The day after Christmas in 2016 brought a post-Holiday surprise to many in the U.S. with spinal muscular atrophy. December 26, 2016 marked the day that Spinraza was approved by the FDA. Read the original article here.
Spinraza is the first therapy for spinal muscular atrophy (SMA), so this was no small feat. Spinal muscular atrophy is a disease that is caused by a mutation in the survival neuron gene 1 (SMN 1). This disease takes away a person’s physical strength by affecting their motor nerve cells in the spinal cord. This eventually causes the patient to lose the ability to walk, eat, or even breathe. Spinal muscular atrophy is the leading cause of genetic death in infants.
Unfortunately, at the time of this article, less than 300 people had received the drug since its approval.
In Michigan alone, there are over 350 families suffering with SMA, but only one fortunate girl in the entire state has been administered the drug. Four-year-old Scarlet, became the lucky recipient on January 12, 2017.
While many large hospitals were not prepared to deliver the medicine, it was a small muscular dystrophy clinic in Farmington Hills that offered to give Scarlet the drug.
This begs the question, why were hospitals having so much trouble beginning the process of administering this new drug?
There are several reasons:
There has not been good communication in the hospitals about the new Spinraza drug and there seems to be a very secretive air about it.
Some hospitals and clinics sent letters out to families declaring that only certain kids were applicable for treatment with the drug or that families would simply have to put their names on a waiting list.
Another factor that is slowing down the process of getting this medicine out more quickly is because there is no clear process for administration. Because this is the first therapy for SMA that hospitals have seen, there is not a set up for an approved therapy in hospitals.
Also, big hospitals were caught unprepared when the FDA approved Spinraza several months sooner than everyone thought.
What can those with SMA look forward to?
There are over 15,000 people worldwide with SMA. Many are patiently awaiting the day when they will be allowed to take this potentially lifesaving medicine.
Kenneth Hobby, president of CurseSMA.org is excited about the new drug. He understands that this new drug will be a change in the SMA community, who can go from just coping to the disease to having hope. The SMA community now has something practical to look forward to.
For every child with SMA, each day without treatment means more muscle loss and the reality that their disease is progressing even more.
