After a tense stand-off with Boston Children’s Hospital, a Massachusetts family is finally getting to use a drug that could potentially save their daughter’s life.
Six-month-old Arianna Condon, of Peabody, is on the verge of paralysis due to a debilitating genetic disorder called Type 1 Spinal Muscular Atrophy affecting one in 10,000 babies.
“She may not be there next month, she might get sick,” said Arianna's mother, Marina Condon. “And she may not make it.”
The disease robs motor function and is also the leading cause of genetic deaths in infants.
By the beginning of next year, the Food and Drug Administration could approve a treatment called Spinraza, made by Cambridge based Biogen in collaboration with Ionis Pharmaceuticals. So far, the results are astounding.
Boston’s Children’s Hospital conducted clinical trials, and is one of the few sites in the nation being allowed to treat patients with this new therapy.
But for nearly a month, hospital leaders denied the drug to the Condon’s, saying it needed to go through an institutional review board, a routine checks and balances for patient safety.
Condon’s own doctors said the later that the infant get the treatment, it may be very difficult to back peddle and regenerate those neurons, according to Marina Condon.
But on Monday, the family received word that the promising treatment will be available to them and other families as well.
“You can imagine our excitement as to what this drug could do,” said Arianna's father, Adam Condon.
After the tense back and forth with the hospital, Arianna's parents are wondering if their daughter lost precious time waiting for a drug, available in their back yard.
“It’s the red tape that they got to be able to break through real quick. They can’t be so scared to bring a drug,” Adam Condon said.
The SMA treatment is still investigational, meaning, parents who want to enroll their children in the program, need to be approved by doctors.