Researchers ready B cells for novel cell therapy
(Seattle Children's) Scientists at Seattle Children's Research Institute are paving the way to use gene-edited B cells -- a type of white blood cell in the immune system -- to treat a wide range of potential diseases that affect children, including hemophilia and other protein deficiency disorders, autoimmune diseases, and infectious diseases. If successful, their research would open the door to offering this experimental cell therapy as the first-of-its-kind in clinical trials at Seattle Children's in as soon as five years. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - May 2, 2019 Category: Infectious Diseases Source Type: news

PHARMAC funds longer-acting treatments for haemophilia
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - May 1, 2019 Category: Drugs & Pharmacology Source Type: news

Enzyre Appoints Dirk Pollet as Chief Executive Officer
Awarded €2M grant and completes seed financing to develop high-precision point-of-care Hemophilia Biochip device NIJMEGEN, Netherlands, April 30, 2019 -- (Healthcare Sales & Marketing Network) -- Enzyre, which is developing breakthrough ambulant diagno... Diagnostics, Personnel Enzyre , Hemophilia Biochip (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 30, 2019 Category: Pharmaceuticals Source Type: news

St. Jude prepares to launch global hemophilia gene therapy trial
St. Jude is trying to establish a gene therapy program to bring next-level treatment options to patients in under-resourced countries. (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - April 23, 2019 Category: Biotechnology Authors: Jason Bolton Source Type: news

St. Jude prepares to launch global hemophilia gene therapy trial
St. Jude is trying to establish a gene therapy program to bring next-level treatment options to patients in under-resourced countries. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - April 23, 2019 Category: American Health Authors: Jason Bolton Source Type: news

Cameroon: World Hemophilia Day - Population Schooled On Treatment Mechanisms
[Cameroon Tribune] Sensitisation activities to commemorate the day end today in Yaounde. (Source: AllAfrica News: Health and Medicine)
Source: AllAfrica News: Health and Medicine - April 18, 2019 Category: African Health Source Type: news

Roche reports a strong start in 2019 and raises the outlook for the full-year
In the first three months of 2019, Group sales rose 8% to CHF 14.8 billion. Sales in the Pharmaceuticals Division increased 10% to CHF 11.9 billion. Key growth drivers were the multiple sclerosis medicine Ocrevus and cancer medicines Perjeta and Tecentriq as well as the new haemophilia medicine Hemlibra. (Source: World Pharma News)
Source: World Pharma News - April 18, 2019 Category: Pharmaceuticals Tags: Featured Roche Business and Industry Source Type: news

World Hemophilia Day: Sanofi Genzyme donation impacts patients across the globe
On World Hemophilia Day April 17th, the global bleeding disorders community unites to raise awareness and understanding of hemophilia and areas of unmet needs for patients. Hemophilia is a rare, genetic bleeding disorder in which the ability of a person's blood to clot is impaired, which can lead to bleeding episodes that can cause pain, irreversible joint damage, and life-threatening hemorrhages. (Source: World Pharma News)
Source: World Pharma News - April 17, 2019 Category: Pharmaceuticals Tags: Featured Sanofi Business and Industry Source Type: news

Gene-based factor VIIa prevents bleeding episodes in animals with hemophilia
(Children's Hospital of Philadelphia) Hematology researchers have further refined how a treatment currently used on an urgent basis to control bleeding in hemophilia patients holds promise as a preventive treatment as well. A study in animals may set the stage for a new therapy for a subset of patients with hemophilia who now develop antibodies to the standard maintenance treatment and then require on-demand 'bypass' therapy. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 15, 2019 Category: International Medicine & Public Health Source Type: news

