If our boy is not a candidate for a transplant, what other options exist?

Unfortunately, once the “transplant” window has closed, there are few other treatment options available. A preliminary study at University of Minnesota is demonstrating promising results in a small number of patients with advanced disease (please see “on the Horizon” section). Other treatments are palliative care—in other words, they cannot alter the course of the disease, but only make life as comfortable as possible for the patient and his family.