Pediatric Gene Therapy: Considerations for Planning, Execution & Long-Term Follow-up

June 20, 2017

The European approvals of Glybera in 2012 and Strimvelis in 2016 seem likely to be the vanguard of the therapeutic revolution heralded by the decoding of the human genome. Both of these treatments originated from academic centers of excellence and became the subjects of agreements with pharmaceutical companies Uniqure/Chiesi and GSK, respectively with the experience to navigate the manufacturing infrastructure and regulatory approvals processes that led to the products’ commercialization. The EMA has indicated the level of follow-up it expects to see for these patients, and the FDA’s perspective will likely be revealed with the first US approvals of gene therapies anticipated in 2017.

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Kalispell Regional Healthcare

Kalispell Regional Healthcare (KRH) is a 343-bed health care system located in Kalispell, Montana. KRH serves more than 170,000 people within a geographical region of 20,000 square miles and employs nearly 4,000 team members. Comprising the health care system are three acute-care hospitals (Kalispell Regional Medical Center, The HealthCenter, and North Valley Hospital), a skilled nursing facility (Brendan House), a fitness facility (The Summit Medical Fitness Center), a mental health and substance abuse facility (Pathways Treatment Center), a durable medical equipment company (Kalispell Medical Equipment) and more than 30 primary and specialty physician clinics.

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