The last couple of weeks has been particularly distressing for Duchenne muscular dystrophy patients and their families. The fate of Sarpeta’s gene therapy for the disease, called Elevidys, has been the center of a dizzying saga that seems to change by the minute.

Sarepta's therapy has been under scrutiny from regulators after two teenage boys died earlier this year from acute liver injury, a known side effect of the treatment. The FDA then requested a pause in shipments of the drug after the death of a third patient taking a different Sarepta therapy.

The Food and Drug Administration has paused distribution of the gene therapy Elevidys following the death of a third patient receiving treatment for muscular dystrophy. Alison and William Small are urging the agency to restore access to the drug,