There is some good news for children suffering from progeria. They can now receive Lonafarnib, the drug for the treatment of disease, locally in India without travelling to the US for the clinical trial. The Progeria Research Foundation (PRF) is the one and only organization in the entire world that works towards discovering treatments and the cure for children with progeria.
The PRF is now working in close coordination with Eiger Biopharmaceuticals – the company that manufactures drug Lonafarnib. The PRF and the drug manufacturer have launched the lonafarnib Managed Access Program (MAP).
This program allows young adults and eligible children with progeria to obtain the drug lonafarnib before it is approved for commercial sale.
The program is the only way of getting access to the drug for the treatment of progeria. “This treatment will be made available through the child’s local physician. Any parent who wishes to enroll their child in this program must have a doctor in India caring for their child, and a child can only be registered for the MAP by his/her treating doctor,” reports ET Healthworld.
A child, the resident of the Yavatmal district of Maharashtra, is the first progeria patient to enrolled for the Managed Access Program (MAP). The program aims at helping more Indian children suffering progeria to get treatment locally. Those who know about any child who may have progeria can get in touch with the PRF via a mail to info@progeriaresearch.org . Doctors who wish to register for the program can also get in touch using the same email id.
PRF is a non-profit organisation that was founded in 1999. During that time, there were no resources available for children with progeria. Hutchinson-Gilford Progeria Syndrome ‘Progeria’ or ‘HGPS’ is a rare disease that causes children to age rapidly.
This disease affects approximately 1 in 20 million people. According to an estimate, there are around 400 children around the world living with this disease. There are some 200 progeria patients who remain undiagnosed. In terms of India, there are 60 children living in India with progeria. But only 18 of them have been identified.