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Archive for category: Congenital Diseases

Salivary vesicles found to activate blood clotting in haemophilia A

, 27 January 2025/in Congenital Diseases, E-News, Research /by panglobal

New research from the Medical University of Vienna has identified a mechanism explaining why people with haemophilia A rarely experience oral bleeding, despite their blood clotting deficiency. The finding could have implications for understanding bleeding patterns in different coagulation disorders.  

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https://interhospi.com/wp-content/uploads/sites/3/2025/01/hemophilia.jpg 730 1096 panglobal https://interhospi.com/wp-content/uploads/sites/3/2020/06/Component-6-–-1.png panglobal2025-01-27 08:00:212025-01-22 12:38:18Salivary vesicles found to activate blood clotting in haemophilia A
gene therapy

New RNA technology enables gene therapy for previously untreatable muscular dystrophies

gene therapy, muscular dystrophy, RNA, 26 November 2024/in Congenital Diseases, E-News, Genetics /by panglobal

A groundbreaking RNA-based technology allows therapeutic genes that are too large for conventional delivery methods to be successfully transported into cells, offering new hope for treating muscular dystrophies and other genetic disorders caused by mutations in large genes.  

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https://interhospi.com/wp-content/uploads/sites/3/2024/11/gene_therapy.jpg 1357 1437 panglobal https://interhospi.com/wp-content/uploads/sites/3/2020/06/Component-6-–-1.png panglobal2024-11-26 08:00:102024-11-19 11:56:11New RNA technology enables gene therapy for previously untreatable muscular dystrophies

Single-dose gene therapy dramatically reduces bleeds in haemophilia B patients

FDA, gene therapy, haemophilia B, Research, 26 September 2024/in Congenital Diseases, E-News /by panglobal

A single-dose gene therapy has demonstrated significant reduction in bleeding episodes for adults with haemophilia B, potentially offering a long-term solution to this genetic disorder.  

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https://interhospi.com/wp-content/uploads/sites/3/2024/09/blood25.jpg 720 960 panglobal https://interhospi.com/wp-content/uploads/sites/3/2020/06/Component-6-–-1.png panglobal2024-09-26 01:00:122024-09-25 13:03:21Single-dose gene therapy dramatically reduces bleeds in haemophilia B patients
Jeffrey Chamberlain and Hichem TasfaoutChamberlain Lab

New gene therapy breakthrough offers hope for Duchenne muscular dystrophy patients

Duchenne muscular dystrophy, Genetics, Nature, treatment, 19 July 2024/in Congenital Diseases, E-News, Genetics /by panglobal

A groundbreaking gene therapy for Duchenne muscular dystrophy (DMD) has demonstrated significant potential in not only arresting muscle decline but also possibly repairing damaged muscle tissue. This novel treatment, developed by researchers at UW Medicine, utilises a series of protein packets delivered via viral vectors to replace the defective DMD gene within affected muscles.  

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https://interhospi.com/wp-content/uploads/sites/3/2024/07/Jeffrey-Chamberlain-left-and-Hichem-Tasfaout.jpg 426 640 panglobal https://interhospi.com/wp-content/uploads/sites/3/2020/06/Component-6-–-1.png panglobal2024-07-19 13:29:132024-07-19 13:29:13New gene therapy breakthrough offers hope for Duchenne muscular dystrophy patients

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