LSD patients, docs appeal to Tamil Nadu CM for treatment support

>Patients suffering from Lysosomal Storage Disorders (LSDs) and their families, along with renowned doctors from the city, gathered at Bharatiya Vidya Bhawan and appealed to the Hon’ble Chief Minister Ms J Jayalalithaa to support the management and treatment of LSDs. Patients and their families signed a petition addressed to Ms J Jayalalithaa to seek State Government support. Lysosomal Storage Disorders Support Society (LSDSS) provided this platform to the distressed families to make their voice heard.

Lysosomal storage disorders (LSD) are a group of around forty five (45) rare, genetic disorders that occur due to the deficiency of specific enzymes in special compartments (lysosomes) of the cells. LSDs occur in about one in 5,000 live births. A majority of LSDs are managed by means of supportive care measures that are disease specific. However, six of the LSDs can now be treated by means of Enzyme Replacement Therapies (ERTs). India currently has about 300- 400 patients, who have been diagnosed with treatable LSDs. Tamil Nadu has a total of 130* patients suffering these genetic disorders, however only 42* of these cases treatable disorders. Keeping in mind the low awareness levels of LSDs, LSDSS organized this event to spread awareness amongst the general public and decision-makers about rare disorders and their impact on patients’ lives and that of their families.

Dr S Suresh, Chief Medical Director, MD, MediScan said: “Awareness amongst masses and support from the government is critical for diagnosis and treatment of rare genetic disorders. Currently in our country, the responsibility of detecting early symptoms and ensuring an accurate diagnosis lies with physicians. Considering that the cost of treatment is quite high for curable LSDs, there could be a ray of hope for these patients with government support.”

Dr Sujatha M Jagadeesh, Geneticist MediScan said: “Since LSDs are rare and the patients are extremely few in number, sufferers and their families face hurdles in locating healthcare facilities and support centres that could address their ailment. The appeal to the Tamil Nadu State Government is therefore to play a crucial role in sensitizing the general masses about LSDs and helping patients to access treatment facilities.”

Pranav, a patient suffering from a rare genetic disorder called Gaucher was present at the event to share his story with the media and the masses. Pranav’s mother, Ms Karthiga Sivakumar said “Pranav has been lucky that his treatment has been supported under a compassionate programme. He has been undergoing Enzyme Replacement Therapy for 6 years and is doing very well now. I appeal to the government to help LSD affected children and to support their treatment.”

Mr Karunagaran, father of a 16 year old MPS patient, Dhivya says “My daughter suffers from MPS, which is a type of LSD. Since the treatment cost is very high, I on behalf of all parents of patient suffering from LSDs, appeal to the government to provide us assistance and help our children lead a regular life.”

Mr Prasanna Kumar Shirol, President, LSDSS said: “A number of patients across the state of Tamil Nadu suffer from LSDs but do not have access to treatment. Only government intervention can save these innocent people and help them lead regular lives. The need of the hour is to receive government support in financing the treatment of these diseases.”

*Source Data: Fetal Care Research Foundation (FCRF). FCRF is one of the nodal centers for clinical research and subsequently developing strategies for preventive, curative and supportive management of birth defects including LSDs