Cochrane's 30 under 30: Ndi Euphrasia Ebai-Atuh
Cochrane is made up of  13,000 members and over 50,000 supporters come from more than 130 countries, worldwide. Our volunteers and contributors are researchers, health professionals, patients, carers, people passionate about improving health outcomes for everyone, everywhere.Cochrane is an incredible community of people who all play their part in improving health and healthcare globally. We believe that by putting trusted evidence at the heart of health decisions we can achieve a world of improved health for all.  Many  of our contributors are young people working with Cochrane ...
Source: Cochrane News and Events - April 12, 2019 Category: Information Technology Authors: Lydia Parsonson Source Type: news

European Commission approves Roche ’s Hemlibra for people with severe haemophilia A without factor VIII inhibitors
Roche today announced that the European Commission has approved Hemlibra ® (emicizumab) for routine prophylaxis of bleeding episodes in people with severe haemophilia A (congenital factor VIII deficiency, FVIII (Source: Roche Media News)
Source: Roche Media News - March 14, 2019 Category: Pharmaceuticals Source Type: news

European Commission approves Roche ’s Hemlibra for people with severe haemophilia A without factor VIII inhibitors
Roche today announced that the European Commission has approved Hemlibra ® (emicizumab) for routine prophylaxis of bleeding episodes in people with severe haemophilia A (congenital factor VIII deficiency, FVIII (Source: Roche Investor Update)
Source: Roche Investor Update - March 14, 2019 Category: Pharmaceuticals Source Type: news

Roche to buy Spark Therapeutics
Roche Holding has entered a definitive merger agreement with Spark Therapeutics, as the Swiss drugmaker seeks to expand its ability to treat rare diseases through gene therapies, as well as build its hemophilia portfolio. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - February 25, 2019 Category: Pharmaceuticals Source Type: news

Roche to buy gene therapy specialist Spark in $4.3 billion deal
Roche Holding AG said on Monday it will buy Spark Therapeutics in a $4.3 billion deal, as the Swiss drugmaker builds its hemophilia portfolio and seeks to keep pace in gene therapy. (Source: Reuters: Health)
Source: Reuters: Health - February 25, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

FDA OKs Turoctocog Alfa Pegol (Esperoct) for Hemophilia A FDA OKs Turoctocog Alfa Pegol (Esperoct) for Hemophilia A
Turoctocog alfa pegol is an extended half-life factor VIII molecule for treatment and prophylaxis of bleeding episodes and perioperative management in adults and children with hemophilia A.FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - February 21, 2019 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news

FDA Approves Esperoct (turoctocog alfa pegol, N8-GP) for Hemophilia A
Bagsværd, Denmark, 19 February 2019 - Novo Nordisk today announced that the US Food and Drug Administration (FDA) has approved the Biologics License Application for Esperoct for the treatment of adults and children with hemophilia A. Esperoct... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - February 20, 2019 Category: Drugs & Pharmacology Source Type: news

Highmark reaches settlement with specialty pharmacy
Highmark and Highmark Benefits Group have settled a federal lawsuit filed by a Florida-based speciality pharmacy that claimed the insurer denied payments for specialty pharmacy medications for people suffering from the blood disease hemophilia. The settlement announced Tuesday afternoon between Highmark and BioMatrix ends the lawsuit. Details weren’t released other than to say that Highmark and BioMatrix, a Weston, Florida, company that manufacturers specialty medications to treat hemophilia,… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - February 12, 2019 Category: Biotechnology Authors: Paul J. Gough Source Type: news

Highmark reaches settlement with specialty pharmacy
Highmark and Highmark Benefits Group have settled a federal lawsuit filed by a Florida-based speciality pharmacy that claimed the insurer denied payments for specialty pharmacy medications for people suffering from the blood disease hemophilia. The settlement announced Tuesday afternoon between Highmark and BioMatrix ends the lawsuit. Details weren’t released other than to say that Highmark and BioMatrix, a Weston, Florida, company that manufacturers specialty medications to treat hemophilia,… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - February 12, 2019 Category: American Health Authors: Paul J. Gough Source Type: news

