A national policy on rare diseases is needed so that there can be a clear framework for early diagnosis, documentation and treatment of rare diseases. It will also ensure better budget allocations for the treatment of all rare diseases.

“Without a national policy for rare diseases, funding will continue to be an issue and many patients will go without treatment. Many will be undiagnosed due to the lack of awareness about rare diseases and treatments that are available,” says Lee Yee Seng, president of the Malaysia Lysosomal Disease Association (MLDA).