Significant improvement of sma patients using Spinerza drug
Dr. Majid Kihani Fard
Tehran
کد عضویت: System number: 79103
sma patients waiting for Spinerza drug
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Disease Asthma is a disease that is known as a dangerous disease due to the progress and speed of the patient's involvement in types zero, one and two, and requires a quick method to treat and care for the patient, it is a rare disease that has affected the movement and genetic muscle of the patient.. It has been discovered and launched for this disease... It's called Spinraza, which is used in the treatment of both children and adults. sma How effective is Spinerza?
Regarding these patients and the drug Spinerza, the question that has occupied the mind of many parents is the extent of its effectiveness and how to treat it through this drug, they want to know how effective the drug Spinerza will be and to what extent they can hope for the recovery of their children, so Dr.. to the oppressed patients and in this regard, here is an article that FDA about this issue and how the approval process and effect of Spinerza drug is mentioned here.
According to the organization FDA or US Food and Drug Administration, Spinerza is the only drug approved by this organization to be used as an injection into the fluid around the spinal cord.
### Report FDA on the drug Spinraza
Regarding Spinerza drug, Dr... Billy Dunn, Director of the Neurological Products Group at the Center and Agency FDA Regarding the common cause of infant mortality in this group, according to Dr. Dunn, says: "The length of treatment and the long time it takes to treat patients It is necessary that the most common cause of death of sick babies is a disease that can affect all stages of the patients' lives.. Sma is a genetic disease in which the loss and degeneration of motor neurons causes weakness and loss of muscles.. In this disease, many variables affect the age of onset, the progression of the disease and its symptoms.. Spinerza is the only drug approved by the FDA It is It is used for all types of muscular atrophy patients...
According to the site reports FDA This organization has been close to the sponsor and financial sponsor of Spinerza drug during the testing and review of the drug, and during the tests, 121 babies whose disease was diagnosed at the age of less than 6 months have been tested. The injection time was less than their age was 7 months and the treatment was random. In two groups, injections were performed in the spinal fluid and experimentally without injection (through touching the skin) (i.e. no treatment)... The result was the percentage of improvement in movement milestones such as head control, sitting, ability to kick while lying on the back (open arch), crawling, standing and walking.
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### Research results in a short time
FDA asked the sponsor of the drug to evaluate the results of the research and preliminary analysis in the shortest possible time, among the patients, 82 were eligible for analysis, and the result showed that 40% of the patients improved in the emotional milestone.
Additionally, uncontrolled open-label clinical studies were conducted in symptomatic patients between patients aged 30 days to 15 years at the time of first injection and asymptomatic infants aged 8 to 42 days at first injection. Tests in patients with muscular atrophy were...
### Specified side effects of Spinerza drug
Common side effects among muscular atrophy patients who were treated with Spinerza include upper respiratory tract infections, lower respiratory tract infections, and constipation.
Precautions include platelet counts and nephrotoxicity, and neurotoxicity has also been shown in animals.
### The mechanism of action of Spinerza drug
This drug is actually a kind of gene therapy that produces healthy and functional protein, the main cell protein is produced through SMN1, while this drug is produced through genetic engineering to produce protein through . lang="FA">is the leader.
### general research results
After conducting tests on type 1, 2 and 3 SMA patients, it was found that this drug has effective and stable effects and treatments on the patients and the motor and neurological abilities of these patients have improved.
### How to take Spinerza medicine
This medicine is injected in 4 weeks, one dose every week 12 mg/5 ml and then 1 dose every four months.
#### Precautions
As mentioned among the side effects of the drug, due to the possibility of bleeding and kidney problems, the patient should be monitored and monitored based on these two cases
#### Spinraza in Iran
Given the exorbitant cost of the drug in question and the lack of approval by the Ministry of Health, for this reason, unfortunately, the task of these patients in Iran is still in an aura of uncertainty and they must use countries where the drug in question has been approved and used to receive help and treatment. sma, you can refer to the relevant article on this site or click here.. Sources:
The official site of the FDA organization
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