Experts urge Centre to recognise Huntington’s disease under NPRD 2021 to improve access to costly treatments and support systems
Huntington’s Disease patients and experts have urged the Indian government to formally include the condition under the National Policy for Rare Diseases (NPRD 2021), citing the urgent need for access to expensive disease-modifying therapies, long-term care, and financial support.
The demand was raised during the ‘Huntington’s Disease Awareness and Meeting’ organised jointly by the Huntington Disease Society of India and National Institute of Mental Health and Neurosciences at the campus of CSIR-Institute of Genomics and Integrative Biology.
What is Huntington’s Disease?
Huntington’s Disease is a rare genetic disorder that gradually damages nerve cells in the brain, affecting movement, cognitive function, and mental health. Symptoms usually begin between the ages of 30 and 40 and worsen over time.
Patients may experience involuntary movements, difficulty in coordination, depression, psychiatric symptoms, memory decline, and loss of cognitive abilities.
Around One Lakh Indians May Be Affected
According to Dr Sanjeev Jain, emeritus senior professor of psychiatry at NIMHANS, the disease may affect nearly one lakh people in India.
“The disease affects around five in one lakh people of European or Eurasian origin, which means about one lakh people in India could be affected,” he said.
He added that because the disorder is inherited, another three to five lakh individuals could be at risk due to genetic links within families.
Demand for Inclusion Under Rare Disease Policy
Chairman of the Huntington Disease Society of India, Dr Venkateswara Rao Koushik, shared details of a dossier submitted to the Ministry of Health and Family Welfare on December 26, 2025.
The letter requested the inclusion of Huntington’s disease under NPRD Group 2 and/or Group 3B categories, arguing that the condition requires lifelong multidisciplinary care and may soon need access to advanced gene therapies.
“The condition is already recognised as a rare disease outside India, but not here. Without official recognition, authorities are unable to extend support,” Koushik said.
Families Share Emotional Burden of the Disease
Amrit Bhasin, whose mother died due to the disease, described how symptoms initially appeared as tingling sensations before progressing to severe cognitive decline, loss of body coordination, and depression.
Experts highlighted that the disease not only impacts patients physically and mentally but also places a major emotional and financial burden on families and caregivers.
Call for National Patient Registry
Dr Archana Seahwag stressed the need for a national-level patient registry.
“We need to make invisible patients visible to the ministry. A registry can help trigger systemic action, including funding and healthcare support,” she said.
Gene Therapy Shows Promise in Clinical Trials
Hope for patients has emerged through a new gene therapy called AMT-130, developed by uniQure.
Results from Phase 1 and 2 clinical trials released in September 2025 reportedly showed that the therapy slowed disease progression by nearly 75 percent.
The treatment works by silencing the huntingtin gene responsible for producing the harmful mutant protein linked to the disease. The therapy is administered through a one-time brain surgery.
Dr Jain described the development as a “major breakthrough” and said Indian scientists and clinicians could potentially adapt the technology for local use through collaborative research.
No Cure Yet, But Research Continues
Currently, there is no cure for Huntington’s disease. Treatment mainly focuses on managing symptoms such as depression, compulsive behaviour, psychosis, and movement disorders.
Researchers worldwide continue to work on advanced therapies aimed at slowing or stopping the progression of the disease, offering fresh hope to affected families.
