Articles
An evaluation of prademagene zamikeracel for the treatment of recessive dystrophic epidermolysis bullosa
Expert Opin Biol Ther. 2026 Mar 18:1-6. doi: 10.1080/14712598.2026.2639393. Epub ahead of print. PMID: 41764660. An evaluation of prademagene zamikeracel for the treatment of recessive dystrophic […]Developing CRISPR-Based Therapies for Epidermolysis Bullosa: A Comprehensive Review of Current Strategies
Drugs. 2026 Feb 23. doi: 10.1007/s40265-026-02285-2. Epub ahead of print. PMID: 41731282. Developing CRISPR-Based Therapies for Epidermolysis Bullosa: A Comprehensive Review of Current Strategies Authors: du […]Challenges and solutions to the sustainability of gene and cell therapies
Nat Rev Genet. 2025 Mar 10. doi: 10.1038/s41576-025-00827-0. Epub ahead of print. PMID: 40065154. Challenges and solutions to the sustainability of gene and cell therapies Authors: […]Overcoming the “valleys of death” in advanced therapies: The role of finance
Social Science & Medicine 366 (2025) 117639 Overcoming the “valleys of death” in advanced therapies: The role of finance Authors: Simona Cosma1, Stefano Cosma2, Daniela Pennetta2, […]Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa
J Dermatolog Treat. 2024 Dec;35(1):2391452. doi: 10.1080/09546634.2024.2391452. Epub 2024 Aug 18. PMID: 39155053. Emerging DNA & RNA editing strategies for the treatment of epidermolysis bullosa Authors: […]Highlights of Gene and Cell Therapy for Epidermolysis Bullosa and Ichthyosis
Dermatol Ther (Heidelb). 2024 Sep;14(9):2379-2392. doi: 10.1007/s13555-024-01239-4. Epub 2024 Aug 7. PMID: 39112824; PMCID: PMC11393223. Highlights of Gene and Cell Therapy for Epidermolysis Bullosa and Ichthyosis […]Treatment of Epidermolysis Bullosa and Future Directions: A Review
Dermatol Ther (Heidelb) (2024). https://doi.org/10.1007/s13555-024-01227-8 Treatment of Epidermolysis Bullosa and Future Directions: A Review Authors: Danescu S 1, Negrutiu M1, Has C2 Affiliation: Abstract: Epidermolysis bullosa (EB) […]The evolution and ongoing challenge of unproven cell-based interventions
Stem Cells Transl Med. 2024 Jul 23:szae050. doi: 10.1093/stcltm/szae050. Epub ahead of print. PMID: 39045646. The evolution and ongoing challenge of unproven cell-based interventions Authors: Taylor […]Advancing Access to Cell and Gene Therapies in Medicaid
JAMA. 2024 Jun 18;331(23):1989-1990. doi: 10.1001/jama.2024.6224. PMID: 38776090. Advancing Access to Cell and Gene Therapies in Medicaid Authors: Joseph T Kannarkat 1, Inmaculada Hernandez 2, Natasha Parekh Affiliation: Abstract: The […]Towards ethical drug pricing: the European Orphan Genomic Therapies Fund
Journal: Gene Ther. 2024 Apr 24. doi: 10.1038/s41434-024-00452-2. Epub ahead of print. PMID: 38658672. Towards ethical drug pricing: the European Orphan Genomic Therapies Fund Authors: Johanna […]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 […]US state laws on medical freedom and investigational stem cell procedures: a call to focus on state-based legislation
Cytotherapy. 2024 Feb 3:S1465-3249(24)00020-3. doi: 10.1016/j.jcyt.2024.01.002. Epub ahead of print. PMID: 38310500. US state laws on medical freedom and investigational stem cell procedures: a call to […]Costs of UK community care for individuals with recessive dystrophic epidermolysis bullosa: Findings of the Prospective Epidermolysis Bullosa Longitudinal Evaluation Study
Skin Health Dis. 2024 Jan 10;4(1):e314. doi: 10.1002/ski2.314. PMID: 38312260; PMCID: PMC10831550. Costs of UK community care for individuals with recessive dystrophic epidermolysis bullosa: Findings of […]Realising the potential of gene therapies for rare and ultra-rare inherited diseases
Hum Gene Ther. 2023 Aug 25. doi: 10.1089/hum.2023.127. Epub ahead of print. PMID: 37624746. Realising the potential of gene therapies for rare and ultra-rare inherited diseases […]Il finanziamento della ricerca clinica in Advanced Therapy Medicinal Products (ATMP): cosa determina l’intervento dellaFinanza?
Keywords: finanza, gap di finanziamento, ATMP, terapie avanzate, ricerca medica, studi clinici, PMIUnproven cell interventions in Poland and the exploitation of European Union law on advanced therapy medicinal products
Keywords: ATMP-HE; advanced therapy medicinal products; hospital exemption; stem cell therapies; stem cells; unproven cell therapies.Advanced therapy medicinal products in China: Regulation and development
MedComm (2020). 2023 Apr 26;4(3):e251. doi: 10.1002/mco2.251. PMID: 37125239; PMCID: PMC10133728. Advanced therapy medicinal products in China: Regulation and development Authors: Jiaqi Lu, 1 Longchang Xu, 1 Wei Wei, 1 and Wu He 1 […]Stairways to Advanced Therapies for Epidermolysis Bullosa
Cold Spring Harb Perspect Biol. 2023 Apr 3;15(4):a041229. doi: 10.1101/cshperspect.a041229. PMID: 36167646; PMCID: PMC10071437 Stairways to Advanced Therapies for Epidermolysis Bullosa Authors: Laura De Rosa # 1, Elena Enzo # 2, Michele […]Cost and availability of novel cell and gene therapies: Can we avoid a catastrophic second valley of death?
EMBO Rep. 2023 Feb 6;24(2):e56661. doi: 10.15252/embr.202256661. Epub 2023 Jan 2. PMID: 36592141; PMCID: PMC9900339. Cost and availability of novel cell and gene therapies: Can we […]The steep uphill path leading to ex vivo gene therapy for genodermatoses
Am J Physiol Cell Physiol. 2022 Sep 1;323(3):C896-C906. doi: 10.1152/ajpcell.00117.2022. Epub 2022 Aug 1. PMID: 35912986. The steep uphill path leading to ex vivo gene therapy […]Clonal analysis of human clonogenic keratinocytes
Methods Cell Biol. 2022;170:101-116. doi: 10.1016/bs.mcb.2022.02.009. Epub 2022 Apr 18. PMID: 35811094. Clonal analysis of human clonogenic keratinocytes Authors: Elena Enzo 1, Camilla Cattaneo 1, Federica Consiglio 2, Maria Pia Polito 1, Sergio Bondanza 2, Michele […]