ISLAMABAD: If the government can provide free treatment for hepatitis C, HIV/Aids and Covid-19, why not Spinal Muscular Atrophy (SMA), genetics specialist and associate professor at Aga Khan University Dr Salman Kirmani said on Saturday.
He was speaking at an event titled ‘SMA is No More Untreatable’ organised by the Strive Trust, a not-for-profit organisation working for improving the quality of life of persons with disabilities.
The Spinal Muscular Atrophy (SMA) is a genetic condition that causes muscles throughout the body to break down.Previously, the disease was untreatable until recently, but the development of new treatments by 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. Although companies give 80pc discount, it is still out of reach for even upper-middle income families.
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Dr Kirmani said he hoped that one day the treatment of SMA would be available in government hospitals and children would not have to die and adults would not have to live with disability just because of its expensive treatment.
He said there were three treatments available in the world for the disease.
“First is Gene therapy that is only recommended for children 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 is very expensive,” he said.
Dr Kirmani warned patients not to go for stem cell transplant for SMA as there was no evidence in the world that it worked, adding that the disease was not only a financial but psychological and emotional burden. There was a time, he recalled, when he used to say to the patients that the disease was not treatable so take their child home.
“But now we face ethical dilemma that although the treatment is available, it is beyond the reach of majority of patients. It becomes hard for them to tell this to the parents of the patients,” he said.
Child Neurologist, Liaquat Hospital Karachi, Dr Raman Kumar said in one child out of 1,000 children was born with SMA. It is a rare but treatable disease, but due to lack of awareness and diagnostic facilities, its prevalence level is not known in Pakistan, he said.
Founder and chair of the trust Mohammad Yasir Khan, who himself suffers from the disease, said: “It is the responsibility of state to provide lifesaving drugs even if they are expensive.”
Parliamentary Health Secretary Dr Shazia Sobia said she would highlight the issue on the floor of the house and encouraged participants to come forward with their proposals and demands.
Published in Dawn, September 25th, 2022