SMA Disease and Treatment Options
Spinal Muscular Atrophy (SMA), is a rare and genetic disease. This disease causes the loss of motor neurons, leading to weakness in muscle strength and progressive muscle wasting. SMA usually occurs in childhood and can lead to serious health problems if left untreated. Therefore, the drugs and treatment methods used in the treatment of the disease are of great importance.
SMA Treatment Drugs in Turkey
In Turkey, there are two main medications for SMA disease: If you are nusiner ve Risdiplam. These drugs play an important role in the treatment of the disease. Nusinersen was added to the Foreign Drug List in 2017 and was licensed and reimbursed in 2022. Risdiplam was added to the list in 2020 and is expected to be licensed in 2024.
Nusinersen Drug Properties
If you are nusiner, stands out as the first treatment option developed against SMA disease. This drug aims to protect the health of motor neurons by supporting the function of the SMN1 gene, which causes spinal muscular atrophy. Clinical studies have shown that Nusinersen increases the quality of life of patients and improves muscle strength. Therefore, it is a very critical treatment option for SMA patients.
Risdiplam Drug Properties
Risdiplam, is an oral medication for the treatment of SMA. This medication aims to stop the progression of the disease by increasing the production of SMN protein. The advantage of Risdiplam is that it allows patients to manage their treatment process more easily. Clinical data supports the effectiveness and safety of Risdiplam.
Future Treatment Options
In the future, the development of new treatments for SMA is a great hope. Research shows that innovative approaches such as gene therapy may play an important role in the treatment of SMA. However, more clinical data is needed to prove the effectiveness of such treatments.
Approaches to SMA Disease
The Ministry of Health meticulously monitors the treatment processes for SMA disease and makes decisions based on scientific data. SMA Scientific Committee, continually evaluates the effectiveness of existing treatment methods and examines new treatment options.
Accessibility of Medicines
Turkey is an important social state worldwide with the pharmaceutical and medical device services it provides in the field of health. In this context, the accessibility of the necessary drugs for SMA disease is of great importance. The Ministry of Health is taking the necessary steps to ensure that patients have access to these drugs.
Conclusion and Evaluation
SMA disease poses a great threat to children as a genetic disorder. However, with drugs such as Nusinersen and Risdiplam available in Turkey, the treatment process of the disease has become more effective. In the future, it is expected that new horizons will be opened in the treatment of SMA with further research and development. This will increase the quality of life of patients and will be an important step in the fight against SMA.
