On the occasion of International Gaucher day 2019, Lysosomal Disorders Support Society (LSDSS), a Patient Advocacy group (PAGs) in association with eminent doctors organized a meeting of key stakeholders at Sir Ganga Ram Hospital, New Delhi.
The event was aimed at raising awareness about Treatable Lysosomal Storage Disorders (LSDs) and its different aspects including the support to the rare diseases’ community at large. Renowned stakeholders including Dr. I. C. Verma, Advisor, Institute of Medical Genetics & Genomics, Sir Ganga Ram Hospital, Dr. S. P. Byotra, Vice Chairman, Sir Ganga Ram Hospital, Dr. Ratna Dua Puri, Chairperson, Institute of Medical Genetics & Genomics, SGRH and Mr. Manjit Singh, President – LSDSS spoke about the various aspects of LSDs, their long-term management and the challenges faced by the patients and families.
Dr. Ratna Dua Puri, Chairperson, Institute of Medical Genetics & Genomics, SGRH said “LSDs are a group of around 50 inherited disorders which occur due to defects in the lysosomal function of the cell. As of now, the treatment is available only for a few of the LSDs – Pompe, Fabry, Gaucher, MPS (Type I, II, IV A, VI, VII), Niemann pick disease and has proven to be effective. If the treatment isn’t provided on time, these conditions can severely impact the life of a patient. Timely treatment is, thus, crucial for the management of these patients.”
The event also highlighted the challenges faced by the families with LSDs and the plight of patients who are yet to receive the treatment from State government.
“As a non-profit organization working for the rights of patients with rare diseases, we have been repeatedly appealing to the Ministry of Health and Family Welfare for treatment support as well as to roll out the policy. The Judiciary directed the Health Ministry to provide Interim treatment to 200 odd patients whose approved applications are pending with them, till the policy is rolled out. In the absence of timely treatment, we are losing about 5 Patients every month. Given this situation, every day’s delay in finalizing the policy for the treatment of these rare disorders means unwanted agony and trauma for patients and their families – as well as unfortunate deaths occurring during this period. We have also started a petition on Change.org to help us reach the right decision makers” said Mr. Manjit Singh, President, Lysosomal Storage Disorders Support Society (LSDSS).”
The event was also a platform to bring patients and families together to raise their collective voice to seek treatment from the central government.