NEW DELHI: The Indian Medical Parliamentarians’ Forum (IMPF), a cross-party collective of 45 medical professionals turned parliamentarians, have issued a public health alert, warning that dozens of children with ultra-rare Lysosomal Storage Disorders (LSDs), a rare inherited metabolic conditions caused by a deficiency of specific enzymes, are at immediate risk due to preventable treatment interruptions under the National Policy for Rare Diseases (NPRD 2021).

In a letter addressed to Prime Minister Narendra Modi, the Forum’s Chairperson, Dr Anil Bonde, has called for urgent, time-bound intervention to halt avoidable deaths and ensure uninterrupted access to Enzyme Replacement Therapy (ERT), the only life-saving treatment for many LSD conditions.

The BJP Rajya Sabha MP from Maharashtra said nearly 100 patients currently on ERT are at imminent risk of discontinuation as funds have stalled.

“Over 60 children and young adults have already died due to delays in therapy initiation or breaks in treatment,” said the letter, which was also addressed to Union Health Minister JP Nadda.  

The letter highlighted that of the 60 patients, 20 have died in the last 12 months

The Forum, which has MPs from both the Houses as its members, 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.

Lysosomal Storage Disorders (LSDs) like Gaucher, Pompe, Fabry, and MPS are rare genetic conditions from missing enzymes, causing toxic buildup in cells, leading to severe, progressive multi-system damage (organs, nerves, bones) from infancy to adulthood.

The center has notified the National Policy for Rare Diseases (NPRD) in March 2021 to provide affordable care for rare disease patients, categorizing diseases into three groups and establishing Centres of Excellence (CoEs) for treatment, diagnosis, and research, offering financial aid up to ₹50 Lakhs for curative treatments.

However, the Forum said, four years after notification, serious implementation gaps are now costing young lives.