January 24, 2018 / Care

Updated Care Consideration Guidelines for Duchenne Published

PPMD is thrilled to announce that after many years in development, the Centers for Disease Control (CDC) has published updated Care Considerations for Duchenne in The Lancet Neurology. Since first being published in 2010, significant advances in Duchenne research and care made it evident that an update to these standards of care was long overdue. Later this year, a family-friendly version of these standards of care will be released.


The Evolution of Care

When you have Duchenne, optimal comprehensive care is critical.  And it must be accessible wherever you are, wherever you live.

One of the first questions following a diagnosis is around the ‘standard of care.’   What components of care are ”standardized,” that is, what have we learned over time and what do the experts recommend?  What does ‘optimal comprehensive care’ look like?  What are the components?  What are the issues that need to be addressed?  What interventions are important and where is the data that will be useful to decisions about care?

In 2001, thanks to our champions in Congress, the Muscular Dystrophy Care Act (MD-CARE Act) was signed into law.  One of the requirements of that legislation was the development of Standards of Care.  The Centers for Disease Control (CDC) orchestrated the first set of standards called Care Considerations for Duchenne, published in The Lancet Neurology in 2010. PPMD was involved in the Steering Committee and members of the PPMD team were honored to have chaired several sections. These were the first international ‘standards’ for Duchenne and the The Lancet Neurology publication, as well as, a family-friendly version, were published and translated into more than 30 languages.

Since 2010, there has been a considerable increase in scientific evidence and new medical practices making it essential for an update. Steroids, aggressive cardiac care, and non-invasive ventilation have added years to the lifespan of Duchenne.

In 2008, PPMD helped lead the re-authorization of the MD CARE Act and again, in 2014, PPMD helped lead the passage of the MD-CARE Amendments Act.  One aspect of those amendments of 2014 included an update the Care Considerations.  PPMD, once again, has been very involved on both the steering committee, as well as chairing several subspecialty sections, in these updated guidelines.

What Has Changed

There are several changes with the update. The original Care Considerations included recommendations for eight areas of care, primarily from a pediatric focus:

  • Diagnostics
  • Corticosteroid management
  • Cardiac
  • Pulmonary
  • Physical therapy/physical medicine & rehabilitation
  • GI/nutrition,
  • Orthopedic/surgical
  • Psychosocial

The updated Care Considerations include all of these eight areas and have added:

  • Primary/Emergency Care
  • Endocrine Management
  • Transition of Care to Adults

These guidelines will be published in three volumes of The Lancet and are available on the PPMD website. Also, with a growing community of adults living with Duchenne, every subspecialty area of the updated Care Considerations includes recommendations for care from diagnosis through adulthood.

A Cornerstone of Our Mission

Making sure that people with Duchenne are receiving the most current and updated comprehensive care is of paramount importance to PPMD. The Certified Duchenne Care Center Program has these recommendations at its core, and works diligently with the Certified Duchenne Care Centers (CDCCs) to make sure that this level of care is provided by all clinicians at each certified center.

PPMD realizes that reading three volumes of The Lancet Neurology may be difficult and time consuming. In an effort to make sure that every family has access to what is included in the Care Considerations, we have initiated an effort with World Duchenne Organization (UPPMD), TREAT-NMD, and MDA to create a Family Guide, a family-friendly version of the necessary components of care.  The Family Guide is in development and will be released this year.

PPMD also worked with World Duchenne Organization (UPPMD) and TREAT-NMD to update the Imperatives for DUCHENNE MD, a one-page snapshot of the necessary elements of care. The Imperatives serve as a guide for families and patients, as well as clinicians who may not have an in-depth knowledge of Duchenne care. This document is now available on the TREAT-NMD website in more than 20 languages. The updated version will be available in translations soon.

Connecting Care Experts & Clinicians

This week, PPMD is hosting our first Duchenne Clinician Conference, a two-day Duchenne specific meeting covering all areas of care, including the newly updated Care Considerations. With more than 100 attendees (including clinicians, researchers, and industry), we expect everyone to leave the meeting having a wealth of new information and a network of expertise from which to draw when needed.

There are many ongoing efforts to ensure that you and your family are receiving the very best in Duchenne care and services. We will continue to include resources and information so that what we are providing is based on the publication of the most current recommendations for care, services, and support.

Care is more than just a pillar of our mission. Since our formation 24 years ago, we have made the care of our loved ones living with Duchenne a top priority. Today’s publication is another important moment in our community and PPMD is extremely proud to be a part of this effort.

Join Our Mailing List