UniQure gene therapy shows promising result in mid-stage trial
Gene therapy company UniQure NV said on Friday its treatment for hemophilia B increased the levels of a protein that helps in blood clotting after 12 weeks in a relatively small study. (Source: Reuters: Health)
Source: Reuters: Health - February 8, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

UniQure gene therapy shows rise in blood-clotting protein levels
Drug developer UniQure NV said on Friday its gene therapy to treat hemophilia B increased the levels of a protein that helps in blood clotting after 12 weeks in a small study. (Source: Reuters: Health)
Source: Reuters: Health - February 8, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

UniQure gene therapy shows rise in blood-clotting protein in trial
UniQure NV said on Friday its gene therapy to treat patients with a blood disorder, hemophilia B, increased the levels of a protein that helps in blood clotting after 12 weeks. (Source: Reuters: Health)
Source: Reuters: Health - February 8, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Roche, Joining Rivals, Donates Hemophilia Drug to Boost Access Roche, Joining Rivals, Donates Hemophilia Drug to Boost Access
Switzerland's Roche will donate its new hemophilia A drug Hemlibra to a World Hemophilia Federation program, it said on Wednesday, joining rivals who also back the effort to help patients in developing countries who face treatment hurdles.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - February 7, 2019 Category: Consumer Health News Tags: Medscape Today News Source Type: news

Takeda ordered to shell out $155m to Bayer for patent infringement
Jurors in a U.S. federal court have ordered a Takeda Pharmaceutical Co. unit to pay Bayer AG $155.19 million for a case related to a hemophilia drug. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - February 7, 2019 Category: Pharmaceuticals Source Type: news

Roche, joining rivals, donates hemophilia drug to boost access
Switzerland's Roche will donate its new hemophilia A drug Hemlibra to a World Hemophilia Federation program, it said on Wednesday, joining rivals who also back the effort to help patients in developing countries who face treatment hurdles. (Source: Reuters: Health)
Source: Reuters: Health - February 6, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Roche joins the World Federation of Hemophilia Humanitarian Aid Program
Roche today announced that it has joined the World Federation of Hemophilia (WFH) Humanitarian Aid Program, a landmark initiative leading the effort to change the lack of access to care and treatment for people with inherited bleeding disorders in developing countries. (Source: Roche Media News)
Source: Roche Media News - February 6, 2019 Category: Pharmaceuticals Source Type: news

U.S. jury orders Takeda to pay Bayer $155 million over hemophilia drug
A U.S. jury has ordered Takeda Pharmaceutical Co's Baxalta unit to pay Bayer AG $155.19 million for infringing a patent related to a Baxalta hemophilia treatment, court records made public on Tuesday show. (Source: Reuters: Health)
Source: Reuters: Health - February 5, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

German firm that serves gene therapy biotechs opens Cambridge HQ
Ahead of the opening this week, the German biotech firm signed two major partnerships with Boston-based Orchard Therapeutics and San Francisco-based Denali Therapeutics, and is also in the process of closing another partnership with a U.S. biotech company focused on hemophilia treatments. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - February 1, 2019 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

CHMP recommends EU approval of Roche ’s Hemlibra for people with severe haemophilia A without factor VIII inhibitors
Roche today announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Hemlibra ®(emicizumab) for routine prophylaxis of bleeding episodes in adults and children with severe haemophilia A (congenital factor VIII deficiency, FVIII (Source: Roche Media News)
Source: Roche Media News - February 1, 2019 Category: Pharmaceuticals Source Type: news

CHMP recommends EU approval of Roche ’s Hemlibra for people with severe haemophilia A without factor VIII inhibitors
Roche today announced that the EU Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion for Hemlibra ®(emicizumab) for routine prophylaxis of bleeding episodes in adults and children with severe haemophilia A (congenital factor VIII deficiency, FVIII (Source: Roche Investor Update)
Source: Roche Investor Update - February 1, 2019 Category: Pharmaceuticals Source Type: news

