This Mother Is Fighting For More Funding To Treat Rare Diseases In Budget 2019
Morquio IVA syndrome is one of the rarest forms of the Mucopolysaccharidosis disease.
By Alyssa J. Oon — 04 Oct 2018, 07:05 PM
The Pakatan Harapan government is now less than a month away from tabling Budget 2019, its maiden Supply Bill
The bill will be tabled on 2 November, and debates on it will carry on for four days.
Finance Minister Lim Guan Eng told Malaysiakini on 27 September that he expects Budget 2019 to be a difficult one.
He added in jest, "It is likely I will end up as the most unpopular finance minister because I have no goodies to give out."
As the bill affects all Malaysians, a mother whose daughter has been diagnosed with a rare congenital disease hopes her appeal will be considered for Budget 2019
Tang Yee Suh made an appeal through a public post on her personal Facebook account.
The post, which went up on 20 September, has over 400 comments and 2.1k shares at the time of writing.
Tang shared her journey thus far in finding out her daughter Joy Qing's diagnosis and how increased government funding could help. She also urged friends and family members to comment "#belanjawan2019" and "#RareDiseasesMPS" below it.
In May, three-year-old Joy Qing was diagnosed with a rare congenital disease called Morquio A syndrome
"Joy Qing was always petite compared to her peers, especially after the age of two," Tang told SAYS.
Doctors had previously assured Tang and her husband that Joy's size was normal, "as both the parents are not tall as well".
"We brought her to see geneticist at University Malaya Specialist Centre (UMSC), which later referred us to University Malaya Medical Centre (UMMC) for a diagnosis confirmation for Mucopolysaccharidosis type IVA (Morquio A syndrome)."
Morquio A syndrome is a rare congenital disease with an occurrence of 1 in 250,000 births
According to The National MPS Society, Morquio IVA syndrome is one of the rarest forms of the disease.
"Her body is lacking one type of enzyme that will cause toxins to be accumulated in her body. (It) will then attack her other body organs progressively," Tang shared in her post.
To SAYS, Tang added, "It was really a big shock for us as we never expected this to happen. For us, (we thought) she may be lacking some growth hormone probably, but it turned out to be way beyond our expectations."
Morquio syndrome patients experience normal growth up until two years old. Tang said the other symptoms such as like pigeon chest, flex wrist, and knock knees have begun to show in Joy.
Joy can be treated with enzyme replacement therapy (ERT), but it is an expensive, lifelong commitment
Tang had joined the Malaysia Lysosomal Diseases Association (MLDA) for support, which was where she found out about ERT treatments at Hospital Kuala Lumpur (HKL).
However, the treatment is too expensive to be afforded by an ordinary family. Tang said treatment for a patient who weighs 10kg could cost roughly RM750k a year.
"My happy, smart, and cheekiest girl is now labelled as a no cure patient and I, being her favourite person as she calls, am financially incapable of getting her the Vimizim that she needs in order to have better quality of life," Tang wrote in her post.
MLDA told The Star that ERT for one patient ranges between RM500,000 and RM1 million a year.
Although Budget 2018 had set aside RM10 million for treatment of rare diseases, it has already run out
Tang said Joy is one of 11 patients on the waiting list for ERT. Only five patients are being treated at the moment.
"According to doctors, ERT should be started once diagnosed as any damage is irreversible," Tang said.
"Government funding this year has reached its quota. If the funding could be increased by another RM10 million a year, then the 11 patients in queue now will likely to be able to get their ERT."
Tang told SAYS that crowdfunding was not an option either, as it is not sustainable.
Now, Tang is on a mission to get Parliament to increase the funding for treatment of rare diseases
"We are not sure whether (government funding for treatment of rare diseases) will carry on for life, but we know that if next year the government reduces or stops the funding... those currently on ERT will also be halted," Tang said.
Tang told SAYS that she has met with her state assemblyman, Kinrara MP Ng Sze Han and is currently arranging to meet with Women, Family, and Community Development deputy minister Hannah Yeoh.
On what she plans to share with Yeoh, Tang said, "I will share about my feelings, worries and difficulties since the diagnosis. I hope for her support on increasing medical funding for MPS patients, and also to include Morquio patients in the new National Health Care Insurance."