Transforming Duchenne Care Update: Part I - Meeting with the MDA

It’s been a little while since our last update, but I wanted to wait until I had definite news. It’s been an exciting month for Transforming Duchenne Care (TDC), and I will do my best to get everyone up to speed. We had two important and informative meetings. Below is an update on our recent meeting with the MDA. Click here to read about our meeting with the Cystic Fibrosis Foundation.


On Monday, November 5, 2012, Pat Furlong, Sharon Hesterlee and I were very fortunate to be able to spend several hours with the Muscular Dystrophy Association in Tucson, AZ.  Nine members of the MDA clinical staff, including Valerie Cwik, Annie Kennedy and Jodi Wolff, discussed how PPMD and the MDA might best partner in order to meet the needs of patients and families living with Duchenne, which were formalized at the TDC Conference in June, 2012.  The Transforming Duchenne Care Initiative was formed in order to meet those needs. The mission of the TDC Initiative was discussed. The mission is as follows:


Transforming Duchenne Care is an initiative dedicated to reducing disparities in the healthcare of patients living with Duchenne and Becker muscular dystrophy (DBMD) in North America. The Transforming Duchenne Initiative strives to provide transparency to families regarding the care provided to DBMD patients in neuromuscular clinics across North America, empowering patients and families to make informed decisions regarding where to obtain components of multidisciplinary care and advocate for a higher level of care. We will encourage neuromuscular providers to engage in quality improvement efforts to move toward baseline optimal care and facilitate family access. We will work to develop methodology that will assure families that those neuromuscular providers who are providing healthcare in adherence with the best and most current guidelines available.


The two phases of the Transforming Duchenne Care Initiative were discussed.

  • Phase I: Transparent Clinical Services and Specialty/Sub-specialty Care

    Goal: To make information regarding access to specialty and subspecialty care available to DBMD patients in North America. This information will be transparent and easily accessible to patients and families via an online website and written materials. This will enable families living with DBMD to make informed decisions regarding access to specialty care at individual clinics.
  • Phase II: Transparent Clinical Care

    Goal: Encouraging clinics to adopt baseline levels of care as described in the CDC Care Considerations and/or more current guidelines, thereby helping to ensure that patients living with DBMD receive consistent, optimal baseline care in accordance with these guidelines. Care in five sub-specialty areas will be reviewed. These areas include: neurology, cardiology, pulmonary, physical therapy/physical medicine and rehabilitation, and coordination/communication.


We began by discussing Phase I.The MDA has recently completed an extensive clinical survey, which gathered clinical data related to clinical services and specialty/sub-specialty care available at MDA clinics across North America. We agreed that making this information available to patients and families would empower families to make informed decisions regarding where to obtain components of multidisciplinary care. The MDA has generously agreed to post this information on their website. The first conference call of the TDC Core Team, made up of 15 members who represent PPMD, industry, the CDC, the MDA, clinicians and parents, was November 7, 2012. The Core Team plans to review the information that the MDA has gathered and determine which key components should be included on the MDA website. The information will be reviewed and key components will be determined before the next call, December 5, 2012. The information will be posted by February 1, 2013.


In addition, the MDA will soon launch their patient registry. The MDA registry will be piloted in 25 clinics across North America this year and, over the next 5 years, will expand to the remainder of their 200 clinics. The amount and extent of the data that they plan to collect is extremely impressive and will tremendously contribute to the enhancement of standards of care. Continued collaboration with the MDA will begin to inform Phase II of the TDC Initiative.

Kathi Kinnett, Director of Clinical Care
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