PPMD Urges HHS Secretary to Update Organ Transplantation Policies to Ensure Pediatric Patients Have Access

Jun 21, 2013, 11:10 ET from Parent Project Muscular Dystrophy

HACKENSACK, N.J., June 21, 2013 /PRNewswire-USNewswire/ -- Parent Project Muscular Dystrophy (PPMD) – the nation's leading voice for patients and families impacted by Duchenne muscular dystrophy (Duchenne) – is urging the Secretary of Health and Human Services to update the nation's organ transplantation polices to ensure pediatric patients with rare diseases are not negatively impacted by outdated regulations.

In the letter, PPMD urged HHS Secretary Kathleen Sebelius to initiate a dialogue around the equitable distribution of available organs to individuals for whom they are appropriate, an issue of importance to patients and families battling Duchenne and who may need transplant organs one day. The issue has made national headlines in recent days when a 12-year-old girl with Cystic Fibrosis (CF) was originally denied because of a policy that keeps children under the age of 12 from having access to adult organs.

In the letter to Secretary Sebelius, PPMD Founding President and CEO Pat Furlong wrote: "Pediatric and adolescent hearts available for transplant are scarce, and life post-transplant is not a panacea. We acknowledge that there must be appropriate use of this precious resource for appropriate patients, with clear and full patient and parent understanding of the risks and benefits entailed with cardiac transplant."

Over the past ten years, several patients with Duchenne have undergone successful cardiac transplants, are doing quite well medically, and are living with an excellent quality of life.

In the letter, Furlong referenced the tragic case of 11-year-old Mitchell Jones, a Duchenne patient with severe cardiomyopathy but with no significant limitations in respiratory or musculoskeletal strength and who was ineligible for a heart transplant because of his underlying diagnosis of Duchenne.

"Mitchell's case makes it abundantly clear that when it comes to organ transplantation and policies, traditional contraindications should not be used as absolute contradiction to transplant. With more patients with Duchenne, CF, and other diseases likely to be in need of transplantations in the years to come, the time is now to have this conversation and to update the laws and policies, so a child's diagnosis or age cannot alone decide his or her fate," Furlong said.

The letter is the latest in a string of actions by PPMD to address urgent issues unique to the rare disease community. For example, last year PPMD championed a number of provisions to accelerate the development of therapies for rare diseases that were incorporated into the FDA Safety & Innovation Act (FDASIA). Accelerated approval has been a top priority of PPMD's comprehensive advocacy agenda and the organization has had multiple meetings with agency leaders on this and related issues.

To learn more about Duchenne and Parent Project Muscular Dystrophy's advocacy agenda, visit ParentProjectMD.org.

About Parent Project Muscular Dystrophy
Duchenne is a fatal genetic disorder that slowly robs young men of their muscle strength. Parent Project Muscular Dystrophy (PPMD) is the largest most comprehensive nonprofit organization in the United States focused on finding a cure for Duchenne muscular dystrophy—our mission is to end Duchenne.

We invest deeply in treatments for this generation of young men affected by Duchenne and in research that will benefit future generations. We advocate in Washington, DC, and have secured hundreds of millions of dollars in funding. We demand optimal care, and we strengthen, unite, and educate the global Duchenne community.

Everything we do—and everything we have done since our founding in 1994—helps boys with Duchenne live longer, stronger lives. We will not rest until every young man has a treatment to end Duchenne. Go to www.ParentProjectMD.org for more information or to learn how you can support our efforts and help families affected by Duchenne.

SOURCE Parent Project Muscular Dystrophy