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​Alnylam halts hemophilia drug trial due to patient death, renewing worries over safety
Alnylam Pharmaceuticals, one of the state ’s largest and most valuable biotechs, has temporarily halted a mid-stage trial of one of its experimental drugs for hemophilia after one patient died, shaving $1 billion from its market cap. Cambridge-based Alnylam (Nasdaq: ALNY) said Thursday that it had suspended the Phase 2 trial of fitusiran after a patient died of brain swelling triggered by a blood clot. The company said the death was “possibly related” to the treatment, and that it hopes to… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - September 7, 2017 Category: American Health Authors: Max Stendahl Source Type: news

Bayer submits marketing authorization application for BAY94-9027 for the treatment of Hemophilia A in the EU (for specialized target groups only)
Pivotal studies with BAY94-9027 showed that bleed protection was achieved with extended dosing intervals (Source: Bayer Company News)
Source: Bayer Company News - September 7, 2017 Category: Pharmaceuticals Source Type: news

Blood Disorders
Centers for Disease Control and Prevention. 07/19/2017 This Web page provides links to information, tips, sources, and treatment centers for several blood disorders: Thalassemia, Hemophilia, Sickle Cell Disease, and Thrombosis and Hemostasis. (Text) (Source: Disaster Lit: Resource Guide for Disaster Medicine and Public Health)
Source: Disaster Lit: Resource Guide for Disaster Medicine and Public Health - September 5, 2017 Category: International Medicine & Public Health Authors: The U.S. National Library of Medicine Source Type: news

PRAC Confirms No Difference in Risk Between Hemophilia A Drugs PRAC Confirms No Difference in Risk Between Hemophilia A Drugs
The EMA's Pharmacovigilance Risk Assessment Committee has confirmed its earlier conclusion that factor VIII medications do not differ in their risk for inhibition.News Alerts (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - September 1, 2017 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news

Prophylaxis Linked to Improved Function, HRQoL in Hemophilia
Improved function, QoL, activity, pain but not joint structure in adults with hemophilia with joint disease (Source: The Doctors Lounge - Oncology)
Source: The Doctors Lounge - Oncology - August 31, 2017 Category: Cancer & Oncology Tags: Internal Medicine, Oncology, Orthopedics, Pediatrics, Pharmacy, Journal, Source Type: news

Bayer submits Biologics License Application in the U.S. for BAY94-9027 – a long-acting factor VIII for the treatment of Hemophilia A (for specialized target groups only)
Pivotal studies with BAY94-9027 showed that bleed protection was achieved with extended dosing intervals (Source: Bayer Company News)
Source: Bayer Company News - August 31, 2017 Category: Pharmaceuticals Source Type: news

Sangamo and Pfizer begin dosing in Phase I/II trial for haemophilia A
Sangamo Therapeutics and Pfizer have begun dosing patients in a Phase I/II clinical trial (Alta) of investigational gene therapy SB-525 to treat patients with a rare genetic blood disease known as haemophilia A. (Source: Drug Development Technology)
Source: Drug Development Technology - August 28, 2017 Category: Pharmaceuticals Source Type: news

Minecraft-based Pfizer app uses gamification to help kids with hemophilia
Pfizer this week launched a modification of the popular sandbox game Minecraft, offering children with hemophilia a new way to learn about staying safe and prepared and maintaining their treatment plans.   (Source: mobihealthnews)
Source: mobihealthnews - August 25, 2017 Category: Information Technology Source Type: news

FDA accepts Roche ’s BLA for emicizumab to treat Haemophilia A
The US Food and Drug Administration (FDA) has accepted Roche ’s biologics licence application (BLA) and granted a priority review for emicizumab prophylaxis (preventative) to treat patients with Haemophilia A with factor VIII inhibitors. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - August 24, 2017 Category: Pharmaceuticals Source Type: news

To End HIV Stigma, We Must Fight a Long History
August 24, 2017Health workers can play a key role in ending discrimination against people who live with HIV.“Because of the lack of education on AIDS, discrimination, fear, panic, and lies surrounded me.” —Ryan WhiteRyan White was diagnosed with AIDS at the age of 13 in Indiana in 1984, following a blood transfusion to treat hemophilia A, his inherited blood disease.It was a time when most people knew little about HIV and AIDS. Ryan and his family experienced overwhelming and ignorant stigma and discrimination. Some feared he might transmit HIV through casual contact, or made stereotyped assumptions about...
Source: IntraHealth International - August 24, 2017 Category: International Medicine & Public Health Authors: mnathe Source Type: news

Roche says FDA grants priority review to hemophilia drug emicizumab
ZURICH (Reuters) - Swiss pharma group Roche said the U.S. Food and Drug Administration (FDA) granted priority review to its emicizumab for hemophilia A with inhibitors. (Source: Reuters: Health)
Source: Reuters: Health - August 24, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

FDA grants Priority Review to Roche ’s emicizumab for haemophilia A with inhibitors
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the US Food and Drug Administration (FDA) has accepted the company ’s Biologics License Application (BLA) and granted Priority Review for emicizumab prophylaxis (preventative) as a once-weekly subcutaneous treatment for adults, adolescents and children with haemophilia A with factor VIII inhibitors. (Source: Roche Media News)
Source: Roche Media News - August 24, 2017 Category: Pharmaceuticals Source Type: news

