Gene Therapy Makes Inroads Against a Form of Hemophilia
FRIDAY, July 22, 2022 -- People with hemophilia B could find their bleeding risk dramatically reduced with just one injection of an experimental gene therapy, a new study reports. Hemophilia B is a rare and inherited genetic disorder in which people... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - July 22, 2022 Category: General Medicine Source Type: news

Gene Therapy Makes Inroads Against a Form of Hemophilia
Title: Gene Therapy Makes Inroads Against a Form of HemophiliaCategory: Health NewsCreated: 7/22/2022 12:00:00 AMLast Editorial Review: 7/22/2022 12:00:00 AM (Source: MedicineNet Chronic Pain General)
Source: MedicineNet Chronic Pain General - July 22, 2022 Category: Anesthesiology Source Type: news

Haemophilia can be cured through 'miracle' therapy
University College London researchers say a one-off gene therapy blood transfusion could stop people with haemophilia having to take weekly injections to manage the rare condition. (Source: the Mail online | Health)
Source: the Mail online | Health - July 21, 2022 Category: Consumer Health News Source Type: news

Gene therapy trial markedly cuts bleeding risk in haemophilia B patients
Single injection of FLT180 removes need for people to inject weekly with clotting factors, study findsA new gene therapy has dramatically cut the risk of bleeding in people with the rare condition haemophilia B, experts have said.Researchersfound that a single injection of the gene therapy, called FLT180a, removed the need for people to inject themselves weekly with clotting factors.Continue reading... (Source: Guardian Unlimited Science)
Source: Guardian Unlimited Science - July 21, 2022 Category: Science Authors: PA Media Tags: Genetics Health Biology Science Research UK news Source Type: news

[Ad hoc announcement pursuant to Art. 53 LR] Roche achieves good results in the first six months of 2022
Basel, 21 July 2022Group sales up 5%1 at constant exchange rates (CER) and 5% in Swiss francsPharmaceuticals Divisionsales increase 3%; continued strong demand for new medicines to treat severe diseases; as expected, the impact of biosimilars slows down furtherDiagnostics Divisionsales grow 11%; ongoing strong base business; demand for COVID-19 tests is expected to decline in the second half of the yearIFRSnet income increases by 12% (12% in Swiss francs), whilecore earnings per share are up 11%Highlights in the second quarter:EU approvals ofPolivy (first-line treatment for aggressive form of blood cancer),Lunsumio (follic...
Source: Roche Media News - July 21, 2022 Category: Pharmaceuticals Source Type: news

[Ad hoc announcement pursuant to Art. 53 LR] Roche achieves good results in the first six months of 2022
Group sales up 5%1 at constant exchange rates (CER) and 5% in Swiss francsPharmaceuticals Divisionsales increase 3%; continued strong demand for new medicines to treat severe diseases; as expected, the impact of biosimilars slows down furtherDiagnostics Divisionsales grow 11%; ongoing strong base business; demand for COVID-19 tests is expected to decline in the second half of the yearIFRSnet income increases by 12% (12% in Swiss francs), whilecore earnings per share are up 11%Highlights in the second quarter:EU approvals ofPolivy (first-line treatment for aggressive form of blood cancer),Lunsumio (follicular lymphoma) andT...
Source: Roche Investor Update - July 21, 2022 Category: Pharmaceuticals Source Type: news

Transformational therapy cures haemophilia B
A patient on the trial says his life is now "completely normal", having grown up fearing getting hurt. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - July 20, 2022 Category: Consumer Health News Source Type: news

New data from phase III HAVEN 6 study reinforce favourable safety and efficacy profile of Roche ’s Hemlibra in people with moderate or mild haemophilia A
Hemlibra continues to demonstrate clinically meaningful bleed control, with 66.7% of participants with moderate or mildhaemophilia A experiencing zero treated bleeds at 55.6 weeks median follow-up [1]New data also reinforceHemlibra ’sfavourable safety profile, with no new safety signals observed [1]There is limited information and treatment guidance on moderate and mildhaemophilia A, which can lead to delayed or missed diagnoses of bleeding episodes [2]Hemlibra is approved to treat people of all ages withhaemophilia A with factor VIII inhibitors in more than 110 countries and for people of all ages without factor VIII in...
Source: Roche Media News - July 11, 2022 Category: Pharmaceuticals Source Type: news

