Give these children a chance to live
WE voted for a change in government at the 14th General Election driven by a desire to build a better future for ourselves and the generations to come – our children.
Some children are especially vulnerable and left behind.
Children with spinal muscular atrophy (SMA) suffer a degenerative problem that affects the motor nerves, resulting in muscle wasting and weakness.
SMA is debilitating and chronic, and causes considerable long-term psychological, medical and financial burden on the child as well as his/her parents, siblings and extended family.
Recent local research shows that SMA occurs in at least one in 20,000 Malaysian live births, or one baby is born with SMA at least every fortnight.
About one in 50 people, or 640,000 Malaysians, are carriers of SMA. These carriers have no symptoms but when a husband and wife who are carriers have a baby, there is a real risk their child will be born with this genetic disease.
SMA is a rare disease with complex medical challenges requiring multi-disciplinary intervention. It requires extra resources to tackle, and not in the same way as the more common diseases.
With the fiscal challenges our country is facing and the expectations of a less popular Budget 2019, we believe not everyone’s needs will be satisfied. Compromises will have to be made.
We are appealing to the Finance and Health Ministers particularly, and the Pakatan Harapan government in general, that when making these compromises, please do not sacrifice the lives of children with SMA who are the most vulnerable. Revolutionary new medicines are available today to stop the progression of SMA.
However, these medicines are inaccessible to children with SMA in Malaysia. All the SMA babies born in 2017 and who stayed in Malaysia have died, according to data compiled by NGOs. Statistics for 2018 are expected to be similarly grim and tragic.
For those who are still surviving, if they are given the required medicines or orphan drugs (medicinal products intended for diagnosis, prevention or treatment of life-threatening or very serious diseases or disorders that are rare), we can dramatically improve their chance to live and thrive.
The government is urged to follow through on its promise prior to GE14 to increase the budget allocations and provide incentives to tackle rare diseases like SMA.
Also, under the Persons with Disabilities Act 2008, children with SMA deserve to enjoy health on an equal basis with persons without disabilities and the government has a duty and responsibility to observe this law.
Access to health is a basic human right, and every child with SMA must be allowed to enjoy equal human rights and respect for their inherent dignity.
Thousands of individuals and companies (both big and small) have rallied to help raise the standards of care and support for children with SMA. Pharmaceutical companies with approved and experimental orphan drugs for SMA are also keen to help. Doctors in over 30 other countries (and counting) strive to get their patients treated with SMA orphan drugs.
With funding available, our neurologists and paediatricians should also rally to treat and make lives better for children with SMA and their families.
It is encouraging that Malaysia has a precedent to rescue children with orphan drugs even though such lifesaving drugs are typically expensive. There is every reason to add SMA to that precedent today.
With Budget 2019, the government can demonstrate equity by expanding treatment for rare diseases, and fairness and equality by funding SMA orphan drugs. This would pave the way for children with SMA to get lifesaving and life-changing medicines like Spinraza.
We also look forward to promising new SMA orphan drugs that are expected to be approved for use in the near future.
The government plays a crucial role in making hopes real for children with SMA. Hence, we urge the government to provide funds for the orphan drugs to treat SMA in Budget 2019.
Just because a disease affects a relatively small number of children, it does not make it irrelevant or less important. Children with SMA should not be left out and left behind.
Please do not give up on our small ones with SMA.