What would you do if you had two children and one of them was getting better on a drug and you just needed this drug for your other son?
Earlier this year, Jenn McNary asked this question, describing her life-long fight for two of her sons who have Duchenne Syndrome, a common form of childhood muscular dystrophy.
Yesterday, the family experienced a very special victory when the drug was finally approved by the FDA after years of families and communities fighting for it’s availability!
For Jenn, it all started when the promising treatment was discovered, but only one of her sons was eligible to receive it in trial since there wasn’t enough evidence for the drug to be approved by the FDA. Due to the rarity of the disease, this process could take years to wait out—giving no hope to those patients who potentially only had months left.
Jenn and other families in the rare disease community understood this very clearly: without the freedom to experiment, innovate, and make personal health decisions, those with a low prevalence disease might never have a treatment. Hence, these hopeful communities made it their mission to get this personalized drug approved for their loved ones, and the thousands of others who could also benefit from the drug’s availability.
After years of petitioning the government and raising their voices with doctors and Duchenne researchers alike, these families received the news they’ve been waiting for. The drug was approved!
Not only does this story prove that communities know how to solve their problems best, but it also speaks of the importance of freedom in health. In the end, individuals know what’s best for themselves and should be in charge of their own health. And while everyone’s needs are unique and different, freedom in health is the only way to ensure patients are getting the personalized care they need—whether they use traditional or integrative medicine, or try a new and risky treatment.