reported in post-marketing surveillance for SMA and in clinical trials for other genetic disorders. # can be avoided by limiting co-exposure to bypass agents containing factor IX.