Publications
8 February 2024

Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa

N Engl J Med. 2024 Feb 8;390(6):530-535. doi: 10.1056/NEJMoa2301244. PMID: 38324486. Ocular Gene Therapy in a Patient with Dystrophic Epidermolysis Bullosa Authors: Arianna Tovar Vetencourt 1, Ibrahim Sayed-Ahmed 1, Jennifer […]
27 February 2024

Trends in orphan medicinal products approvals in the European Union between 2010–2022

Orphanet J Rare Dis. 2024 Feb 27;19(1):91. doi: 10.1186/s13023-024-03095-z. PMID: 38413985. Trends in orphan medicinal products approvals in the European Union between 2010–2022 Authors: Luísa Bouwman 1 2, Bruno […]
14 March 2024

Regenerative Therapy for Corneal Scarring Disorders

Biomedicines. 2024; 12(3):649. https://doi.org/10.3390/biomedicines12030649 Regenerative Therapy for Corneal Scarring Disorders Authors: Christine Chandran 1,Mithun Santra 1,Elizabeth Rubin 1,Moira L. Geary 1 andGary Hin-Fai Yam 1,2 Affiliation: Abstract: The cornea is a transparent and […]
20 March 2024

Orofacial Anomalies in Kindler Epidermolysis Bullosa

JAMA Dermatol. 2024 Mar 20:e240065. doi: 10.1001/jamadermatol.2024.0065. Epub ahead of print. PMID: 38506824; PMCID: PMC10955352. Orofacial Anomalies in Kindler Epidermolysis Bullosa Authors: Susanne Krämer 1 2, Anna Lena Hillebrecht 3, Yao […]
24 March 2024

Challenges and progress related to gene editing in rare skin diseases

Adv Drug Deliv Rev. 2024 Mar 23:115294. doi: 10.1016/j.addr.2024.115294. Epub ahead of print. PMID: 38527624. Challenges and progress related to gene editing in rare skin diseases […]
9 April 2024

Inpatient Management of Epidermolysis Bullosa: Consensus-Based Hands-On Instructions for Neonates and Post-Neonates.

J Am Acad Dermatol. 2024 Apr 9:S0190-9622(24)00583-8. doi: 10.1016/j.jaad.2024.04.014. Epub ahead of print. PMID: 38604489. Inpatient Management of Epidermolysis Bullosa: Consensus-Based Hands-On Instructions for Neonates and […]
12 April 2024

Exploring stem cell frontiers: definitions, challenges, and perspectives for regenerative medicine

Biol Open. 2024 Apr 15;13(4):bio060245. doi: 10.1242/bio.060245. Epub 2024 Apr 9. PMID: 38592154 Exploring stem cell frontiers: definitions, challenges, and perspectives for regenerative medicine Authors: Miriana […]