Symptomatic cerebral cavernous malformations are a rare sporadic or familial disease, which may cause haemorrhagic strokes or epileptic seizures. In 2015, a James Lind Alliance Priority Setting Partnership ranked targeted drug therapies as fourth of the top 10 research priorities for cerebral cavernous malformations. There are no disease-modifying drugs for cerebral cavernous malformations, but there are several promising candidates with proof of concept in human and animal studies. There are no platform trials for cerebral cavernous malformations.
