"How can parents and patients be assured they are receiving the best care and treatment and what are the components of optimal care?”


PPMD gathered clinical providers, hospital administrators, industry, government, and parents together for a 2-day pre-conference meeting in order to address these critical issues. In the United States and around the world, there is considerable variability in care. Parents often feel it is necessary to travel long distances in order to access optimal care. In addition, variability in care impacts the ability to conduct clinical studies and trials. Below is a summary of findings and needs that came out of this meeting. PPMD is interested in partnering with the MDA to further develop the process of stratifying and credentialing clinics. A meeting is planned in the coming month. We will keep you informed as this project evolves.

Transforming Duchenne Care Summary


The Transforming Duchenne Meeting was held June 27-28, 2012.  Attending were 36 providers and hospital administrators representing 17 different hospitals from around the US.  Attendees also included 8 parent representatives, as well as representation from PPMD, MDA, CDC, Cystic Fibrosis Foundation, NeuroNext and industry.


  • Parents are dissatisfied with discrepancies in care and current models of care
  • Industry is having a difficulty conducting clinical trials/research due to discrepancies in care and models of care

Summary of needs:

  • Multidisciplinary clinics with coordination of services and concierge services
  • Communication back to families, primary care, local NM care providers, as well as in reverse (from primary care and local care providers back to NM clinic)
  • “Call center”:  available for communication regarding care for patients with DMD to the greater community (parents, emergency rooms, community providers), 24/7
  • Clinic stratification (such as Level I local clinics, level II regional clinics) that would inform parents of services offered at clinics (not Centers of Excellence, as that infers that others are not “excellent”)
  • Accreditation process for clinics in order to ensure that sites are indeed providing the services advertised
  • Transparency (clinic information readily available to parents)
  • Partnering with existing entities in order to coordinate funding, registries, research, etc. (PPMD, MDA, etc)
  • Outcome measuresfor 5 focus areas:
    • Communication
    • Glucocorticoids
    • Pulmonary
    • Cardiac
    • Bone health/endocrine

Kathi Kinnett, Director of Clinical Care
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Comment by Claire Diemler on July 17, 2012 at 9:03pm

Great summary of the needs of our community.

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