Rare Disease Fund extends support to another rare disease with $6.7 million gift

19 August 2021, Singapore – The Rare Disease Fund (RDF) today received a pledge of $6.7 million from businessman and philanthropist, Tan Sri Datuk Dr Chen Lip Keong. With this donation, the RDF can now extend its support to patients with the rare disease of Mucopolysaccharidosis type VI (MPS VI)*. The cheque was presented to RDF Committee Chairperson, Laura Hwang, at a ceremony witnessed by Professor Alex Sia, CEO of KK Women’s and Children’s Hospital (KKH). With the Government’s 3:1 co-matching of the funds raised, a total of $26.8 million will be added to the RDF.

Mrs Hwang said, “On behalf of the RDF, I would like to thank Tan Sri Dr Chen for this generous gift. With this donation, which is a marvellous launch of our 2021 fund-raising, RDF will be able to add MPS VI* to its list of supported conditions.”

“Lifelong medications to treat Rare Diseases are expensive and can cost up to hundreds and thousands of dollars each year for a patient. Donations to the RDF will build a funding model that is sustainable for the long term, so that we can assure qualified patients of our continued support for them. We hope that Tan Sri Dr Chen’s transformative donation will encourage other generous contributions from the public so that new conditions and medications can be added to benefit even more patients,” added Mrs Hwang.

A medical doctor in his early career, Tan Sri Dr Chen shares RDF’s vision for a caring and inclusive society where the community and government partner to help patients with rare diseases. His donation will be disbursed over five years.

Tan Sri Dr Chen said, “For each country where we operate in, I believe kindness works both ways. To be kind to the host nation, money made from the society must return to the community in some form. Singapore has one of the best healthcare and social security systems, if not the best in Asia Pacific.”

The RDF currently supports five conditions including MPS VI*, with seven medications. Most rare and severe genetic conditions are diagnosed in very young children. Therapies applied early can substantially extend the child’s lifespan and improve his or her quality of life.

*Annex 1

RDF Supported Conditions

Based on funds raised to date, the following lists medicines supported under the RDF for conditions that generally present with symptoms in childhood. As more funds are raised, the RDF can be expanded to cover more medicines or rare conditions.

About Mucopolysaccharidosis type VI (MPS VI)

MPS VI is a rare genetic lysosomal storage disorder characterised by complete or partial lack of enzyme activity for arylsulfatase B (also called N-acetylgalactosamine-4-sulfatase), leading to the accumulation of complex carbohydrates called glycosaminoglycans (GAG) in the body. This results in progressive damage to body cells, tissues, and organs. Today, only three children and one adult are known to have MPS VI locally.

MPS VI typically presents in childhood and can cause some patients to experience few clinical manifestations while others have life-threatening complications. Most patients will have normal intellectual development but will develop serious complications irrespective of whether they have rapidly or slowly progressive form of MPS VI.

Rapidly progressive MPS VI is associated with an onset of symptoms before three years of age. Affected individuals may develop walking problems by the age of 10 and experience delayed or no puberty. They are usually at risk of heart failure into their 20s or 30s. Slowly progressive MPS VI presents with later onset of symptoms, and usually diagnosed from five years of age and into adulthood. Despite a slower progression, individuals may still experience a decrease in overall function and physical ability by late adolescence.

Without treatment, severely affected individuals may survive till late childhood or adolescence. Those with milder MPS VI usually live into adulthood with shortened life expectancy. Galsulfase is designed to replace the natural enzyme which MPS VI patients lack to transiently restore enzyme activity, reduce clinical manifestations and improve symptoms.