Totally endoscopic concomitant aortic and mitral valve surgery in junctional epidermolysis bullosa: a case report
Junctional epidermolysis bullosa is a rare skin and mucosal disorder characterized by blister formation in response to minor trauma and extracutaneous manifestations. There have been no reports of cardiac surg... (Source: Journal of Cardiothoracic Surgery)
Source: Journal of Cardiothoracic Surgery - February 20, 2024 Category: Cardiovascular & Thoracic Surgery Authors: Kazufumi Yoshida, Soshi Yoshida, Yoshimasa Hori, Hideki Tsubota, Ryosuke Mochizuki, Tohru Nagano and Tadaaki Koyama Tags: Case Report Source Type: research
Recent and anticipated novel drug approvals for 2024
CONCLUSION: More oncology agents, including gene therapies, oral agents, and monoclonal antibodies, are in the pipeline this year. Additional diseases targeted by new novel drugs, including cellular and gene therapies, are hemophilia, nonalcoholic steatohepatitis, Alzheimer's disease, and rare diseases such as galactosemia and epidermolysis bullosa.PMID:38373160 | DOI:10.1093/ajhp/zxae046 (Source: American Journal of Health-System Pharmacy : AJHP)
Source: American Journal of Health-System Pharmacy : AJHP - February 19, 2024 Category: Drugs & Pharmacology Authors: Matthew H Rim Brittany L Karas Farah Barada Andrew M Levitsky Source Type: research
Recent and anticipated novel drug approvals for 2024
CONCLUSION: More oncology agents, including gene therapies, oral agents, and monoclonal antibodies, are in the pipeline this year. Additional diseases targeted by new novel drugs, including cellular and gene therapies, are hemophilia, nonalcoholic steatohepatitis, Alzheimer's disease, and rare diseases such as galactosemia and epidermolysis bullosa.PMID:38373160 | DOI:10.1093/ajhp/zxae046 (Source: American Journal of Health-System Pharmacy : AJHP)
Source: American Journal of Health-System Pharmacy : AJHP - February 19, 2024 Category: Drugs & Pharmacology Authors: Matthew H Rim Brittany L Karas Farah Barada Andrew M Levitsky Source Type: research
Intravenous gentamicin therapy induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients: An open label clinical trial
CONCLUSIONS: IV gentamicin induced readthrough of nonsense mutations in RDEB patients and restored functional C7 in their skin, enhanced wound healing, and improved clinical parameters. IV gentamicin may be a safe, efficacious, low cost, and readily available therapy in this population of RDEB patients.TRIAL REGISTRATION: Clinicaltrials.gov Identifiers: NCT03392909.PMID:38366625 | DOI:10.1093/bjd/ljae063 (Source: The British Journal of Dermatology)
Source: The British Journal of Dermatology - February 17, 2024 Category: Dermatology Authors: David T Woodley Michelle Hao Andrew Kwong Brandon Levian Jon Cogan Yingping Hou Daniel Mosallaei Elana Kleinman Kate Zheng Claire Chung Gene Kim David Peng Mei Chen Source Type: research
Intravenous gentamicin therapy induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients: An open label clinical trial
CONCLUSIONS: IV gentamicin induced readthrough of nonsense mutations in RDEB patients and restored functional C7 in their skin, enhanced wound healing, and improved clinical parameters. IV gentamicin may be a safe, efficacious, low cost, and readily available therapy in this population of RDEB patients.TRIAL REGISTRATION: Clinicaltrials.gov Identifiers: NCT03392909.PMID:38366625 | DOI:10.1093/bjd/ljae063 (Source: The British Journal of Dermatology)
Source: The British Journal of Dermatology - February 17, 2024 Category: Dermatology Authors: David T Woodley Michelle Hao Andrew Kwong Brandon Levian Jon Cogan Yingping Hou Daniel Mosallaei Elana Kleinman Kate Zheng Claire Chung Gene Kim David Peng Mei Chen Source Type: research
Intravenous gentamicin therapy induces functional type VII collagen in recessive dystrophic epidermolysis bullosa patients: An open label clinical trial
CONCLUSIONS: IV gentamicin induced readthrough of nonsense mutations in RDEB patients and restored functional C7 in their skin, enhanced wound healing, and improved clinical parameters. IV gentamicin may be a safe, efficacious, low cost, and readily available therapy in this population of RDEB patients.TRIAL REGISTRATION: Clinicaltrials.gov Identifiers: NCT03392909.PMID:38366625 | DOI:10.1093/bjd/ljae063 (Source: The British Journal of Dermatology)
Source: The British Journal of Dermatology - February 17, 2024 Category: Dermatology Authors: David T Woodley Michelle Hao Andrew Kwong Brandon Levian Jon Cogan Yingping Hou Daniel Mosallaei Elana Kleinman Kate Zheng Claire Chung Gene Kim David Peng Mei Chen Source Type: research
