Access to Health Care and to Approved Therapies is Essential for MDA Families

Despite the progress in the quest for treatments and cures, individuals living with the life-threatening diseases that newly approved drugs treat are facing a new challenge — gaining access to the approved therapies. Today, some individuals and families are unable to access FDA approved therapies, which can significantly impact the health and life of the person denied access. Not having access to appropriate therapies is unacceptable. A treatment cannot change the lives of families and individuals if they do not have access to it.

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What is Advocacy and Why Should You Care?

health care reform bill

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.

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I’m an Adult. Talk to Me Like One.

I’m a 25-year-old college graduate. I’m an adult and I can prove it. I have bills, grown-up responsibilities and the obligation to do things I don’t want to do. Still, regardless of the date of birth listed on my Colorado State ID, some people still treat me like a kid. Unfortunately, a lot of the world finds it easy to infantilize people with disabilities.

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MDA Cohosts and Sends Representatives to SMA Patient Focused Drug Development Meeting with FDA

PFDD

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.

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