New name, new offices for blood services agency
The new year will see a new name and location for the Hemophilia Center of Western New York. The organization has rebranded as WNY Bloodcare, a name it says better reflects the range of blood disorders it serves.  While it will continue to serve individuals with hemophilia, its staff of hematologists, laboratory and specialty pharmacy professionals diagnoses and treats patients with a r ange of bleeding and clotting disorders. The agency has long been based at 936 Delaware Ave. and now plans… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - January 16, 2019 Category: Health Management Authors: Tracey Drury Source Type: news

Game-changing research reveals that parental experience is epigenetically imprinted onto 14 successive generations
(Natural News) We all know that we inherit certain specific genetic characteristics from our parents and that these are indelibly written in our DNA code. These genetic traits determine everything from the color of our eyes to whether or not we have dimples. Unfortunately, certain genetic diseases like cystic fibrosis and hemophilia can be passed... (Source: NaturalNews.com)
Source: NaturalNews.com - January 14, 2019 Category: Consumer Health News Source Type: news

Takeda/haemophilia: blood test
a breakthrough inIf gene therapy lives up to its promise, it reallywill justify the song and dance (Source: FT.com - Drugs and Healthcare)
Source: FT.com - Drugs and Healthcare - December 5, 2018 Category: Pharmaceuticals Source Type: news

Roche says Hemlibra effective in pivotal study
Roche's Hemlibra provided sustained bleed control in the largest pivotal study to date of children with a form of haemophilia, the Swiss drugmaker said on Monday. (Source: Reuters: Health)
Source: Reuters: Health - December 3, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Roche ’s Hemlibra provided sustained bleed control in the largest pivotal study to date of children with haemophilia A with factor VIII inhibitors
Roche today announced data from the primary analysis of the phase III HAVEN 2 study evaluating Hemlibra ® (emicizumab) prophylaxis in children younger than 12 years of age with haemophilia A with factor VIII inhibitors, including longer follow-up for once-weekly dosing and new data for less frequent dosing schedules (every two weeks or every four weeks). (Source: Roche Media News)
Source: Roche Media News - December 3, 2018 Category: Pharmaceuticals Source Type: news

Roche ’s Hemlibra provided sustained bleed control in the largest pivotal study to date of children with haemophilia A with factor VIII inhibitors
Roche today announced data from the primary analysis of the phase III HAVEN 2 study evaluating Hemlibra ® (emicizumab) prophylaxis in children younger than 12 years of age with haemophilia A with factor VIII inhibitors, including longer follow-up for once-weekly dosing and new data for less frequent dosing schedules (every two weeks or every four weeks). (Source: Roche Investor Update)
Source: Roche Investor Update - December 3, 2018 Category: Pharmaceuticals Source Type: news

St. Jude research into leukemia, sickle cell and other blood disorders presented at ASH
(St. Jude Children's Research Hospital) The 60th Annual Meeting of the American Society of Hematology will feature research from St. Jude Children's Research Hospital on topics ranging from the genomic basis and vulnerabilities of leukemia to an update on gene therapy for hemophilia B to advances in sickle cell disease and beta-thalassemia. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - November 30, 2018 Category: Cancer & Oncology Source Type: news

Bayer will shift hemophilia drug work to Berkeley, but won't add jobs
Bayer AG will cut 12,000 jobs globally and shut a recently constructed hemophilia drug-making facility in Germany, consolidating manufacturing of its blood-clotting drugs in Berkeley. Bayer has no plans to recall people to the roughly 400 manufacturing-related jobs it has cut over the past year in Berkeley, the only unionized biotech workforce in the United States. But the moves disclosed Thursday allow Bayer to mop up excess capacity in response to a more-competitive hemophili a market — including… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - November 29, 2018 Category: Biotechnology Authors: Ron Leuty Source Type: news

