COMMENT | Eleven years ago, our family received news that would change our lives in a totally unexpected way - our younger son Branden, then only one year old, was diagnosed with the rare genetic disease Spinal Muscular Atrophy (SMA).

The doctor told us that the disease was progressive and had no cure or treatment (then) and that our son was not expected to live for long. SMA is a leading genetic cause of death in infants due to progressive, debilitating muscle weakness.

It has been a very long journey, full of ups and downs, despair and joy, but Branden is still alive and doing relatively well today, although he requires 24 hours’ care physically from us (we have both given up our corporate jobs to become full-time caregivers and SMA activists).

Three life-changing treatments, including a gene therapy that is a cure if given before the child is two years old (Zolgensma by Novartis), have been developed and approved by the US FDA for SMA during this time. One of them, Evrysdi (risdiplam) by Roche, is approved for use in Malaysia.

With a lot of lobbying by ourselves and other SMA activists, and the help of the Ministry of Health (MOH) and doctors, both Novartis and Roche have provided a small number of Malaysian SMA children with their treatments for free through their respective compassionate use programmes since 2020.

Branden is one of the fortunate recipients. And as we have seen for ourselves, the treatment outcomes are very meaningful and have a...