Air travel is essential to being able to live, work or go to school where you choose—and can determine whether someone can see a specialty provider or participate in a clinical trial that is far from home. MDA believes accessto air travel is essential to living unlimited. That’s why we’re working with policy makers, federal agencies, industry, and other advocacy and disability rights organizations to help improve access to air travel for MDA families.
Spending time in our nation’s capital with dozens of other MDA families gave me a new sense of empowerment and taught me to apply a skill I’ve already honed in my personal life to the public policy arena. It was inspiring and exciting to see so many people come together in one place to do our part in making this country a better place for people who live with muscle-debilitating diseases.
A delegation of dozens of MDA advocates from 23 states visited more than 100 congressional offices on Capitol Hill earlier this week to speak to their representatives about the importance of programs and policies that help accelerate the development of treatments and cures for individuals and families living with muscular dystrophy, ALS and related neuromuscular diseases.
In advance of MDA’s first ever Advocacy Conference, taking place April 23-25, we asked several advocates to unpack the idea of advocacy: what is it, why they became advocates and why it is necessary for others to follow their examples. Their responses follow.
Being chosen to speak in front of the FDA and represent the SMA community makes me feel incredibly honored. Today’s SMA Patient-Focused Drug Development (PFDD) meeting with the FDA means we have a voice as patients living with rare muscle diseases. The PFDD meeting was set up so that patients with SMA and the parents of children with SMA could stand before the FDA and voice their concerns, discuss their experiences with research and address the need for continued research that evolves to include more patients. This is my story.
As policy makers are considering various options to modify or replace the Affordable Care Act (ACA), MDA is working to ensure that individuals and families affected by chronic, serious and life-threatening diseases maintain health care access and coverage.
Many families whose children have spinal muscular atrophy (SMA) endure a long and difficult diagnostic odyssey. This may soon change, however, based on recommendations MDA recently made with a coalition of partners that SMA should be added to the list of conditions newborns are screened for at birth in the United States.