MF4, SCD when in sickle cell crisis provides an opportunity to move RD forward, not a new disease indication. As an orphan indication it would also move forward at lower cost and lower regulatory burden.
Think of sticking your hand into boiling water and having the ER physicians feel ambivalent in giving pain relief because your breathing might slow. The indication is not SCD, it is the RD created in trying to control the worst possible pain. Many of those requiring treatment are not just children, but adults. Success in the orphan indication will allow testing in postoperative pain and routine use in the recovery room where patients with central sleep apnea would also be slow to resume breathing.
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