APNCR

The Adult & Pediatric Neuromuscular Clinical Research Network (APNCRN) was established to create a team of SMA clinical research experts to achieve the best possible clinical trial outcomes for the SMA community. The APNCRN has been involved in the seminal clinical trials that have led to the U.S. Food and Drug Administration (FDA) approvals of breakthrough SMA treatment options that have changed forever the natural course of the disease. Sites that comprise the APNCRN for SMA include:

• Boston Children’s Hospital, Boston, MA
• Children’s Hospital of Philadelphia, Philadelphia, PA
• Columbia University Irving Medical Center, New York, NY
• St. Jude Children’s Research Hospital, Memphis, TN
• Stanford University, Palo Alto, CA
• Lucile Packard Children's Hospital (LPCH) – Stanford Medicine, Palo Alto, CA
• Ann & Robert H. Lurie Children's Hospital of Chicago, Chicago, IL
• Northwestern Memorial Hospital – Northwestern University, Chicago, IL
• and the data coordinating center at the University of Rochester, Rochester, NY.

The APNCRN was originally established by the SMA Foundation in 2004 to create a robust natural history dataset which has been used as a key benchmark to assess effectiveness of potential therapies, and ultimately to obtain recent treatment approvals. Cure SMA became sole sponsor of the Network in 2018 in order to expand the scope of the network to collect real world evidence on approved treatments. Since 2018, Cure SMA has provided over $13M to the network.

Since 2012, the Trainor family has been building an endowment at Cure SMA to support several critical strategic initiatives. In the most recent year, the Erin Trainor Memorial Fund committed support to the efforts of the APNCR network. Cure SMA thanks the Trainors and their friends and family for specifically supporting the strategic goal of bringing new and especially adult-focused treatment and care hospitals into this important network.

APNCRN is eager to build on its past accomplishments. The Network’s work continues to develop and refine more sensitive outcome measures, conduct clinical trials for new treatment approaches, and to publish findings on the impact of approved treatments to help support broad access.