Two toddlers to undergo life-saving ERT therapy

According to Dr Mohandas Nair, head of the department of Pediatrics, Kozhikode Medical College, rare diseases like LSDs are complex and heterogeneous and require a sustainable support mechanism for diagnosis and treatment. Dr Mohammed Asheel, Executive Director, Kerala Social Security Mission, commended the Kerala High Court's efforts to raise money for rare disease patients who have been ignored for a long time.

Two baby girls, aged 2 and 3 years and diagnosed with Pompe disease, a form of Lysosomal Storage Disorders (LSDs), have been cleared to start their life-saving Enzyme Replacement Therapy (ERT) at the Government Medical College, Kozhikode. While the first patient from Kannur will receive her infusion therapy on Friday, the second patient will begin her treatment in November.

The infusion therapy was started following an order of the Kerala high court on August 14 this year on a petition filed by the Lysosomal Storage Disorders Support Society(LSDSS), a patient advocacy group, set up to fight the cause of rare disease patients across the country, a society press release said. Both these patients were supported by Sanofi Genzyme under its charitable AccessProgrammeand provided the short-term bridging therapy considering their medical condition.

According to ManojManghat, Kerala state co-ordinator of LSDSS, this was a unique financing model where not just the Union Government, but the state of Kerala and the Kerala High Court Advocates Association too contributed towards a corpus fund, set up to start treatment for the two children. This innovative financing model is a step in the right direction, and could be explored by other states to provide sustainable funding support for LSD patients, awaiting treatment without any institutional support so far, he said.

While the Centre allotted a Rs 1.5 crore, the state sanctioned another Rs 50 lakh for the treatment of these two rare disease patients. Members of the Kerala High Court Advocates Association, following an appeal by the High Court, also mobilised an amount of Rs 5 lakh for the treatment.

Health experts and patients are now hopeful that the Kerala government, under its ambitious Kerala Social Security Mission (KSSM), will create a dedicated fund pool to support the treatment of all LSD patients in the state. According to Dr Mohandas Nair, head of the department of Pediatrics, Kozhikode Medical College, rare diseases like LSDs are complex and heterogeneous and require a sustainable support mechanism for diagnosis and treatment.

Dr Mohammed Asheel, Executive Director, Kerala Social Security Mission, commended the Kerala High Court's efforts to raise money for rare disease patients who have been ignored for a long time. He said there was a need for sustainable treatment funding in the National Policy for Rare Diseases 2020.

The bench, headed by Chief Justice S Manikumar, while passing the order, had also directed the Kerala State Legal Services Authority to explore the possibility of raising funds for providing treatment to the children who suffer from rare diseases.