Parents of child with Spinal Muscular Atrophy appeal for increased support for rare disease treatment in Budget 2019

PETALING JAYA: Parents of a child with Spinal Muscular Atrophy (SMA) have urged the Federal Government to expand treatment for rare diseases in  Budget 2019.

Edmund Lim and Yap Sook Yee, whose son Branden who has SMA Type 2, called on the government to show fairness and equality by funding the drugs which allow children with SMA to get life-saving and life-changing medicines like Spinraza. 

Below is the full open letter:


Dear YB Lim Guan Eng and YB Datuk Seri Dr. Haji Dzulkefly bin Ahmad,

Our country voted for a change in government at the 14th General Election driven by a desire to build a better future, for ourselves and 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 their parents, siblings and extended family.

Recent local research shows that SMA occurs in at least 1 in 20,000 Malaysian live births or 1 baby is born with SMA at least every fortnight. About 1 in 50 people or 640,000 Malaysians are carriers of SMA. Carriers of SMA 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 afflicted by this genetic disease.

Are we sacrificing the lives of children with SMA?

SMA is a rare disease that presents fundamentally different challenges from those of more common non-communicable diseases, with complex medical challenges requiring multi-disciplinary intervention. It requires extra resources to tackle, and not in the same way compared to 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 to the extent preferred. Compromises will be made.

This letter appeals to you and the Pakatan Harapan Government that in making compromises, please do not sacrifice the lives of children with SMA who are most vulnerable with significant unmet needs.

Revolutionary new medicines are available today that stops the progression of SMA, but are inaccessible to children with SMA in Malaysia. All the SMA babies born in 2017 and stayed in Malaysia have died from data compiled by non-governmental organisations.

Statistics for 2018 are expected to be similarly grim and tragic. 

For the children with SMA 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 the 14th General Election to increase the budget allocations and provide incentives to tackle rare diseases like SMA.

Also, under the Persons with Disabilities Act 2008 (“PWD Act”), children with SMA deserve the enjoyment of health on an equal basis with persons without disabilities and the Government has a duty and responsibility to fulfil this promise.

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.

To become a truly inclusive and fair nation

Thousands of individuals and companies (both big and small) have rallied to help raise the standards of care and support for children with SMA, by donating and volunteering. Pharmaceutical companies with approved and experimental orphan drugs for SMA are 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 serve, to treat and to 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 paves the way for children with SMA to get lifesaving and lifechanging 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.

Allocate to treat SMA in Budget 2019

The Government plays a crucial role in making hopes real for children with SMA.

We urge the Government to provide for orphan drugs like Spinraza to treat SMA in Budget 2019.

Just because a disease affects a relatively small number of children 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 sma-ll ones with SMA.  
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