Patient Advocacy Organizations Join Forces to Improve Care for Spinal Muscular Atrophy

Thursday, March 21, 2019


Patient advocacy organizations are partnering to obtain real-world evidence to advance research, drug development, and access to therapies for children and adults with spinal muscular atrophy (SMA). 

Cure SMA and Parent Project Muscular Dystrophy (PPMD) will collaborate to aggregate clinical data from patients with either SMA or Duchenne muscular dystrophy. Prometheus Research, the organizations’ partner for data and technology, will integrate and repurpose data from disparate healthcare sources. 

The Cure SMA clinical data registry collects electronic health records of patient data from a growing number of sites in the Cure SMA Care Center Network. This data will be the basis for a wide range of clinical research and quality-improvement initiatives. 

The primary goals of the Cure SMA Care Center Network are to understand how current therapies are working for SMA patients and to establish an evidence-based standard of care that will benefit patients. 

 "With new treatments, the experience of SMA is rapidly shifting, and healthcare providers need to adapt quickly to these changing needs," said Kenneth Hobby, president of Cure SMA. "The goal of the SMA Care Center Network is to develop an evidence-based standard of care that will address this changing landscape, expand access to approved SMA treatments, and help accelerate and enhance insurance coverage and reimbursement."

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