Poland today stands among Europe’s leaders in the treatment of spinal muscular atrophy (SMA). Since 2019, all patients — regardless of age or disease progression — have had access to the world’s first approved SMA therapy, nusinersen. Clinical observations over these years show that treatment has successfully halted disease progression in all patients, while over 70% have experienced clinically significant improvement. Physicians emphasize that the Polish model of SMA care is now one of the most comprehensive in Europe.
The B.102 program, launched in 2019 by the National Health Fund (NFZ), represents a systemic model of SMA treatment in Poland. It provides full reimbursement, continuous monitoring of outcomes, and centralized collection of clinical data. The program now includes more than 1,150 patients, with some having been observed for over seven years, making it one of the longest-running SMA clinical initiatives in Europe.
“Our drug program gives patients access to all three innovative therapies registered in Europe. Since its launch in 2019, we’ve been able to treat patients across the full age spectrum, regardless of disease severity. This allowed us to publish long-term follow-up data for adults and older children treated with nusinersen — results that frankly exceeded our expectations,” said Professor Anna Kostera-Pruszczyk, Chair of the Scientific Council of the SMA Foundation and Head of the Department of Neurology at the Medical University of Warsaw, in an interview with Newseria.
By mid-2025, over 900 patients had begun nusinersen therapy under the B.102 program — making it one of the largest SMA treatment programs in Europe. Observational data show that disease progression has been halted in all patients, while 72% experienced functional improvement deemed clinically significant. Among children with type 1 SMA, average CHOP-INTEND scores improved by 5 points after 3 months and 15 points after 19 months. For type 2 and type 3 SMA patients, improvements reached 8 points — twice the threshold considered clinically meaningful. Even adult patients with type 1c SMA, who had never achieved independent sitting, demonstrated functional stability or improvement.
Real-world clinical data confirm that treatment benefits increase with continued therapy — patients not only maintain mobility but also regain lost motor functions.
“At the start of the program, we considered stabilization a success — simply halting disease progression. But we’ve seen more: with every dose, even small, incremental gains accumulate. Over years of therapy, our patients haven’t just stopped losing abilities — they’re regaining them. This translates into better daily functioning, greater independence, and the ability to study, work, and participate in family life,” noted Prof. Kostera-Pruszczyk.
“It’s a real improvement in quality of life. It allows us to think about the future — to make life decisions, not survival decisions. For example, whether to study, take a job, or send children with SMA to kindergarten so they can enjoy social life with peers,” added Katarzyna Pedrycz, Vice President of the SMA Foundation.
Poland’s SMA Care Model as a European Benchmark
Physicians stress that Poland’s SMA treatment system has for years set an example for other European countries. While SMA Europe confirms that all three approved therapies are reimbursed in many nations, access remains limited elsewhere due to strict eligibility criteria — based on age, weight, SMA type, or respiratory condition. In some countries, coverage is partial, or treatment is offered only through individual patient programs.
“The Ministry of Health has been open to many of our requests — including the crucial introduction of newborn screening. Thanks to this, most children born with SMA in Poland receive treatment within the first month of life, which guarantees maximum therapeutic benefit. Another unique feature of the Polish system is the ability for women with SMA to continue nusinersen treatment during pregnancy,” said Prof. Kostera-Pruszczyk.
Through systematic monitoring, Poland now holds one of the world’s largest real-world SMA data sets, offering invaluable insights into long-term outcomes. Experts in medicine and health economics note that the program has dramatically improved patient prognosis and quality of life.
“We should make broad use of such data, especially given the scale of public spending. The SMA drug program costs over half a billion zlotys, but the therapies have proven to be highly effective and safe. We should continue to analyze therapeutic value, patient numbers, and spending patterns — this is an outstanding example of success in modern drug policy,” emphasized Prof. Marcin Czech, President of the Polish Society of Pharmacoeconomics and Head of Health Economics at the Mother and Child Institute in Warsaw.
Growing Population of Adult SMA Patients
According to the SMA Foundation, the number of adult patients in treatment has doubled over the past few years — a natural consequence of improved survival and therapy effectiveness. Poland currently has 20 specialized centers treating adult SMA patients, though their workload continues to grow.
“The most challenging situation now involves adults. We urgently need more treatment centers for adult SMA patients and a smoother transition process for those turning 18 to ensure continuity of care,” said Prof. Kostera-Pruszczyk.
Patients and clinicians have welcomed the October 2025 program update, which allows temporary switching between therapies or returning to the initial drug (nusinersen) if needed.
“I’m very pleased that the program is evolving. It’s a positive, pragmatic decision giving clinicians flexibility to tailor treatment to patients’ needs. If a therapy proves less effective, we can return to a previous drug,” said Prof. Marcin Czech.
“Previously, patients feared they wouldn’t be allowed to resume a treatment once changed. Now we can decide together,” added Katarzyna Pedrycz.
Looking Ahead: Higher Doses and New Delivery Methods
At the start of 2026, both the medical and patient communities are awaiting the registration of a higher-dose nusinersen formulation and results from clinical trials of a subcutaneous delivery port, designed to simplify long-term administration.
“Expectations for the higher dose are enormous. We hope it will be more potent, eliminate the wearing-off effect, and help us maintain even better function over time,” said the Vice President of the SMA Foundation.
