First-of-its-kind gene ther­a­py ap­proved for heal­ing wounds in “but­ter­fly chil­dren”

Chil­dren born with a rare dis­ease that makes their skin as frag­ile as a but­ter­fly’s wings, leav­ing them vul­ner­a­ble to fre­quent wound­ing and con­stant pain, will fi­nal­ly have ac­cess to a new treat­ment that aims to fix the ge­net­ic cause of the con­di­tion.

The ther­a­py, known as Vyju­vek and made by Krys­tal Biotech, was ap­proved by the FDA on Fri­day af­ter­noon for treat­ing pa­tients six months or old­er with dy­s­troph­ic epi­der­mol­y­sis bul­losa, or DEB. The drug is a long-await­ed so­lu­tion for many fam­i­lies af­flict­ed by the hell­ish con­di­tion, for which there are no ap­proved treat­ments in the US.

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