Several therapies are presently in the pre-clinical development phase for ... models to stimulate Recessive Dystrophic Epidermolysis Bullosa (RDEB) cells, which cannot produce COL7, to generate ...

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 ...

and was filed last month for the additional indication of epidermolysis bullosa, a serious rare genetic disease in which minor friction causes blisters or erosion of the skin. News of a filing ...

Immunobullous diseases vary in clinical presentation and have different histopathologic and immunologic features (see Table 1). Immunofluorescence (IMF) can demonstrate the presence of antibodies ...

Some of these drugs will never hit the shelf of pharmaceuticals even after countless clinical trials. On the other hand, if a biotech company gets a new drug sanctioned by the FDA and the demand ...

Clinical development and trial testing of vesleteplirsen ... Investigators said the unique features of chimeric antigen receptor (CAR) T-cell therapy may warrant novel approaches to cost ...

Ethicist Art Caplan discusses a recent misdiagnosed organ donor candidate in Kentucky and the need to highlight safeguards when promoting organ donation.