Children living with rare and debilitating lysosomal storage diseases in India are facing a devastating setback as their access to life-saving treatment has been abruptly halted. The one-time financial support provided by the Union health ministry for these conditions has run out, leaving families desperate for a sustainable solution.
These rare genetic disorders, like Gaucher disease and Hurler syndrome, cause the malfunction of enzymes needed to break down harmful substances in the body. This leads to a progressive accumulation of these substances, damaging organs and impacting vital functions. Enzyme replacement therapy (ERT), while expensive, can significantly improve quality of life and manage symptoms.
For many families, the ministry’s one-time grant was a lifeline, granting their children access to crucial ERT. However, with that funding exhausted and no long-term support mechanism in place, the future looks bleak. Children are being forced to discontinue treatment, facing the prospect of their conditions worsening and their health deteriorating.
Patient advocacy groups are raising their voices, urging the government to implement a policy for consistent and comprehensive funding for lysosomal storage diseases. They highlight the need for a long-term, sustainable approach that recognizes the chronic nature of these conditions and ensures patients have uninterrupted access to essential treatment.
The lives of hundreds of children hang in the balance. Addressing this urgent need through immediate action and a permanent support system is crucial. Only then can families facing this daunting challenge hold onto hope for their children’s future.