Evaluation of the ‘SMA Science Board’ from the Turkish SMA Foundation

The SMA Scientific Committee meeting organized by the Ministry of Health was held yesterday. SMA patients and their families in Turkey are eagerly waiting for,

The SMA Science Council meeting organized by the Ministry of Health was held yesterday. Expectations about whether the two drugs Risdiplam and Zolgensma, which are two drugs used in the world in the treatment of the disease, will be met by the state, which SMA patients and their families in Turkey are waiting for, have been met with the licensing of the drug that is suitable for oral use. It was stated that the evaluation of the Scientific Committee on Zolgensma, the other third drug used in the treatment of SMA, continues.

Before the meeting, Bilim Sıhhat News Agency (BSHA) had received statements from the Science Board regarding the expectations of SMA patients and their families, as well as foundations and associations. SMA Foundation of Turkey made an assessment to BSHA regarding the decisions taken after the meeting. Ece Soyer Demir, Deputy Leader of the Board of Directors of the Foundation, brought back the demands that all treatment methods related to SMA disease be in Turkey and that patients benefit from treatment in an equal and fair manner, free of charge.

“It is pleasing that the Survival Rate is 100% in Early Treatment”

Deputy Chairman Ece Soyer Demir said that the survival rate of 100% in the first six months is promising with the early initiation of treatment in those diagnosed as a result of SMA scans. Demir continued his statements as follows: “SMA Science Council; There was a report yesterday about SMA screening results, treatment processes and developments in treatment. In the statement made after the meeting, newborn and carrier screening test results and evaluations of Spinraza, Risdiplam, Zolgensma treatments were shared. With the newborn screening started in May 2022 in our country, 753 thousand 350 babies were screened for SMA and the early treatment of those diagnosed, the survival rate of 100 percent in the first six months was a pleasing data for us. Premarital SMA screening is also a valuable factor in reducing the long-term incidence of SMA, which is a preventable disease. Within the scope of this program, which started in 2021, it is possible for married couples to be screened free of charge.”

“All Treatments Should Be in Turkey”

As Turkey SMA Foundation; Demir stated that all treatment formulas should be available in our country and patients should benefit from these treatments equally and fairly, free of charge. “Taking SMA drugs into the SGK reimbursement system; We expect adjustments to be made on medical equipment, medical materials, improving physical therapy rights, providing a basis for electricity bills for patients connected to breathing apparatus, and pension rights for mothers who care for the disabled at home. In terms of treatment techniques, it is a promising development that a second drug (Risdiplam) is available to our patients. In the statement, it was informed that 1024 patients in our country are currently benefiting from Spinraza treatment and that the licensing phase of Risdiplam effective drug, which is used orally as a second treatment technique, has been reached. However, at this stage, there is no clarity about which type of SMA patients and which age groups will benefit.

“We are waiting for the SUT Communiqué on the Use of the New Drug”

Stating that there are four types of SMA, Deputy Leader Ece Soyer Demir pointed out that there are uncertainties about the new drug, whether all types will benefit from the drug, whether there will be an age criterion, and many other topics need to be clarified.

“All types are cured Will it be faulty, will there be an age criterion, what will be the transition process in the middle of the treatments, will the patients who have received Spinraza treatment so far be able to benefit from the Risdiplam treatment or will they be able to return to the Spinraza treatment if they want to change the treatment again? Stating that the answers to questions such as It will be clarified with the publication of the Health Implementation Communiqué in the coming days. T As all patients/patient relatives, we are waiting with interest. An individual evaluation should be made for each patient with the approach of “There is a patient, not a disease”. In this case, physicians may not see the treatment technique that they consider appropriate for one patient for another patient. Therefore, our wish is that all treatment methods are covered by payment.” (BILIM SAHHAT NEWS AGENCY)