Real-life stories
Meet Louise
Louise and fighting to expand access to SMA medicines in Denmark
Louise
Forty-year-old Louise lives in Denmark, sharing a small house in the city with her lap dog. For the past several years, she has devoted her energies to advocating for access to medicines and early diagnosis for people living with SMA in Denmark.
Louise received her first wheelchair when she was two years old. To Louise, living with disabilities has always been an integral part of her life, but is by no means the only part. She has lots of friends with common interests, likes travelling, shopping, and hanging out.
When disease-modifying medicines for SMA were approved in Europe, the patient advocacy community in Denmark took action to ensure that the government would grant access to medicine to people living with SMA in this country. The national organisation Muskelvindfonden had a prominent role, but independent individuals also took initiative. Some people wrote letters to newspapers, others appeared on TV or talked to politicians. Louise started using her social media platforms to inspire others and show what it is like to live with disabilities, and she wrote to politicians and media outlets to convince them of the importance of giving people living with SMA a chance to treatment through medicines.
The first group of people living with SMA who achieved access to medicines were the youngest children. Then, nationwide newborn screening for SMA was approved. Louise felt proud to have contributed to something meaningful. Despite all efforts, however, progress was slow. In 2023, individuals up to 25 years old were included, but older adults were still left out, with no chance to receive medicines.
“Some people took it as a victory, and it was. Yet, some of us felt left behind and forgotten. As an adult over 25 years old, it was like a slap in the face.”
Undeterred, the community continued advocating until a year later, in August of 2024, all age barriers were removed. To Louise, the key to this success laid in a combination of relentless awareness-raising and advocacy pressure until the government-appointed medical council and drug manufacturers reaching an agreement on pricing.
Access to SMA medicines in Denmark, however, is not yet universal. Several start and stop criteria can prevent individuals from receiving medicines. In Denmark, the combination of these criteria can prove strict. For instance, Denmark is one of the countries people who use permanent ventilation, defined in Denmark as the use of respiratory support for more than 16 hours a day for 21 consecutive days, are denied access. In addition, to start treatment in Denmark, everyone needs to pass assessment tests to show that they do not perform either too strongly or poorly to be eligible for access.
Having been involved in the advocacy, Louise was aware that performance criteria might be set in place. For over a year and a half, she practiced specific tasks intensely, just to pass her motor function assessment. Despite the targeted training, she barely made it. Having passed the test, one month ago, she finally started receiving medicines for SMA.
She received the news on the evening before her fortieth birthday, a perfect gift and, in her words, a surreal experience. Louise is thrilled now, but the process was frustrating. To pass the test, she had spent a lot of time training to accomplish activities that are not relevant to her in her daily life, like picking up a coin. To her, assessing how someone feels, whether they can still drive their wheelchair or use the bathroom, would be much more informative, though more subjective or difficult to capture.
Just because progress cannot necessarily be measured, it does not mean that a person does not feel it."
Louise also cannot help but worry about the future. What if she gets pneumonia and needs to turn to fulltime respiratory support? What if she does not to pass the tests again next year, when her motor function needs to be reassessed? What if she gets weaker, like everyone does when they get older? Still, for now, she is basking in her joy.
“I am very grateful and hopeful that it will work. Even if it could just slow down or stop disease progression, that would make the future a little less uncertain and I would feel more at ease.”
Having the opportunity to receive these medicines is a great responsibility, too. Louise realises that this is an enormous financial investment that is being expended on her. At the same time, she also feels that this is a flawed way of reasoning.
Being in a position where you are basically being told that your life is not worth spending so much money on, because it looks like you have a horrible life anyway, just hits you hard.
Start and stop criteria determine who will get treatment based on evaluations about who has enough need, and who will benefit sufficiently to justify receiving an expensive medicine. Inherently, these calculations translate to assumptions about the quality of life, and which lives are worth living, for people living with different degrees of disability. But, Louise asks, who gets to be the judge of what a meaningful life is? And based upon what experiences and criteria?
Lots of work still needs to be done to ensure that all people living with SMA in Denmark have access to SMA medicines. For this, Louise extends a call for action to all fellow people living with SMA:
Although we are extremely happy, we need to remember about those who are still left behind. We need to continue fighting for them. Even though it might take time and be frustrating, advocating helps! We won’t stop fighting until every person with SMA in Denmark has access to the treatment.”
To protect contributors’ privacy, the names used here are pseudonyms. Interview conducted on 5 November 2024.
Advocacy for access to SMA medicines is a continuing process to ensure no person living with SMA is left behind, now and in the future.
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