Baking Up An Angel Food Cake!
Recently we posted that a recommendation was being made that would severely limit who received access to the miracle drug, Spinraza. Provincial government officials will be reviewing these recommendations and then making the decision about this new drug. Right now, we believe that it will be beneficial to write to your local Member of Provincial Parliament, your Provincial Minister of Health, and your Provincial Deputy Minister of Health and express your concern. To assist you, we have drafted a letter that you may want to use - click on the province in which you live. These are in rich text format, which will hopefully work easily with the word processing program of your choice. One is drafted for those living in British Columbia (click here to download) and another is for those living in Ontario (click here to download) - we've used these two provinces as that is where the majority of Emmett's family lives. If you live in another province, we encourage you to write to your provincial leaders. If you need help adapting this letter, please let us know.
What does this young baker have to do with needing a drug called Spinraza? Absolutely everything. Without Spinraza Emmett could not attempt to stir the batter. Without Spinraza he would not be able to even hold something as heavy as this wooden spoon. Without Spinraza Emmett could not hold his head up. Without Spinraza Emmett would be so limited in what he could do. Please take the time to copy one of these letters, fill in your information, and mail or email it one or hopefully all three of the listed names for your province. All Canadian's with Spinal Muscular Atrophy need to have access to this drug. Our Government needs to talk to Biogen, the company that makes Spinraza, and get the cost of this drug down to a more reasonable level. Patients cannot get pricing changes but the government has the ability and purchasing power to make that happen.
Thank you for your help and support.
Don't want to click and download? Here's the text of the letter that you can copy and paste.
Below is a sample letter that may help you develop yours. Items underlined can be changed for the specifics of your letter. At this point we feel the most valuable contacts would be your MLA (link below for BC MLAs), Adrian Dix (BC Minister of Health), and Doug Hughes (BC Deputy Minister). If you don’t live in BC, we would appreciate you taking the time to send to your provincial representatives. Approval of Spinraza is something that will be a challenge all across Canada. Thanks you so much for your support of our family, and all other families affected by SMA!
Alisha, Josh, and Emmett
Date ??, 2018
Subject: Approval of Spinraza (Nusinersen) treatment for all patients with Spinal Muscular Atrophy
Dear Honorable [MLA]:
My name is _______________ and I have resided in the _______________ area for over ______ years. I am a (your job). My ___(friend, family)________, Emmett Willms was diagnosed with Spinal Muscular Atrophy, Type 1 when he was five months old. SMA is a genetic, life-threatening and seriously debilitating neuromuscular condition for which there is no cure. In December 2016, the US FDA approved a medication called Spinraza and Health Canada followed suit in July 2017 with their approval. This news brought hope to the SMA communities worldwide as this treatment was proving transformative, especially for those starting young. Emmett began a clinical trial just over two years ago (for Spinraza). Patients in the trial achieved milestones such as the ability to move arms, legs, and sit unassisted when they would otherwise be unexpected to do so. They reached these milestones when specialists expected that they would be forever lost, and lifespans are now, rather than terrifyingly short, happily extended. The overall findings of the controlled double blind trials support the effectiveness of Spinraza across the range of SMA patients, and appear to support the early initiation of the treatment.
For the first seven months of Emmett’s life (before starting on Spinraza), he lost the ability to move his legs or any significant arm movement. He lost the ability to swallow. He could not sit, hold his head up, or even sit in a car seat. His physicians doubted that we would ever hear him talk. They prepared us for the fact that he would not survive. We feared for his future.
In the past two years since beginning the Spinraza Trial, Emmett has gained strength in so many ways. He has gained very significant core strength and head control. He is gaining strength in his arms and movement is returning to his legs. He can sit in a car seat and, with lower back support, can sit and hold his own head up. He is incredibly bright and eager – chattering like many other two and three year olds. Emmett has had a power wheelchair since he was 16 months old – he enjoys the freedom that this mobility offers him.
Without Spinraza, Emmett would never have been able to do these things and, as SMA is a degenerative and terminal disease, his lifespan was drastically shortened. With Spinraza, Emmett is literally so much more physically involved in everything that is around him. Raising a child with SMA is taxing and emotionally and physically draining on caregivers. Spinraza will ease the burden on families because patients will be stronger and more capable.
Spinraza (manufactured by Biogen) will soon be under review at the provincial level and I hereby request that you support the decision to approve this treatment for all those suffering from Spinal Muscular Atrophy. It is a rare condition that affects 1 in 6,000 to 1 in 10,000 patients, so there are a small number of patients living in British Columbia with the condition. All of them deserve a better quality of life and access to treatment.
Other countries such as Sweden, France, Israel, and the US have treatment available for all types of SMA regardless of age, the number of backup genes and other symptoms.
Below is a link to a blog that documents Emmett’s improvements while on the clinical trial.
If you are unable to use the link above, if you search “Emmett’s Journey SMA” on Google or in YouTube, you will find links to Emmett’s Journey.
Thank you in advance for your attention to this very important and urgent matter. I look forward to hearing from you with regard to this request. Please feel free to contact me at the address and phone number above.