ER-004, a breakthrough therapy for the prenatal treatment of XLHED, a life-threatening rare genetic condition was developed by EspeRare.
Breakthrough Designation was obtained by the promising results from three XLHED subjects with a course of ER-004 intra-amniotic injections during the third trimester of pregnancy.Hence results obtained from this prenatal treatment have a profound and life-changing effect on these infants.
Rare diseases such as XLHED often don't receive much attention and research investments that patients and their families deserve.This breakthrough therapy approach has the potential to fundamentally change the lives of patients and may also pave the way for other prenatal treatments to correct genetic diseases before birth.
US FDA grants Breakthrough Therapy Designation for Investigational Prenatal Treatment ER-004 in X-Linked Hypohidrotic Ectodermal Dysplasia (XLHED)protein replacement therapy for the treatment for the prenatal treatment of XLHED, a life-threatening rare genetic condition.
