Zubair Qureshi
Participants of a seminar on Saturday about Spinal Muscular Atrophy a genetic condition that causes muscles throughout the body to break down – were apprised about the treatment which is now available in Pakistan.
The panelists called on the government, philanthropists, private donors, and pharmaceutical companies to help support the expensive treatment available to all to save lives.
The event “SMA is No More Untreatable” was organized by the Strive Trust, a not-for-profit organization working for improving the quality of life of Persons with Disabilities (PWDs), on Saturday.
Previously, the disease was untreatable until recently, but the development of the new treatments by the world’s leading biotech companies have changed this fact. One of these treatments introduced in Pakistan has been heralded as a major breakthrough in the fight against the disease.
However, the cost of the drug made it inaccessible for many patients. The annual cost of the drug is between Rs2 million to Rs7 million after 80 percent discounts by the company still it’s out of reach for even upper-middle income families.
Dr. Salman Kirmani, Genetics Specialist and Associate Professor at Agha Khan University said currently there are three treatments available in the world for the disease.
First is Gene therapy that is only recommended for the children of up to two years of age. The two other medicines are oral and injectable.
Oral treatment is now registered and available in Pakistan but it’s too much expensive, he said. He warned the patients to never go for stem cell transplant for SMA as there is no evidence in the world that it works.
Dr. Kirmani said the disease is not only a financial but psychological and emotional burden.
Dr. Raman Kumar, Child Neurologist, Liaquat Hospital, Karachi said in 1000 children one child is born with SMA. It’s a rare but treatable disease. But due to the lack of awareness and diagnostic facilities, it prevalence level is known in Pakistan.
Explaining about the disease he said, SMA can be classified into four types depending upon the severity of the disease.
This happens when the body cannot produce essential protein called “survival motor neuron,” which is needed for specialized nerves called motor neurons to function properly. It can affect a child’s ability to crawl, walk, sit up, and control head movements. Severe SMA can damage the muscles used for breathing and swallowing leading to child death. It’s a progressive disease and cause lifelong disability in adults. There’s no cure for SMA, but ongoing treatments can help children with SMA live a better life.
