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During the meeting with SMA-DER Vice Chairman, Ece Soyer Demir and General Secretary Mehmet Demir, Social Security Institution President, İsmail Yılmaz and SSI General Health Insurance General Manager, Dr. Mustafa Özderyol on Friday, September 4, 2020 expressed the problems of the patients and their relatives, and asked questions about current problems.


1.For Type 1 patients who continue to use Spinraza, the usage principles for the 11th dose and after are not yet clear. Families wonder when the Health Implementation Notification will be announced and its content.

1. While determining the principles of use of Spinraza, our Scientific Committee stated that they did not have a very long-term prediction and they would make an evaluation and make a decision in line with the data from physicians. The Scientific Committee will announce as soon as possible, I don't think the criteria will be any more difficult than what is now.

  1. Which Health Practices Notification will be valid for Type 2-3 patients who continue loading doses in using of Spinraza, what is the reason for such a change?
    2. Such a change was made as a result of the decision of the Scientific Board. For patients using medication, the new notification will be valid, and their physicians will apply in accordance with the new notification.



  1. Health Minister Fahrettin Koca, in his statement on August 19, 2020, said, "I would like to say that a similar drug, which has been recently licensed and taken orally, has been developed and approved, and both SGK and us are involved.We also reach this drug." Therewith, we would like to learn the details such as when patients will start using the drug, when the Health Practice Statement will be announced, which patients will be covered, whether it can be used with Spinraza or not, and whether patients who have been discontinued Spinraza treatment will be covered or not.

  2. Our negotiations with the pharmaceutical company continue. It is currently not possible for us to give a date. But of course, this will also have criteria, there is no such thing as unconditional administration to patients. We do not know if patients who use Spinraza but stop taking the drug, can benefit from this drug. Oral use is an advantage comparing to Spinraza. For example, if this drug was used during the pandemic period, patients would be able to continue their treatments.


  1. After Zolgensma has received FDA and EMA approval, patients and their relatives are trying to collect medication costs through individual campaigns. As patient relatives, we understand this effort of families, but we think that individual campaigns are not solution-oriented. Campaigns organized in this way are not both inclusive and patients have to go abroad. We want all treatment modalities to be compensated for all patients. What is the approach of the Ministry of Health and SSI to Zolgensma?

  2. For Zolgensma, the firm demands a very high amount. We have about 1300 patients who applied for Spinraza, who discontinued the drug or continued. It constitutes 10% of the market in the world. The number of patients in countries that are about to be covered is much less than we do. In addition, scientific studies and clinical research results are not sufficient. We do not want to put our patients at risk with these clinical results. Our negotiations with the company continue.

Carrier Screening

  1. Carrier screening, as you know, became a current issue during the period of Health Minister Ahmet Demircan and Health Minister Fahrettin Koca. We care about future generations as much as the treatment and quality of life of our current patients. My son has been connected to devices for 4 years. As a mother, I think of what he would like to eat if he was a healthy child every time I put food in a baby food bag. I want no mother to think like me, no child born with SMA, or aspirated. What is the latest situation regarding performing carrier screening before marriage and pregnancy?
  2. We know that the Ministry of Health is very keen on pre-marital screening and is working on it. It is not possible for us to provide information as it is the domain of the Ministry of Health.

PGD (Preimplantation Genetic Diagnosis)

  1. Regulations have been made oriented that "The married couple who has a hereditary disease determined by the Institution or known to be a carrier for this disease to have a healthy child" with the amendments in the Article 29 of the Law respecting Amendment on Certain Health-Related Laws and Decree Laws, published on December 5, 2018. We repeated our request during the meeting of the Assembly Investigation Commission Established to Determine the Treatment and Care Methods Used in ALS, SMA, DMD, MS Diseases and Other Diseases with Unknown Treatment and the Problems and Solutions Experienced by People with These Diseases and Their Relatives. Head of General Health Insurance Drug Department, Dr. Dilek Yılmaz said that the diagnosis list was ready and preparations were made. What are the developments in this regard?

1. We are working with the Ministry of Health on this issue, we determine which diseases will be covered. SMA is definitely included in these diseases, we will be operational by the end of the year.