Bone marrow transplant facility for MPS at city hosp
Times of India | 16 May 2025
123 Kolkata: The Centre of Excellence (CoE) for rare diseases in eastern India, situated at IPGMER, has begun the procedure to set up a bone marrow transplant (BMT) facility for children suffering from MPS (Mucopolysaccharidoses), a group of rare and inherited metabolic disorders.IPGMER is among the 12 health institutes across the country designated as CoE by the health ministry for the treatment of rare diseases."Another good treatment option for children suffering from two categories of MPS - 1 and 2 is BMT when they are below two years of age. We are moving forward with the procedure to have such a BMT unit for children suffering from this rare disease," said neonatology head Suchandra Mukherjee, the nodal official at the IPGMER CoE, on World MPS Day on Thursday.At present, only two CoE for rare diseases at AIIMS New Delhi and PGI Chandigarh have BMT facilities for this rare disease. While there is no cure for MPS, treatments such as enzyme replacement therapy(ERT) and stem cell transplantation are available to slow down the disease progression. On average, a child is given ERT twice a month. The treatment is costly and beyond the reach of most parents.Under the National Policy for Rare Diseases, a patient with any recognised rare disease, including MPS, can get one-time financial aid of Rs 50 lakhs for treatment. However, many times, the funds get exhausted, leaving these patients in the lurch.In fact, of the 25 children with MPS, including a few from the neighbouring states, are registered with the SSKM CoE for treatment. The funds for four children have already been exhausted, forcing them to take up supportive treatment."Early diagnosis is key to a better treatment outcome. We are holding regular training and sensitisation programmes for paediatricians for awareness. At IPGMER, we are also starting pre-natal screening. This is an inherited disease," added Mukherjee.