Bayer will shift hemophilia drug work to Berkeley, but won't add jobs
Bayer AG will cut 12,000 jobs globally and shut a recently constructed hemophilia drug-making facility in Germany, consolidating manufacturing of its blood-clotting drugs in Berkeley. Bayer has no plans to recall people to the roughly 400 manufacturing-related jobs it has cut over the past year in Berkeley, the only unionized biotech workforce in the United States. But the moves disclosed Thursday allow Bayer to mop up excess capacity in response to a more-competitive hemophili a market — including… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - November 29, 2018 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

27.11.18: Not intended for U.S. and UK Media
Bayer receives EU approval for its hemophilia A treatment Jivi®The safety and efficacy profile of Jivi has been demonstrated in more than five years of clinical studies / Prophylaxis with Jivi enables sustained factor VIII concentrations in the blood over timemehr ... (Source: Bayer IR Newsfeed: Events)
Source: Bayer IR Newsfeed: Events - November 27, 2018 Category: Pharmaceuticals Source Type: news

Bayer receives EU approval for its hemophilia A treatment Jivi ® (for specialized target groups only)
The safety and efficacy profile of Jivi has been demonstrated in more than five years of clinical studies / Prophylaxis with Jivi enables sustained factor VIII concentrations in the blood over time (Source: Bayer Company News)
Source: Bayer Company News - November 27, 2018 Category: Pharmaceuticals Source Type: news

BioMarin sets stage early for hemophilia cure off-Broadway
BioMarin Pharmaceutical Inc is turning to the theater to establish its name with hemophilia patients, long before its experimental cure for the bleeding disorder could reach the market. (Source: Reuters: Health)
Source: Reuters: Health - November 21, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

Harvey's gang: Haemophilia patient trades places with doctor
James McGleenan, 11, swaps places with his doctor to find out what happens when blood is taken. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - November 20, 2018 Category: Consumer Health News Source Type: news

Harvey's gang: 11-year-old haemophilia patient trades place with doctor
James McGleenan, 11, swaps places with his doctor to find out what happens when blood is taken. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - November 20, 2018 Category: Consumer Health News Source Type: news

Hemophilia, The Musical? It ’s Funded By Biotech BioMarin
Rare disease drug company BioMarin put on a show called ‘Hemophilia: The Musical’ performed by teenagers affected by blood disorders. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - November 16, 2018 Category: Pharmaceuticals Authors: Michela Tindera, Forbes Staff Source Type: news

Medical News Today: Hemophilia, coagulation, and blood clotting
If blood does not clot, life-threatening bleeding can occcur. Find out about how blood clots, how clotting factors work, and how treatment has improved the outlook for people with hemophilia. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - November 16, 2018 Category: Consumer Health News Tags: Blood / Hematology Source Type: news

Morning Break:'Hemophilia the Musical'; Another ARB Recall; Global Contraception Use
(MedPage Today) -- Health news and commentary from around the Web gathered by the MedPage Today staff (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - November 13, 2018 Category: American Health Source Type: news

Teenage haemophiliacs were called in groups to be told if they’d been infected with HIV
Almost all the dozens of photographs Adrian, 47, has catalogued from his youth show the faces of schoolfriends who have died, most before they were 30 years old. (Source: the Mail online | Health)
Source: the Mail online | Health - October 23, 2018 Category: Consumer Health News Source Type: news

FDA expands Roche hemophilia med approval
The drug, Hemlibra, won approval in the U.S. only for hemophilia A patients with factor VIII resistance, or inhibitors last year (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - October 8, 2018 Category: Pharmaceuticals Source Type: news

U.S. FDA Expands Indication for Roche's Bleeding Disorder Drug U.S. FDA Expands Indication for Roche's Bleeding Disorder Drug
Roche said on Thursday it received approval from U.S. regulators for its hemophilia A drug Hemlibra (emicizumab)for use in nearly all patients, as the Swiss drugmaker pushes deeper into diseases beyond cancer to replace revenue from older, patent-expired drugs.FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - October 5, 2018 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news