In the midst of the ongoing Coronavirus (COVID-19) global pandemic, Cure SMA is excited to announce its new funding for the Pediatric Neuromuscular Clinical Research Network (PNCRN). In 2020, Cure SMA will provide $1.2 million to this Network of highly skilled clinical trial investigators, clinical evaluators, clinical coordinators, statisticians, and data management personnel.
The PNCRN was established by the SMA Foundation in 2004 to create a team of SMA clinical trial experts that would integrate clinical research, education, and care to help achieve the best possible clinical trial outcomes for the SMA community. Cure SMA has collaborated with the SMA Foundation as co-sponsor of the Network since 2018 and is eager to build on its past accomplishments. Most notably, the SMA Natural History Study was clearly defined by the Network and has been used as a key benchmark to assess safety and effectiveness of potential therapies, and ultimately to obtain recent treatment approvals.
In recent years, the PNCRN 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. The Network’s work continues to develop and refine more sensitive outcome measures, conduct ongoing clinical trials, accelerate newborn screening programs, and identify promising new treatment approaches. Cure SMA sees its support of the PNCRN leading to more ground-breaking collaboration in SMA research and clinical care, especially as we expand the number of effective treatments for SMA and pursue the ultimate vision of a cure.
Even during this pandemic, Cure SMA is pleased to have been able to rapidly provide emergency support programs to thousands of families, while keeping our funding for core programs moving forward. This includes new basic research grants that we recently awarded and the expansion of our Cure SMA Care Center Network to now 20 sites. We have even been able to take on new activities such as the PNCRN, and see the investment in the Network as critical in our mission to advance clinical research and trials and increase treatment and care.
Six sites comprise the PNCRN for SMA, including Boston Children’s Hospital, Boston, MA; Children’s Hospital of Philadelphia, Philadelphia, PA; Columbia University Irving Medical Center, New York, NY; Nemours Children’s Health System, Orlando FL; Stanford University, Palo Alto, CA; and the data coordinating center at the University of Rochester, Rochester, NY. These clinical research and treatment sites have also now been integrated into the established Cure SMA Care Center Network, which will lead to real-world evidence that increases access to approved treatments for SMA and improves care for individuals and families affected by SMA.