Because of our connections to the SMA community, we are able to advance a comprehensive research program that is working to attack SMA from all sides.

Our Strategy

Cure SMA’s research strategy for spinal muscular atrophy (SMA) focuses on three main areas:

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Basic Research

Basic research investigates the causes and biology of SMA, often revealing new and more effective ways of making drugs.

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Drug Discovery

Drug discovery converts what we have learned about the causes and biology of SMA into new drug candidates that can be tested in clinical trials.

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Clinical Trials

A clinical trial tests new drugs and treatments, or new applications for approved drugs or treatments (e.g., different combinations or for different diseases).

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Pediatric Neuromuscular Clinical Research Network

In 2020, Cure SMA announced its funding commitment for the Pediatric Neuromuscular Clinical Research Network (PNCRN), providing $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 started collaborating with the SMA Foundation as a co-sponsor of the Network in 2018. 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.

The PNCRN has also 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.

Six sites comprise the PNCRN for SMA: 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 with SMA.