Ultrarare disease sufferers and advocates stage a mock funeral at FDA headquarters to grieve the lack of approved treatments for conditions that they say are killing their children.
Lilly says its investigational type 2 diabetes triple agonist retatrutide met its key primary and secondary endpoints in a Phase 3 trial.
Ultrarare disease sufferers and advocates stage a mock funeral at FDA headquarters to grieve the lack of approved treatments for conditions that they say are killing their children.
Lilly says its investigational type 2 diabetes triple agonist retatrutide met its key primary and secondary endpoints in a Phase 3 trial.
Two Mintz attorneys say a CDRH Warning Letter to a medical device sponsor of clinical research and compassionate use protocols may be the first time the agency has rejected the new right-to-try law as justification for delivering unapproved devices to hospitals without agency approval.
Sarepta says it will submit to FDA by the end of April sNDAs to convert the accelerated approvals of its Amondys 45 and Vyondys 53 Duchenne muscular dystrophy treatments to traditional approvals.
