pharmaphorum2y
FDA turns down Amryt's epidermolysis bullosa drug
its drug for rare and debilitating skin disease epidermolysis bullosa (EB). Shares in the Ireland-based rare disease specialist fell sharply after news of the complete response letter (CRL ...
BBC7y
'Butterfly child' given life-saving skin
Hassan, who lives in Germany, has a genetic disease - junctional epidermolysis bullosa - that leaves his ... [for each form of EB] so we need formal clinical trials." But if they can make it ...
INmune Bio treatment of epidermolysis bullosa receives FDA orphan designation
INmune Bio’s (INMB) treatment of epidermolysis bullosa received FDA orphan designation, according to a post on the agency’s site.Don't Miss Our ...
pharmaphorum2d
FDA clears Vyjuvek as first topical gene therapy
The FDA has approved Krystal Biotech’s gene therapy Vyjuvek for dystrophic epidermolysis bullosa (DEB), a rare ... who live far from specialised clinical centres. In the 31-patient GEM-3 ...
The Pharma Letter9d
City Therapeutics appoints Andrew Orth as chief exec
City Therapeutics appoints Andrew Orth as CEO, leveraging his expertise in RNA and gene therapies as the company advances its ...