Raising the Voice of the SMA Community

MDA advocacy efforts are focused on accelerating research advances and treatment discovery while expanding the resources available to those affected with the disease

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by Amy Madsen on August 29, 2013 - 10:37am

Today, research discoveries are bringing us closer than ever to effective therapies for spinal muscular atrophy (SMA) and other neuromuscular diseases. Medical knowledge of neuromuscular disease is broadening, and children and adults with these diseases are living longer, fuller lives.

But significant challenges remain.

Many federal agencies are not receiving the funds and resources needed to catalyze change. And people with physical disabilities due to neuromuscular disease continue to encounter barriers to accessing necessary resources and supports.

In addition to being a strong force in SMA therapy development, MDA is a dedicated advocate for public policy to secure resources that can help people with SMA live vibrant, purpose-driven lives.

Advocacy is an undercurrent in all of MDA's programs and services. MDA's national advocacy office formally projects the voice of the MDA community and protects the interests of the individuals and families that MDA serves.

Here’s an overview of current MDA advocacy efforts of particular interest to the SMA community.

Orphan Drug Tax Credit

At the forefront of MDA's current advocacy efforts is a push to maintain the Orphan Drug Tax Credit (ODTC) as Congressional tax reform proposals are developed.

The tax credit, part of the 1983 Orphan Drug Act, provides industry with a financial incentive to leverage the long-term investments MDA and other advocacy groups have made in science, manpower, infrastructure and therapy development. It allows companies to estimate an adequate return on investment for the otherwise risky business of developing drugs for very small potential markets.

Through the tax credit incentive, and the promise of seven years of market exclusivity, the Orphan Drug Act hastens the process of bringing vital treatments to people who desperately need them.

MDA has been told by many industry partners that the ODTC is among the most effective incentives ever offered in terms of attracting companies to invest their finite research and development resources in rare and orphan diseases like SMA.


The MDA community’s advocacy efforts are building momentum as new co-sponsors sign on in support of the Achieving a Better Life Experience (ABLE) Act of 2013 (H.R. 647 and S. 313).

This legislation amends section 529 of the Internal Revenue Code of 1986 to provide for the establishment of “ABLE accounts” for the care of family members with disabilities.

Similar to college savings accounts, ABLE accounts encourage and assist individuals and families in saving private funds that can be used to support the health, independence, education and quality of life of people living with disabilities.

The goal of the Able Act is to provide secure funding for disability-related expenses on behalf of designated beneficiaries with disabilities. These funds supplement — but do not supplant — benefits provided through private insurances, Medicaid, Supplemental Security Income (SSI), the beneficiary's employer and other resources.

Transitions policy

MDA is partnering with federal agencies and other groups serving youth with disabilities to break down barriers for young people transitioning from childhood to adulthood.

Barriers identified by MDA’s transitional services program range from finding accessible transportation and housing, to funding personal care attendant services, to locating knowledgeable physicians who treat adults with pediatric-onset diseases. Others include employment and Medicaid benefit eligibility, pain and associated depression, mental health issues and health care self management.

Fragmented and complex federal and state systems of support also present barriers to much-needed resources.

As longevity increases, transitions issues will continue to be highly relevant for people with SMA. Efforts to address these issues are being led, informed and guided by MDA's SMA community.

For more on MDA's transitions efforts for youth and young adults with neuromuscular diseases — and their families — visit the MDA Transitions Center; there you’ll find resources on careers, education, independent living and more.

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