A new SMA treatment program will start on September 1

On September 1, 2022, a new unified program for the treatment of spinal muscular atrophy (SMA) will enter into force. This program includes three treatment options: the previously used drug Spinraza and the newly introduced drugs: Zolgensma (gene therapy) and Evrysdi (oral therapy).

Since April 2021, SMA has been included in the Newborn Screening Program, which allows for the most effective treatment of this progressive disease in the period before its symptoms appear. Thus, Poland is among 4 countries in the world with such a comprehensive approach to diagnostics (screening tests) and therapy. In addition to the Spinrazy used so far, the list includes the most modern drugs for the treatment of SMA: Zolgensma (onasemnogene abevarvovec - gene therapy) and Evrysdi (risdiplam - oral therapy). All three drugs will be available in Poland under one integrated drug program that will cover the entire population of people with spinal muscular atrophy.

At the same time, we would like to deny the false information circulating in the media that children over 6 months of age or younger, previously treated with nusinersen, were left untreated. We declare that all children diagnosed with spinal muscular atrophy are and will continue to be treated with nusinersen. So there is no reason to say that anyone left their children untreated.

The criteria for including patients in the drug program have been widely discussed by experts in pediatric neurologists and neurologists, and are based on scientific data drawn from available studies and literature on the subject. The program has been designed so that all patients receive the care they deserve and are not left untreated.

We hope that the media will hear the fact that Poland has introduced a comprehensive SMA treatment program with three therapies for all those in need, and not the information that the Ministry of Health does not reimburse Zolgensma for a dozen or so children (treated with Spinraza). The reimbursement of three drugs and an active newborn screening program puts Poland at the forefront of European countries in terms of diagnostics and access to SMA therapy.

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