2 hrs ago
Family Seeks Access to Rare Disease Drug for Toddler
Arav is a 20-month-old child in India with a very rare genetic disease.
The disease makes it difficult for thyroid hormones to reach his brain.
This can affect movement, muscle strength, and development.
A medicine called Triac may help reduce some harmful effects of the condition.
The medicine is not officially available in India.
Arav’s family is bringing it from Germany, but it costs about Rs 3 lakh every month.
His mother and doctor are asking the government to help make the medicine easier and cheaper to obtain.
They say quick treatment is important because young children’s brains develop rapidly.
Twenty-month-old Arav has Allan-Herndon-Dudley Syndrome, also known as MCT8 deficiency, a rare genetic disorder.
Doctors estimate that Arav is among only three or four known cases in India.
Triac, the medicine studied for MCT8 deficiency, is not available in India and is reportedly awaiting FDA approval.
Arav’s family is obtaining the drug through intermediaries in Germany at a cost of about Rs 3 lakh per month.
Arav’s mother and pediatrician are urging the Government of India to reduce regulatory barriers and provide affordable access.
- Who
- Twenty-month-old Arav, his mother Pavitra Sirolikar, and pediatrician Dr Sivaranjani Santosh are seeking help from the Government of India.
- What
- They are requesting access to Triac, a medicine for Allan-Herndon-Dudley Syndrome, also called MCT8 deficiency.
- Where
- Arav lives in India, while his family currently obtains the medicine through intermediaries in Germany.
- When
- Arav was diagnosed earlier this year, after developmental concerns began around seven months of age.
- Why
- Triac is not available in India, and the family says the current import and regulatory barriers make lifelong treatment unaffordable and difficult to obtain.
Key facts
- Patient
- Arav, aged 20 months
- Condition
- Allan-Herndon-Dudley Syndrome, also known as MCT8 deficiency
- Known Indian cases
- Approximately three to four, according to Arav’s pediatrician
- Medicine
- Tiratricol, also called Triac
- Current monthly cost
- About Rs 3 lakh through intermediaries in Germany
- Government request
- Affordable access, reduced regulatory barriers, and support through rare-disease programs
Quotes
Dr Sivaranjani Santosh
Arav’s pediatrician in Hyderabad, advocating access to Triac and broader rare-disease support.
“If the government could help make the medicine available in India, it would be a big relief. In the future, if the medicine could be made available in India at a lower cost and with fewer cross-border formalities, it would help families like ours greatly.”
firstpost.com
“The trial for the medicine has finished; it is waiting for FDA approval but is available for compassionate use in many countries. However, not in India.”
firstpost.com
Sources
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