Kuala Lumpur, 13 August — According to studies, at least one child is born each week with Spinal Muscular Atrophy (SMA) in Malaysia. Without effective treatment, most of those with Type 1 SMA will not live beyond their second birthday. Around 4,000 Malaysians are estimated to be with SMA and 700,000 are estimated to be carriers of the gene which causes this genetic disorder.

In conjunction with August as SMA Awareness Month, the Galen Centre for Health and Social Policy calls on the Government of Malaysia to continue its commitment and efforts in working with the SMA community and the pharmaceutical industry to provide timely access to lifesaving treatment to save and transform the lives of those living with this condition. Children with SMA, in particular, are being left behind.

“Despite there currently being no cure for SMA, there are effective treatments which when delivered early, are life-changers for infants and young children, allowing them to breathe with depending on a ventilator, sitting up on their own, crawling and walking,” said Azrul Mohd Khalib, Chief Executive of the Galen Centre for Health And Social Policy.

“These one-off treatments are life-savers and could potentially improve upon quality of life, and life expectancy. It is also possible for children with SMA to survive long into adulthood and live healthy, fulfilling and productive lives. However, these therapies are not available in Malaysia.”

“We call upon the relevant pharmaceutical manufacturers to submit their applications to the National Pharmaceutical Regulatory Agency for appraisal and registration of their respective SMA therapies in Malaysia. The continued absence of effective treatments for SMA in Malaysia denies children, young people and adults with this condition, the chance of living a different and better future.”

“Thanks to the combined voices and efforts of organisations and advocates such as WeCareJourney, the strength, resilience and courage of children, young people and adults with SMA, there is now an opportunity to learn from the COVID-19 crisis on addressing gaps, fulfilling needs, and mobilising the necessary resources to make the impossible, possible.”

Spinal muscular atrophy (SMA) is a rare genetic condition that deprives people of physical strength by affecting the motor nerve cells in the spinal cord, and impacting their ability to walk, eat, and even breathe.

SMA treatments are most effective when delivered early. It is necessary to treat before symptoms appear, as there is rapid degeneration that affects motor neurons needed for muscle strength. Lost motor neurons cannot be healed or restored.

The US Food and Drug Administration has approved three treatments for SMA: Spinraza (2016), Zolgensma (2019), and Evrysdi (2020). These therapies have made it possible for people with SMA today to stop the progression of the disease, improve motor function, achieve developmental milestones, and live independently and contribute to their communities.