PPMD Wins Bipartisan Legislative Backing of Its Muscular Dystrophy Goals

While federal funding for fiscal year 2020 has not been finalized, 79 members of Congress have vowed to back Duchenne muscular dystrophy (DMD) research, patient support efforts and drug development.

On its website, Parent Project Muscular Dystrophy (PPMD) heralded results of its annual bipartisan campaign, expressing optimism that agencies that affect Duchenne will be sufficiently funded. President Donald Trump last month sent his $4.75 trillion budget proposal to Congress for the fiscal year beginning Oct. 1.

Over the past several months, the organization and advocates met 143 times with federal lawmakers and sent more than 2,000 emails. It said some 59 representatives and 20 senators signed on in “strong support” of PPMD’s initiatives.

“We are pleased to report a very successful outcome for the Duchenne Fiscal Year 2020 funding letters signatures,” the organization stated.

Since the 2001 enactment of the Muscular Dystrophy Community Assistance, Research and Education Act, federal commitments to research DMD and other forms of muscular dystrophy have expanded. Such commitments have also resulted in more public-private investigations.

However, the act only provides the legislative framework for federal focus and investment, not the funding. Because of that, the PPMD spearheads an annual push not only for continued disease funding, but for the inclusion of Duchenne and muscular dystrophy-specific language in the report that accompanies the budget. The report includes specific priorities and guidance regarding agency funds appropriated.

Specifically, the PPMD requested funding for the U.S. Department of Health and Human Services (HHS) agencies including the Centers for Disease Control and Prevention (CDC), the National Institutes of Health (NIH), the Centers for Medicare & Medicaid Services (CMS), the Food and Drug Administration (FDA) and the Social Security Administration (SSA).

Lawmakers were asked to sign a letter seeking language calling for continued funding of $6 million for the CDC’s Muscular Dystrophy Research and Tracking program; expanded NIH support of Duchenne research, particularly challenges with gene therapy and novel clinical trial designs, and supporting continued refinement of FDA policy making available how patient experience data is used in the agency’s new-product reviews.

They also were asked to urge the SSA to make available data on the rate at which Duchenne and Becker patients use SSA programs, and to have the CMS issue a report on its newly established International Classification of Disease (ICD-10) code, which pertains to claims billing and processing.

The nonprofit PPMD works to improve patient care and ultimately find a cure. Since 1994, it has invested more than $50 million in research into Duchenne. Visit the website for more information on the organization’s mission and impact.

Duchenne and Becker, the most common forms of muscular dystrophy in children, affect roughly one in every 5,600 to 7,700 males in the United States.