Drug delivery for lysosomal Storage Diseases- A milestone in rare disease therapy

 February 08, 2025 Kerala Medical Services Corporation Limited (KMSCL) 3 Comments

Kerala has made remarkable strides in the treatment of rare diseases. For the first time in the state, a scheme has been launched to provide medications for lysosomal storage diseases. Lysosomal storage disorders are rare conditions wherein organs suffer damage due to the deficiency of enzymes necessary for the proper functioning of lysosomes in body cells. 

This initiative commenced at the hospital, where the drug was administered to five children. Medications worth Rs 20 lakh per month were distributed free of cost through the K.M.S.C.L (Kerala Medical Services Corporation). In the first phase, medicines valued at Rs 53 lakh were successfully delivered. The state has accorded special importance to the treatment of rare diseases, resulting in the hospital's elevation to the status of a centre of excellence. 

Additionally, SAT was introduced for the first time for the treatment of rare diseases. The hospital initiated a clinic for SMA (Spinal Muscular Atrophy) and subsequently, a scheme was devised to provide expensive medications for rare diseases. Free medicines were distributed to 56 children affected by SMA (Spinal Muscular Atrophy)Moreover, in a ground-breaking development in the government sector, the Thiruvananthapuram Medical College Hospital has commenced state-of-the-art surgery to correct the curvature of the spine in affected children. SAT was introduced for the first time in medical colleges in connection with this endeavour. 

Furthermore, permission has been granted to launch a genetics department at the hospital. The Genetic and Metabolic Lab at CDC, Thiruvananthapuram, has received NABL (National Accreditation Board for Testing and Calibration Laboratories) approval for the diagnosis of rare diseases

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