New data from phase III HAVEN 6 study reinforce favourable safety and efficacy profile of Roche ’s Hemlibra in people with moderate or mild haemophilia A
Hemlibra continues to demonstrate clinically meaningful bleed control, with 66.7% of participants with moderate or mildhaemophilia A experiencing zero treated bleeds at 55.6 weeks median follow-up [1]New data also reinforceHemlibra ’sfavourable safety profile, with no new safety signals observed [1]There is limited information and treatment guidance on moderate and mildhaemophilia A, which can lead to delayed or missed diagnoses of bleeding episodes [2]Hemlibra is approved to treat people of all ages withhaemophilia A with factor VIII inhibitors in more than 110 countries and for people of all ages without factor VIII in...
Source: Roche Investor Update - July 11, 2022 Category: Pharmaceuticals Source Type: news

Could haemophilia be 'cured' by a new gene therapy that costs £2.5million per jab?
In trials, a single jab of Roctavian left nine in ten patients symptom-free and no longer in need of the daily injections usually required to control their condition. (Source: the Mail online | Health)
Source: the Mail online | Health - July 3, 2022 Category: Consumer Health News Source Type: news

BioMarin Receives Positive CHMP Opinion in Europe for Valoctocogene Roxaparvovec Gene Therapy to Treat Adults with Severe Hemophilia A
European Commission Approval Expected Q3 2022 1st Gene Therapy for Treatment of Hemophilia A Recommended for Approval in Europe More than 20,000 Adults with Severe Hemophilia A in BioMarin Territories Across Europe, the Middle East and Africa Conf... Biopharmaceuticals, Regulatory BioMarin Pharmaceutical, Valoctocogene Roxaparvovec, Hemophilia (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 24, 2022 Category: Pharmaceuticals Source Type: news

Debated: Nonfactor Versus Gene Therapy for Hemophilia Debated: Nonfactor Versus Gene Therapy for Hemophilia
The question debated was, should patients with hemophilia A stick with effective nonfactor therapy or join a clinical trial for a potential cure with gene therapy?Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - June 23, 2022 Category: Consumer Health News Tags: Hematology-Oncology News Source Type: news

For haemophiliacs, most of the world is still living in the dark ages
A sufferer explains how the differences in treatment between the UK and his native India highlight a global medical apartheidLike the Hindu deity Krishna, I was born with blue skin. My body bruised at the trauma of simply being held. And so the family arranged for a ritual to appease the gods.Haemophilia is a genetic blood disorderthat makes it very hard for the body to stop bleeding. If your haemophilia is severe like mine, you bleed spontaneously, without an injury or known cause. A handshake once took me to A&E.To stop bleeding, you need clotting injections. In much of the developing world, these injections are avai...
Source: Guardian Unlimited Science - June 14, 2022 Category: Science Authors: Tarun Gidwani Tags: Health Global development Genetics Women NHS Science Society Pharmaceuticals industry Business Source Type: news

For people with haemophilia, most of the world is still in the dark ages
Differences between the UK and India in treating the blood-clotting disease highlight a global medical apartheidLike the Hindu deity Krishna, I was born with blue skin. My body bruised at the trauma of simply being held. And so the family arranged for a ritual to appease the gods.Haemophilia is a genetic blood disorderthat makes it very hard for the body to stop bleeding. If your haemophilia is severe like mine, you bleed spontaneously, without an injury or known cause. A handshake once took me to A&E.To stop bleeding, you need clotting injections. In much of the developing world, these injections are available only to...
Source: Guardian Unlimited Science - June 14, 2022 Category: Science Authors: Tarun Gidwani Tags: Health Global development Genetics Women NHS Science Society Pharmaceuticals industry Business Source Type: news

An infant with severe hemophilia A with intracranial hemorrhage mistaken for child abuse: a case report - Youn JW, Shim YJ, Byun JC, Kwon SM.
[The publisher has not provided an abstract for this article.] Language: en... (Source: SafetyLit)
Source: SafetyLit - June 13, 2022 Category: International Medicine & Public Health Tags: Age: Infants and Children Source Type: news