Children suffering from ultra-rare Lysosomal Storage Disorders at risk, warns MPs’ forum

New Delhi, Dec 11 (PTI) The Indian Medical Parliamentarians Forum (IMPF) -- ”a cross-party group of 45 MPs who are also medical professionals -- ”has sought uninterrupted access to Enzyme Replacement Therapy for children suffering from Lysosomal Storage Disorders conditions.

The IMPF warned that dozens of children with ultra-rare Lysosomal Storage Disorders (LSDs) are at immediate risk due to preventable treatment interruptions under the National Policy for Rare Diseases (NPRD) 2021.

The forum serves as an informed action group to influence health policy-making and highlight public health issues in Parliament.

In a formal submission to the prime minister, the forum, led by its Chairperson Dr Anil Bonde (Rajya Sabha), has called for urgent, time-bound intervention to prevent avoidable deaths and ensure uninterrupted access to Enzyme Replacement Therapy (ERT), the only life-saving treatment for many LSD conditions.

A similar letter has also been submitted to Union Health Minister JP Nadda.

The IMPF's representation underscores a rapidly deteriorating situation. It stated that about 60 patients have already breached the Rs 50 lakh cap, leaving them without any viable path for continued care.

Also, nearly 100 patients currently on ERT are at imminent risk of discontinuation as funds stall.

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.

To avert further tragedy and uphold the commitments of the NPRD 2021, the forum has urged the Government of India to immediately remove or significantly extend the Rs 50 lakh annual funding cap for Group 3(a) LSD patients, recognising that current limits do not reflect clinical realities or lifetime treatment needs.

It also suggested putting in place a predictable, ring-fenced continuum-of-care financing mechanism, ensuring long-term therapy without interruption for eligible patients.

It also called for streamlining fund utilisation and disbursal processes across Centres of Excellence, eliminating administrative delays that directly endanger patient survival. PTI PLB MNK MNK