RG3039 is a new chemical entity that is the subject of worldwide composition of matter patents which, if allowed, will remain in force until 2028 prior to any patent term extensions.

About Families of Spinal Muscular Atrophy

Families of SMA funds and directs the leading SMA research programs to develop a treatment and cure for the disease. The successful results and progress that the organization has delivered, from basic research to drug discovery to clinical trials, provide real hope for families and patients impacted by the disease. The charity has invested over $50 million in research and been involved in funding half of all the ongoing novel drug programs for SMA. Families of SMA is a nonprofit 501(c)3 organization, with 31 Chapters and 85,000 members and supporters throughout the United States, and is dedicated to creating a treatment and cure by: funding and advancing a comprehensive research program; supporting SMA families through networking, information and services; improving care for all SMA patients; educating healthcare professionals and the public about SMA; enlisting government support for SMA; embracing all touched by SMA in a caring community. For more information: www.curesma.org.

About the Muscular Dystrophy Association

The Muscular Dystrophy Association (MDA) is the leading nonprofit health agency dedicated to curing muscular dystrophy, ALS, SMA and related diseases by funding worldwide research. The Association also provides comprehensive healthcare and support services, advocacy and education. In addition to funding more than 300 research projects worldwide, MDA maintains a national network of some 200 hospital-affiliated clinics; orchestrates hundreds of support groups for families affected by neuromuscular diseases; facilitates extraordinary local summer camp opportunities for thousands of youngsters fighting progressive muscle diseases. Known globally for the MDA Labor Day Telethon, the Association is the first nonprofit organization to receive a Lifetime Achievement Award from the American Medical Association “for significant and lasting contributions to the health and welfare of humanity.”

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