ISLAMABAD: Spinal Muscular Atrophy (SMA), an inherited disease that affects nerves and muscles, causing muscles to become increasingly weak and that leads to permanent disability, was not treatable till few years back.
Strive Trust a not for profit organization on Thursday organized an international conference to create awareness among public about the disastrous affects of SMA and recent advances in the medical science for its treatment.
The disease mostly affects infants and children but can also develop in adults. Approximately 10,000 to 25,000 children and adults are living with SMA in the United States.
There is no data available about SMA patients in Pakistan, however, it is believed that cases may number in thousands. The rare disease affects one out of 6,000 to 10,000 children.
Pakistan is among the countries that have hardly any facilities, special person, particularly for SMA patients.
Former Senator and educationalist Sehar Kamran in her speech at the moot emphasized the need for collaborative efforts to make the treatment available and affordable for patients in Pakistan.
She said there is a need to establish an educational channel at the state level. “Had such a channel been available, the experts who had been sharing valuable knowledge, would have been accessible to a wider audience” she added.
Ruling Pakistan Tehreek e Insaaf Member National Assembly and Parliamentary secretary ministry of human rights Lalchand Malhi sharing his thoughts said the government is trying to ensure health facilities to every citizen. It has introduced Insaaf health card; and differently-abled persons should take advantage of the facility. He said Pakistan Bait-ul-Maal is another benevolent organization available for the needy including patients.
He assured his ministry’s cooperation for any steps that can improve quality of life of persons with disabilities.
He mentioned that there is no permanent cure for SMA and treatment available is very costly around the globe.
“I will raise the voice about SMA disease in concerned ministries to ensure afforadable treatment,” he maintained.
Founder Strive trust, Yasir Khan, himself a victim of SMA said that about 60% of patients have severe type also known as Werdnig-Hoffman disease.
Symptoms appear at birth or within an infant’s first six months of life infants with this type of SMA have difficulty swallowing and sucking.
He lamented that people at the policy level and government institutions were not sensitive about disabled persons.
Many persons with disabilities who can be treated, die because they are not provided proper treatment.
He said since treatment of SMA is available internationally, government of Pakistan must ensure that it is available to the patients in Pakistan.
Jasdev Singh Sandhu and Nerish Sasedharan, leading Asian Robotics company Cyberdyde from Japan shared their views about SMA disease. Robotic technically has been proven to assist people who are recovering from strokes and people who have abstained injuries that effect their daily tasks. They announced plans to launch their products in Pakistan by next year.
The speakers and neuro experts agreed that there is no permanent cure for SMA yet, however treatment depends upon the type of SMA and symptoms. Recent advancements in science has given hope to the patients with SMA. The available treatments are very costly though.
Many people with SMA benefit from physical and occupational therapy and assistive devices, such as orthopedic braces, crutches, walkers and wheelchairs.
They were of the view that some drugs are now available in the market after a thorough research on SMA globally, including Nusinersen (Spinraza®) for children ages 2 to 12.
It is injected into the space around the spinal canal. Similarly, a different medication, Risdaplam (Evrysdi®), helps adults and children older than two months.
Children younger than two may benefit from a one-time intravenous (IV) infusion of a drug called Onasemnogene Abeparvovec-xioi (Zolgensma®). This therapy replaces a missing or faulty SMN1 gene with a functioning gene.
The government of Pakistan recently approved a newly discovered drug “Evrysdi”- a medicine used to treat SMA.
The drug costs a patient around $ US 340,000 per year. Other treatments available include gene-therapy by Novartis where, their injection named Zolgensma costs British pounds 1.7 million per dose. The treatment was labeled the most expensive drug in the world.
Strive trust demanded government of Pakistan, international donor agencies, health experts and especially pharmaceutical companies to ensure availability of these drugs in Pakistan at affordable price to save lives of hundreds of people who are suffering from SMA.
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