Please Save My Child: Drug Company and Compassionate Use

Today is Father’s Day and if a drug company refused to save my daughter’s life, I imagine I would have also writen a strongly worded Change.Org Petition, like Radha Rangarajan’s mother, Sonal Patel:

On the other hand, the Shire enzyme being developed for metachromatic leukodystrophy (known as SHP611 or HGT1110) has no published data that support its effectiveness. A study of 24 patients appears to have been completed in January 2017, however, as of June 2018, the results have not been disclosed by Shire.

Why is it taking more than a year after study completion to release the results of the SHP611 study? Considering it is a study of only 24 patients, what is the reason for the delay?

Did Shire decline to give SHP611 to Radha because SHP611 did not work?

Did Shire decline because they are heartless and lack compassion?

The 24 patients who volunteered for the Shire SHP611 / HGT-1110 study as well as patients like Radha and their families deserve to know what is going on. In the absence of official word from Shire, we can only speculate and suspect the worst. This may not be fair to Shire but it is the consequence of their silence.