Children with ultra-rare Lysosomal Storage Disorders are facing life-threatening risks as treatment interruptions continue under the National Policy for Rare Diseases 2021.
The Indian Medical Parliamentarians Forum, a cross-party group of 45 MPs who are also medical professionals, has urged the government to ensure uninterrupted access to Enzyme Replacement Therapy, the only life-saving option for many of these conditions.
In submissions to the prime minister and the Union Health Minister, the forum warned of a rapidly worsening situation. Around 60 patients have already crossed the existing annual funding cap of Rs 50 lakh, leaving them without further support. Nearly 100 children currently on therapy are at immediate risk of discontinuation as funds stall, and more than 60 children and young adults have already died due to delays or breaks in treatment.
The forum cautioned that even short interruptions in therapy can trigger acute metabolic crises, irreversible organ damage, and fatal complications. It has called for immediate removal or substantial revision of the funding cap for Group 3a Lysosomal Storage Disorder patients, along with a predictable long-term financing system that ensures continuous care.
It also pressed for streamlining fund disbursal across Centres of Excellence, warning that administrative delays are directly endangering patient survival and undermining the intent of the national rare disease policy.
