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year, the prevalence of the disease in the U.S. is currently estimated to be between 5,000 and 7,000 boys and young men. Comparatively, the incidence of SMA is estimated to be 1 in 10,000 live births, or approximately 380 new cases per year. Given the young age and relatively similar size of the patient population, Zolgensma’s market access quest is of particular interest to GMaX. Of greatest interest, despite evidence of clinical benefit, the manufacturer has experienced reimbursement obstacles, with approximately 35% of payers restricting access to the patient populations included in clinical trial programs. Zolgensma has become today’s most expensive one-time therapy at $2.125 million. The manufacturer’s efforts have been further exacerbated by ongoing challenges with implementing novel financing solutions and outcomes measurements. In late October is was reported that about 100 infants have received treatment with Zolgensma, exceeding analysts’ expectations. According to the manufacturer’s CEO, approximately 90% of commercial patients and 30% of Medicaid patients are currently covered, and he expects public coverage to grow. Many additional gene therapies are expected to be approved over the next few years. Updated data generated by the MIT NEWDIGS consortium* projects that between 70 and 90 emerging gene therapy products will launch by 2031 (see Figure 1). This area will be used for addtional callouts and points of interests. Heading Here Adipiscing elit, sed do eiusmod tempor incididunt ut labore et dolore magna aliqua. Ut enim ad minim veniam. quis nostrud exercitation ullamco laboris nisi ut aliquip ex ea commodo consequat. 9 CONFIDENTIAL - FOR INTERNAL USE ONLY: DO NOT DISTRIBUTE Halpern SD, Ubel PA, Caplan AL. Solid-organ transplantation in HIV-infected patients. N Engl J Med. 2002;347:284-7.