The first medication to combat spinal muscular atrophy (SMA) has recently been approved by the United States Food and Drug Administration. It is approved for use by children as well as adults.
This rare disease is hereditary and causes the muscles around the spine to weaken and waste away. The lack of motor neurons in the spine and brain are the cause of the weakened muscles. There are many sub-classifications for SMA based on the location of affected area, age of onset, and severity of muscle loss.
In a controlled clinical trial, nearly half of all patients who received Spinraza showed some degree of increased motor function. Within the control group, not a single patient who received the mock treatment showed any signs of increased motor function. This clinical trial was restricted to infants less than seven months old.
In uncontrolled clinical trials, the drug was administered to a much wider array of patients. They ranged up to 15 years old and as young as eight days old. Results of these studies were encouraging as well. However, without a control in these studies, scientific conclusions cannot be drawn effectively.
Reported side effects include respiratory infections and constipation.
The FDA helped to expedite the regulatory process as a result of the encouraging studies that led up to the clinical trials. This aid took the form of preferred status and vouchers for immediate review.
More information concerning this rare disease can be found by clicking here.
