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Mashup Score: 0Fitusiran in haemophilia: a breakthrough drug with many unknowns - 1 year(s) ago
Haemophilia A, defined by factor VIII (FVIII) deficiency, and haemophilia B, defined by factor IX (FIX) deficiency, are X chromosome linked diseases characterised by reduced thrombin generation and bleeding symptoms.1 Prophylaxis with replacement FVIII and FIX is considered the gold standard in haemophilia treatment. Over the past two decades, the treatment landscape in haemophilia has changed…
Source: The LancetCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0Fitusiran prophylaxis in severe haemophilia without inhibitors - 1 year(s) ago
Haemophilia is a congenital X-linked bleeding disorder caused by deficiency of coagulation factors VIII (haemophilia A) or IX (haemophilia B). Recurrent joint bleeding, leading to the development of arthropathy, is the hallmark of haemophilia and is more pronounced in people with moderate to severe forms of the disease (<1–5% of normal factor VIII or IX concentrations).1 Prophylaxis is the...
Source: The Lancet HaematologyCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0Alok Srivastava on fitusiran for patients with haemophilia A and B - In conversation with... - 1 year(s) ago
Professor Alok Srivastava tells us about two clinical trials being published in The Lancet and The Lancet Haematology on a new siRNA therapy called fitusiran for patients with haemophilia A and B.Read the two trials:Efficacy and safety of fitusira…
Source: BuzzsproutCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0Alok Srivastava on fitusiran for patients with haemophilia A and B - In conversation with... - 1 year(s) ago
Professor Alok Srivastava tells us about two clinical trials being published in The Lancet and The Lancet Haematology on a new siRNA therapy called fitusiran for patients with haemophilia A and B.Read the two trials:Efficacy and safety of fitusira…
Source: BuzzsproutCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0
In participants with haemophilia A or B without inhibitors, fitusiran prophylaxis resulted in significant reductions in annualised bleeding rate compared with on-demand clotting factor concentrates and no bleeding events in approximately half of participants. Fitusiran prophylaxis shows haemostatic efficacy in both haemophilia A and haemophilia B, and therefore has the potential to be…
Source: The Lancet HaematologyCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0Fitusiran in haemophilia: a breakthrough drug with many unknowns - 1 year(s) ago
Haemophilia A, defined by factor VIII (FVIII) deficiency, and haemophilia B, defined by factor IX (FIX) deficiency, are X chromosome linked diseases characterised by reduced thrombin generation and bleeding symptoms.1 Prophylaxis with replacement FVIII and FIX is considered the gold standard in haemophilia treatment. Over the past two decades, the treatment landscape in haemophilia has changed…
Source: The LancetCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0Fitusiran prophylaxis in severe haemophilia without inhibitors - 1 year(s) ago
Haemophilia is a congenital X-linked bleeding disorder caused by deficiency of coagulation factors VIII (haemophilia A) or IX (haemophilia B). Recurrent joint bleeding, leading to the development of arthropathy, is the hallmark of haemophilia and is more pronounced in people with moderate to severe forms of the disease (<1–5% of normal factor VIII or IX concentrations).1 Prophylaxis is the...
Source: The Lancet HaematologyCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 1Haemophilia—unparalleled progress but inadequate access - 1 year(s) ago
Haemophilia is an inherited bleeding disease estimated to affect 818 928 people worldwide in 2021. The condition can be life-threatening if an external bleed cannot be controlled or when there is an internal bleed to a vital organ. Internal bleeding around joints is the most common complication for patients, which can result in major disability and mobility issues, reducing patients’ quality of…
Source: The Lancet HaematologyCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0Integrated Interprofessional Pathway for Gene Therapy: A Model to Optimize Care in Hemophilia - 1 year(s) ago
How can your institution prepare to offer gene therapy for patients with hemophilia?
Source: MedscapeCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
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Mashup Score: 0Integrated Interprofessional Pathway for Gene Therapy: A Model to Optimize Care in Hemophilia - 1 year(s) ago
How can your institution prepare to offer gene therapy for patients with hemophilia?
Source: MedscapeCategories: Hem/Onc News and Journals, Latest HeadlinesTweet
"Fitusiran might be the first prophylactic option for people with haemophilia B and perhaps an alternative approach... in haemophilia A" Comment @TheLancet by @flora_peyvandi, Isabella Garagiola & Maria Abbattista #bleedingdisorders #hemophilia https://t.co/ic1QgkpCt2