Archive · 11 Dec 2025
Children with Rare Disorders at Risk Due to Treatment Interruptions
A group of 45 doctors who are also members of parliament in India are very worried about children who have a very rare disease called Lysosomal Storage Disorders.
These children need a special treatment called Enzyme Replacement Therapy to stay alive.
The group says that because of some rules, many children might not be able to get this treatment, which could make them very sick or even cause them to die.
They have written to the prime minister and the health minister to ask for help.
They want the rules to be changed so that all children can get the treatment they need without any breaks.
They also want the government to make sure that the money for the treatment is given on time and without any delays.
The Indian Medical Parliamentarians Forum (IMPF) has sought uninterrupted access to Enzyme Replacement Therapy (ERT) for children with Lysosomal Storage Disorders (LSDs).
About 60 patients have already breached the Rs 50 lakh cap, leaving them without continued care.
Nearly 100 patients currently on ERT are at risk of discontinuation due to funding stalls.
More than 60 children and young adults have died due to delays in therapy initiation or breaks in treatment.
The IMPF has urged the government to remove or extend the Rs 50 lakh annual funding cap and streamline fund disbursal processes.
- Who
- Children with ultra-rare Lysosomal Storage Disorders (LSDs) and the Indian Medical Parliamentarians Forum (IMPF)
- What
- Risk of treatment interruptions due to funding caps and administrative delays in Enzyme Replacement Therapy (ERT)
- When
- Ongoing, with urgent intervention needed
- Where
- India, under the National Policy for Rare Diseases (NPRD) 2021
- Why
- To prevent avoidable deaths and ensure uninterrupted access to life-saving treatment
- Organization
- Indian Medical Parliamentarians Forum (IMPF)
- Number of MPs
- 45
- Condition
- Lysosomal Storage Disorders (LSDs)
- Treatment
- Enzyme Replacement Therapy (ERT)
- Funding Cap
- Rs 50 lakh
- Patients at Risk
- Nearly 100
- Patients Who Died
- More than 60