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2023 28
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Beremagene Geperpavec: First Approval.
Dhillon S. Dhillon S. Drugs. 2023 Aug;83(12):1131-1135. doi: 10.1007/s40265-023-01921-5. Drugs. 2023. PMID: 37432558 Review.
In May 2023, beremagene geperpavec received its first approval in the US for the treatment of wounds in patients 6 months of age with dystrophic epidermolysis bullosa with mutation(s) in the COL7A1 gene. A Marketing Authorization Application for beremagene …
In May 2023, beremagene geperpavec received its first approval in the US for the treatment of wounds in patients 6 months of age with d
Long-Term Safety and Tolerability of Beremagene Geperpavec-svdt (B-VEC) in an Open-Label Extension Study of Patients with Dystrophic Epidermolysis Bullosa.
Marinkovich MP, Paller AS, Guide SV, Gonzalez ME, Lucky AW, Bağcı IS, Agostini B, Fitzgerald K, Chen S, Chen H, Conner MM, Krishnan SM. Marinkovich MP, et al. Am J Clin Dermatol. 2025 Jul;26(4):623-635. doi: 10.1007/s40257-025-00942-y. Epub 2025 Apr 12. Am J Clin Dermatol. 2025. PMID: 40220208 Free PMC article. Clinical Trial.
BACKGROUND: Patients with dystrophic epidermolysis bullosa have pathogenic variants in COL7A1, leading to skin fragility. Beremagene geperpavec-svdt (B-VEC) is a modified, herpes simplex virus type 1-based gene therapy vector that topically delivers COL7A1 to …
BACKGROUND: Patients with dystrophic epidermolysis bullosa have pathogenic variants in COL7A1, leading to skin fragilit …
Prademagene zamikeracel for recessive dystrophic epidermolysis bullosa wounds (VIITAL): a two-centre, randomised, open-label, intrapatient-controlled phase 3 trial.
Tang JY, Marinkovich MP, Wiss K, McCarthy D, Truesdale A, Chiou AS, Eid E, McIntyre JK, Bailey I, Furukawa LK, Gorell ES, Harris N, Khosla RK, Peter Lorenz H, Lu Y, Nazaroff J, Grachev ID, Moore AJ. Tang JY, et al. Lancet. 2025 Jul 12;406(10499):163-173. doi: 10.1016/S0140-6736(25)00778-0. Epub 2025 Jun 23. Lancet. 2025. PMID: 40570869 Clinical Trial.
BACKGROUND: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genetic skin disease caused by mutations in the COL7A1 gene encoding type VII collagen. ...
BACKGROUND: Recessive dystrophic epidermolysis bullosa (RDEB) is a rare genetic skin disease caused by mutations in the …
Type VII Collagen Disorders Simplified.
Camisa C. Camisa C. Cutis. 2025 Aug;116(2):205-209, 214, E3-E4. doi: 10.12788/cutis.1267. Cutis. 2025. PMID: 41056121 Review.
The functionality of type VII collagen is vital to the skin and mucous membranes because it makes up the anchoring fibrils that adhere the epithelium to the underlying connective tissue. Dystrophic epidermolysis bullosa (DEB), which bears some clinical simila …
The functionality of type VII collagen is vital to the skin and mucous membranes because it makes up the anchoring fibrils that adhere the e …
Dystrophic epidermolysis bullosa - From biochemistry to interventions.
Nyström A. Nyström A. Matrix Biol. 2025 Apr;136:111-126. doi: 10.1016/j.matbio.2025.02.001. Epub 2025 Feb 6. Matrix Biol. 2025. PMID: 39922469 Free article. Review.
Blistering and wider tissue fragility in the genetic disease dystrophic epidermolysis bullosa (DEB) caused by collagen VII deficiency illustrate the essential function of collagen VII in supporting skin integrity. ...
Blistering and wider tissue fragility in the genetic disease dystrophic epidermolysis bullosa (DEB) caused by collagen …
A scalable and cGMP-compatible autologous organotypic cell therapy for Dystrophic Epidermolysis Bullosa.
Neumayer G, Torkelson JL, Li S, McCarthy K, Zhen HH, Vangipuram M, Mader MM, Gebeyehu G, Jaouni TM, Jacków-Malinowska J, Rami A, Hansen C, Guo Z, Gaddam S, Tate KM, Pappalardo A, Li L, Chow GM, Roy KR, Nguyen TM, Tanabe K, McGrath PS, Cramer A, Bruckner A, Bilousova G, Roop D, Tang JY, Christiano A, Steinmetz LM, Wernig M, Oro AE. Neumayer G, et al. Nat Commun. 2024 Jul 11;15(1):5834. doi: 10.1038/s41467-024-49400-z. Nat Commun. 2024. PMID: 38992003 Free PMC article.
We present Dystrophic Epidermolysis Bullosa Cell Therapy (DEBCT), a scalable platform producing autologous organotypic iPS cell-derived induced skin composite (iSC) grafts for definitive treatment. ...
We present Dystrophic Epidermolysis Bullosa Cell Therapy (DEBCT), a scalable platform producing autologous organotypic …
Dipeptidyl Peptidase-4-Mediated Fibronectin Processing Evokes a Profibrotic Extracellular Matrix.
Zeyer KA, Bornert O, Nelea V, Bao X, Leytens A, Sharoyan S, Sengle G, Antonyan A, Bruckner-Tuderman L, Dengjel J, Reinhardt DP, Nyström A. Zeyer KA, et al. J Invest Dermatol. 2024 Nov;144(11):2477-2487.e13. doi: 10.1016/j.jid.2024.03.020. Epub 2024 Apr 1. J Invest Dermatol. 2024. PMID: 38570029 Free article.
To investigate this, we employed recessive dystrophic epidermolysis bullosa as a disease model with progressive, severe dermal fibrosis. Fibroblasts from donors with recessive dystrophic epidermolysis bullosa in 2-dimensional and 3-dimens …
To investigate this, we employed recessive dystrophic epidermolysis bullosa as a disease model with progressive, severe …
Herpes simplex virus gene therapy for dystrophic epidermolysis bullosa (DEB).
Epstein AL, Haag-Molkenteller C. Epstein AL, et al. Cell. 2023 Aug 17;186(17):3523-3523.e1. doi: 10.1016/j.cell.2023.07.031. Cell. 2023. PMID: 37595560 Free article.
The FDA has recently approved Krystal biotech's beremagene geperpavec (B-VEC, Vyjuvek) to treat the wounds of dystrophic epidermolysis bullosa (DEB) patients. This represents a giant step, not only toward the treatment of this devastating disease, but also fo …
The FDA has recently approved Krystal biotech's beremagene geperpavec (B-VEC, Vyjuvek) to treat the wounds of dystrophic epidermol
127 results