June 25, 2022

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In India, Parents of Children with Rare Disease Plea for Help Online

2 min read

ELURU, India — When her baby started struggling to breathe, Stella Praveen had a terrible feeling that something was gravely wrong with her 14-month-old daughter, Ellen.

She ran barefoot to a nearby clinic, but the doctors there said the child needed to see a specialist right away. Without an ambulance, she jumped on the back of a motorcycle and rode 35 miles to a children’s hospital in another town, where Ellen remained in intensive care for 12 days.

Two weeks later, Ms. Praveen learned that her daughter, who had never been able to lift her neck nor roll over, was suffering from spinal muscular atrophy, a rare condition often fatal by age 2.

“We had not even heard of this disease,” Ms. Praveen said as tears rolled down her face. “She was misdiagnosed many times.”

The Praveen family was momentarily heartened when it learned that a promising gene therapy treatment was available, but was quickly crestfallen when it heard the cost: $2.1 million.

In India, and in many of the world’s poorer countries, the pharmaceutical industry’s latest advances for rare diseases are often agonizingly out of reach, impossible for almost all but the wealthiest families to afford and not covered by health insurance.

In desperation — and encouraged by the occasional success story — families are turning to social media to raise funds.

Every morning, Ellen’s father, Rayapudi Praveen, sends hundreds of emails on five crowdfunding websites like ImpactGuru and GoFundMe, asking people to contribute money to save his daughter’s life.

“Dear Sir, my daughter Ellen is suffering,” every email begins. “Can you help us?”

With only four months left before Ellen turns 2, time is running out — and the family is still far from its goal.

Spinal muscular atrophy is an inherited neuromuscular disease that kills more infants worldwide than any other genetic disorder. In India, one study put its prevalence at one in every 7,744 live births, or about 3,200 Indian babies each year.

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