There are not enough clinical trials of gene therapy in SMA disease
It was stated that there are not enough clinical trials related to the efficacy and long-term side effects of the drug used in the gene therapy, which has come to the fore in Spinal Muscular Atrophy (SMA), which is a deadly disease, and it is stated that time is needed to speak precisely about its effectiveness and reliability. >
Üsküdar University Faculty of Medicine, Head of Internal Medicine Department and Pharmacology Specialist Prof. Dr. Tayfun Uzbay told Anadolu Agency (AA) that SMA is a genetic muscle disease.
Stating that especially type 1 emerges from birth and resulted in death within two years, Uzbay emphasized that the types whose symptoms started later have a longer life span with appropriate care and some supportive treatments.
Uzbay, while one in 10 thousand of incidence in the world of the disease, pointing out that this rate of 6 thousand a in Turkey, in which voiced no contribution of not more of consanguineous marriages.
- "Drug-related data is very limited"
that the SMA drug treatment options are extremely limited and expensive striking Uzbay, "It's a biotechnological drugs in Turkey and possible reimbursement by the Ministry of Health. The price of this drug 70 thousand euros around." said.
Uzbay gave the following information about the treatment:
"The newer, the US Food and Drug Administration (FDA) and European Medicines Agency (EMA) approved the drug is not yet available in Turkey. Recently, a comprehensive gene from a new drug the FDA based on the technology 2 live up children was released with approval for use.
The drug works by replacing a gene called the missing or mutated SMN 1 gene. The Ministry of Health has not yet approved these two drugs. However, by applying to the Ministry of Health, if deemed appropriate, imported drug status can be obtained without reimbursement. Although the drug is used only in one dose, its price is over 2 million dollars. "
Stating that SMA is a disease with a poor prognosis, families are looking for a way out, Uzbay made the following evaluations:
"It is normal for families to have ear raised and want to use a drug with a single dose of reports that it is very effective, but some points should not be overlooked. Data related to the drug is quite limited. Currently, there are only 58 articles published in reliable scientific sources and more than half of them include reviews and comments on drug-related research articles.
There are not enough clinical trials related to the efficacy and long-term side effects of the drug, nor has it been a long time since its use. So time is needed to be precise about effectiveness and reliability. In addition, the drug has been approved for children up to the age of 2. It is either not possible at other ages or may be subject to special ethical clearance. If the consent is given up to the age of two, it is likely that the drug will be ineffective in delayed administration.
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