As researchers seek a cure for the rare bone disease called FOP, fund-raisers are also hard at work.
Money from the National Institutes of Health covers less than a quarter of the University of Pennsylvania laboratory's $1 million-plus annual costs. The rest comes mostly from families who organize all kinds of events.
On a farm in Harestone, Scotland, 6-year-old Cameron Barclay and his parents have hosted three barn dances, moving their 70 cows into the fields for the occasion.
"We hosed down the barn and laid down a wooden dance floor," says his mother, June Barclay.
Despite his FOP, she said, Cameron "takes his little pedal John Deere tractor out."
And he helped with the balloons, while neighbors and friends pitched a tent as a "casino" for roulette, blackjack and dice, moving small wooden racehorses along with bets.
Across the Atlantic, a fund-raiser at New York's Waldorf-Astoria hotel earlier this year was called "Songs for Sophia." The evening featured tenor Placido Domingo, mezzo-soprano Frederica von Stade, baritone Bryn Terfel and other singers from New York's Metropolitan Opera, including soprano Constance Green, Sophia Forshtay's mother.
"It's very important to find a solution quickly. We are here singing for love," Domingo said at the benefit.
After Sophia was diagnosed, "Placido asked me, 'What can I do?' " Green said.
What he did was to lead a program of Broadway and opera tunes delivered by some of the world's finest voices.
Sophia romped around the golden Waldorf ballroom, a waif in a pink silk gown daring to tell Domingo as he hummed her a song, "No singing!"