Halting the Progression is Enough! Spinal Muscular Atrophy (SMA)

Halting the Progression is Enough! Spinal Muscular Atrophy (SMA)

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Tyler P started this petition to Health Insurance & Medicaid

Halting the Progression is Enough! Spinal Muscular Atrophy (SMA)

The goal of this petition is to raise awareness and change the absurd criteria for the continuation of a proven medical treatment. Spinraza stops the progression of an otherwise lethal disease. Health Insurance & Statewide Health plans are requiring unreasonable criteria from patients with SMA, denying a life saving treatment. 

SMA, the number one genetic killer of babies under two years of age, is a progressive, childhood, neuromuscular disease caused by a mutation in a single gene. In December 2016, Spinraza became the first approved drug used in treating this disorder. Spinraza contains an antisense oligonucleotide — a molecule that works by increasing the body’s ability to produce a protein called survival motor neuron (SMN), which is essential for motor neuron health. SMA patients don’t produce enough SMN.

All people have two genes — SMN1 and SMN2 — that encode for the SMN protein. Most of this protein is made from SMN1, though SMA patients have a mutation in the SMN1 gene which renders their bodies unable to produce enough SMN protein. The SMN2 gene can also make SMN protein, but due to a difference in the coding sequence of this gene, most of the protein that is made from this gene is shorter and gets destroyed by the cells.

Spinraza increases the ability of the SMN2 gene to produce full-length SMN protein. Spinraza has been proven to completely halt the progression of an otherwise, devastatingly rapid deterioration in muscle.

During the clinical trials of Spinraza motor milestone tests were used on infants to detect the effectiveness of the drug. This worked to partially demonstrate the drug’s effectiveness as any and all stabilization, or motor improvement was contradictory to the disease itself. However, after the FDA approved the drug, insurance companies and statewide health plans adopted these tests to determine the continuation of the drug in patients.

These motor milestone tests are subjective and should not be a factor in determining someone’s right to life. Depending on the doctor, how a patient is feeling on that day or even the unwillingness of an infant to partake in the test can play a factor in hindering scoring improvement. For these reasons, someone with SMA can be denied a proven medical treatment.

Further, many adults with SMA have contractures due to living with the disease for years. Contractures will hinder your scoring on motor milestone tests. What’s more, although Spinraza can stop the disease from worsening it cannot restore the motor neurons which have already died in adult patients. Asking them to sit to stand for a test score is impossible. But should that mean they do not have the right to access a drug that will prevent their disease from worsening?

This is comparable to telling someone with diabetes “We have a successful medication but because the illness has already taken your leg, you cannot wiggle your toes to score perfectly on our test. Therefore, we will no longer continue treatment.”

This is unfair. Halting of the disease and stabilization should be enough to meet criteria. Any improvement is a blessing, but it should not determine continuation of a drug which has been proven to halt a fatal disease. I’m calling upon the SMA community and all of those with compassion to sign, share, and promote this petition. Please bring this to the attention of state officials to bring change and save lives.

Halting progression of a fatal disease is enough to continue Spinraza treatment!

0 have signed. Let’s get to 2,500!
At 2,500 signatures, this petition is more likely to get picked up by local news!