Spinal Muscular Atrophy (SMA) is a genetic disorder that affects the motor neurons in the spinal cord and brainstem, leading to muscle weakness and wasting. It is caused by a deficiency of a protein called survival motor neuron (SMN), which is essential for the normal functioning of motor neurons. SMA is a progressive disease, meaning that it gets worse over time, and its severity can vary widely between individuals. There are four main types of SMA, ranging from Type 1, which is the most severe and typically diagnosed in infancy, to Type 4, which is the mildest and usually diagnosed in adulthood.
Treatment
While there is currently no cure for SMA, there are treatments available that can help manage symptoms and improve quality of life, such as Spinraza, a drug that increases SMN production. Recent developments have resulted in adherence of a safe virus which delivers a fully functional human SMN gene to the targeted motor neurons. On the prevention side, genetic testing and counseling can help families understand their risk of passing on the condition to their children and make informed decisions about family planning. With early diagnosis and treatment, individuals with SMA can live longer, more fulfilling lives.
Cost Table for Treatment of SMA
Weight
Dose
Days/bottle (60 mg)
Days after Free Bottles
Cost/bottle
Monthly
Quarterly
Yearly
20+
5 mg/day
12
60
PKR 1,115,569
PKR 565,532
PKR 1,696,595
PKR 6,786,378
14
3.53 mg/day
17
85
PKR 1,115,569
PKR 399,199
PKR 1,197,596
PKR 4,790,385
08
2.03 mg/day
29
145
PKR 1,115,569
PKR 234,013
PKR 702,039
PKR 2,808,157
| Weight | Dose | Days/bottle (60 mg) | Days adding Free Bottles | Cost/Bottle | Monthly | Quarterly | Yearly |
|---|---|---|---|---|---|---|---|
|
20+ |
5 mg/day |
12 |
60 |
PKR 1,115,569 |
PKR 565,532 |
PKR 1,696,595 |
PKR 6,786,378 |
|
14 |
3.53 mg/day |
17 |
85 |
PKR 1,115,569 |
PKR 399,199 |
PKR 1,197,596 |
PKR 4,790,385 |
|
08 |
2.03 mg/day |
29 |
145 |
PKR 1,115,569 |
PKR 234,013 |
PKR 702,039 |
PKR 2,808,157 |
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