THE MALAYSIA Lysosomal Diseases Association (MLDA) unveiled its latest fund-raising effort recently, working with the creative talents of Fusion Wayang Kulit to shine a spotlight on the many difficult circumstances faced by patients with lysosomal storage diseases (LSDs), which involve many challenges, from diagnosis to treatment. LSDs are rare metabolic disorders that primarily affect children, impacting their growth and development. Many of the symptoms are non-specific, affect multiple organs, or mimic more common conditions, making them difficult to diagnose. With only 16 rare disease specialists in Malaysia, this often leads to delays in diagnosis. The challenges persist in securing access to treatment following diagnosis. Enzyme Replacement Therapy (ERT) comes with a high cost, often exceeding RM500,000 per year, and depending on the specific rare disease and the patient’s unique requirements, the expenses could escalate further.
Given the constraints of government-based funding,
