Family carves out its own path in fight against 4-year-old’s rare disease

Excerpted from The Washington Post
By Robert Gebelhoff
September 7, 2015

“Arturo Estopiñan stands at his son’s bed, wearing a surgical mask and blue gloves. The boy, who has an extremely rare genetic disease that destroys muscles, has been unable to move or speak for three years.
 
Months after the diagnosis, the family contacted Michio Hirano, a New York neurologist at Columbia University Medical Center experimenting with a new medication, deoxynucleotide monophosphate, that significantly extended the life of mice that had been given the mutated gene. [read more]