Every week for several years, Ms. Bueso has received intravenous infusions of the replacement enzyme that treats her disease, Mucopolysaccharidosis VI, or MPS-6, which causes dwarfism, clouded vision and spinal cord compression, among other abnormalities.
“Stopping this therapy will dramatically shorten her life span,” said Paul Harmatz, the pediatric gastroenterologist who was involved in the original trial and has been treating Ms. Bueso since 2003 at the U.C.S.F. Benioff Children’s Hospital in Oakland, Calif.
The clinical trial had struggled to find patients, and without Ms. Bueso’s participation, Dr. Harmatz said, it would not have taken place. The breakthrough that came from the trial has helped people with the disease live longer than 30 years, he said. Before the drug, they rarely survived past 20.
Across the country, doctors and immigration lawyers scrambled to understand the new policy and its consequences. In Miami, it meant possibly keeping an 8-year-old girl with nerve cancer from participating in an experimental treatment at Memorial Sloan Kettering Cancer Center in New York. Her father, who is in the country illegally, is the only parent available who can travel with her, said Tammy Fox-Isicoff, a Miami immigration lawyer who is representing the family.
Without deferred action, the man cannot legally drive or board an airplane from Miami to New York, where the girl must go each month for the treatment.
In a statement, Boston Medical Center, a research hospital in a city that is a hub for clinical trials and innovative treatments, said it was “deeply concerned” about the new policy and how it would affect its patients who are receiving treatment for “extremely serious medical conditions.”
“We oppose any actions that could prevent people from accessing the health care they need,” the hospital said.