New Delhi, Dec 11 Access to Enzyme Replacement Therapy (ERT) for children and adults suffering from rare life-threatening diseases is facing significant delays, increasing the risk of death, said the Indian Medical Parliamentarians’ Forum (IMPF), a team of 45 members of parliament who are also medical professionals, on Thursday.
In a formal representation submitted to Prime Minister Narendra Modi – and also shared with Union Health Minister J P Nadda -- the doctors raised an urgent alarm over disruptions to the treatment faced by children and adults living with Lysosomal Storage Disorders (LSDs).
Lysosomal storage disorders are a group of more than 40 inherited metabolic disorders characterised by an abnormal build-up of various toxic materials in the body’s cells as a result of enzyme deficiencies. LSD is estimated to affect 1 in 7,000-8,000 births, and includes conditions such as Gaucher disease, Pompe disease, and Fabry disease.
While there is currently no registry of these patients in India, about 1-10 lakh people in the country could have these conditions.
Preventable treatment interruptions under the National Policy for Rare Diseases (NPRD) 2021 are causing an immediate risk to dozens of children with LSDs, warned the doctors led by its Chairperson, Dr Anil Bonde (a Rajya Sabha MP), calling for urgent and time-bound intervention to prevent avoidable deaths.
The IMPF, which serves as an informed action group to influence health policymaking and highlight public health issues in Parliament, stated that about 60 patients have already breached the Rs 50 lakh cap, leaving them without any viable path for continued care.
They added that a delay in funds is putting nearly 100 patients currently on ERT at an imminent risk of discontinuation, while more than 60 children and young adults have already died due to delays in therapy initiation or breaks in treatment.
The forum warned that even a brief disruption in ERT can cause acute metabolic crises, irreversible organ damage, and often death, turning every delay into a life-threatening event.
The doctors appealed for an urgent waiver or extension of the Rs 50-lakh cap for LSD patients to ensure no individual on therapy is pushed into regression or fatality due to funding gaps. They also urged the creation of a continuum-of-care funding framework under the NPRD to secure long-term, uninterrupted treatment.
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