Just days before Christmas 2016, the FDA gave a present to some of the children in the United States—they approved the first treatment ever to treat spinal muscular atrophy (SMA).
However, the announcement was met with more than just joy. In fact, when a new treatment is approved for a disease, those with the disease and their loved ones experience a rush of conflicting emotions. The parents of Madison Smith, a girl living with SMA, took to their website to share what they experienced when the announcement was made.
Spinal muscular atrophy is a rare, degenerative disease that causes slow weakening of muscles in children as they grow. The neurons in the body are not communicating with the muscles, causing them to waste away. Frequently, children with SMA will need wheelchairs. In severe cases, they are unable to sit up on their own or swallow foods. To learn more about it, click here.
Of course, Aaron and Jen Smith were incredibly grateful that there was finally a treatment that may slow or stop the progression of Madison’s disease. However, they also professed to feeling stress, uncertainty, and heartache.
Parents will sacrifice just about anything for the health and happiness of their children, but that doesn’t prevent from feeling the stress that may come along with it. The cost of medical care for a disease like SMA can easily run into the thousands per month. Adding the cost of the newly approved treatment of course adds to that financial strain.
Additionally, just because a drug is approved by the FDA does not guarantee that it will work for everyone. Every parent and every child with SMA is hoping that the new drug will work for them, but that’s just not possible.
It’s hard not to let your hopes get up when thinking about the new drug’s possible effects on Madison and her family’s lives. Even if it works for Madison, it is not a cure. Madison’s SMA may slow or even stop progressing, but she will still have to manage it and cope as she grows. But the small hopes with drug approvals like Spinraza, like hoping it restores her ability to walk, are what keep some of us in the rare disease world going.