FDA grants Priority Review to Roche ’s emicizumab for haemophilia A with inhibitors
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that the US Food and Drug Administration (FDA) has accepted the company ’s Biologics License Application (BLA) and granted Priority Review for emicizumab prophylaxis (preventative) as a once-weekly subcutaneous treatment for adults, adolescents and children with haemophilia A with factor VIII inhibitors. (Source: Roche Investor Update)
Source: Roche Investor Update - August 24, 2017 Category: Pharmaceuticals Source Type: news

Children's Healthcare of Atlanta's Aflac Cancer and Blood Disorders Center awarded $6 million grant
The Aflac Cancer and Blood Disorders Center at Children's cares for more than 450 newly diagnosed children a year and follows 3,200 patients with sickle cell disease, hemophilia and other blood disorders. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - August 11, 2017 Category: American Health Authors: Ellie Hensley Source Type: news

Gene Therapy Corrects Factor VIII Levels in Hemophilia A Gene Therapy Corrects Factor VIII Levels in Hemophilia A
Transferring a form of the gene for factor VIII using a viral vector corrected plasma levels of the factor and stopped bleeding episodes for patients with severe hemophilia A, a study shows.Medscape Medical News (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - August 7, 2017 Category: Consumer Health News Tags: Cardiology News Source Type: news

Lawsuit: New TennCare rates endanger hemophilia patients
A Tennessee lawsuit claims the state is breaking federal law and putting people with bleeding disorders like hemophilia in great danger by reimbursing less for prescription blood-clotting drugs through its Medicaid program (Source: ABC News: Health)
Source: ABC News: Health - July 18, 2017 Category: Consumer Health News Tags: Health Source Type: news

Novel Agent Reduces Antithrombin Levels to Treat Hemophilia Novel Agent Reduces Antithrombin Levels to Treat Hemophilia
Fitusiran, an RNA interference agent, safely reduces antithrombin levels to allow clot formation to treat hemophilia A or B in patients with or without inhibitors, a study shows.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 14, 2017 Category: Consumer Health News Tags: Cardiology News Source Type: news

Roche and Chugai Pharma report positive Phase III results of haemophilia A drug
Roche and UK-based Chugai Pharmaceutical have reported positive data from the Phase III HAVEN 1 clinical trial of emicizumab in patients suffering from haemophilia A. (Source: Drug Development Technology)
Source: Drug Development Technology - July 11, 2017 Category: Pharmaceuticals Source Type: news

Emicizumab Replaces Factor VIII Function in Hemophilia A Emicizumab Replaces Factor VIII Function in Hemophilia A
The bispecific monoclonal antibody emicizumab replaced the function of factor VIII in people with hemophilia A and overcame neutralizing antibodies against factor VIII, results of the HAVEN 1 trial show.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 11, 2017 Category: Consumer Health News Tags: Cardiology News Source Type: news

UK to launch inquiry into tainted blood product that killed 2,400 patients
UK officials will launch an inquiry into why thousands of patients, many of them hemophiliacs, became infected with HIV and hepatitis C from blood products used more than 30 years ago. (Source: CNN.com - Health)
Source: CNN.com - Health - July 11, 2017 Category: Consumer Health News Source Type: news

Roche, Shire Court Fight Underscores High Stakes in Hemophilia Roche, Shire Court Fight Underscores High Stakes in Hemophilia
Roche's bid to muscle in on Shire's share of the $11 billion hemophilia drug market took a new, contentious turn this weekend when the British drugmaker won a court injunction against how the Swiss drugmaker talks about its new medicine.Reuters Health Information (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 11, 2017 Category: Consumer Health News Tags: Internal Medicine News Source Type: news

Celebrating 10 years of success for Infusion
As the business took off, more space was needed, and the company moved in 2009 to “real” office space just up the road in Haddam, CT, across the street from the Connecticut River, eventually employing a staff of 10 that year. A year later, in 2010, Infusion had doubled in size and became part of the UDG/Ashfield family. Infusion continued to grow at a steady pace into new therapeutic areas with more clients and employees, and in October 2016, Infusion left its country setting and moved into larger corporate offices in downtown Middletown, CT. Our staff now comprises 45 people and includes account executives, me...
Source: Ashfield Healthcare News - July 11, 2017 Category: Pharmaceuticals Authors: Sarah Edwards Tags: Ashfield Source Type: news

Alnylam and Sanofi commence Phase III programme for haemophilia drug
Alnylam Pharmaceuticals and Sanofi Genzyme have commenced the Phase III clinical programme (ATLAS) of RNAi therapeutic fitusiran in patients with haemophilia A or B, with or without inhibitors. (Source: Drug Development Technology)
Source: Drug Development Technology - July 10, 2017 Category: Pharmaceuticals Source Type: news

Novel Hemophilia A Treatments Advance
(MedPage Today) -- Monoclonal antibody, RNAi tx show promise in trials (Source: MedPage Today Pediatrics)
Source: MedPage Today Pediatrics - July 10, 2017 Category: Pediatrics Source Type: news

Disruptive technology reduces bleeding in hemophilia by 87 percent
Researchers at Children's Hospital Los Angeles developed a new therapy called emicizumab that decreases bleeding episodes by nearly 90 percent. (Source: Health News - UPI.com)
Source: Health News - UPI.com - July 10, 2017 Category: Consumer Health News Source Type: news

Shire, Roche slug it out in billion-dollar haemophilia drug battle
ZURICH (Reuters) - Swiss drugmaker Roche's bid to take a chunk of the $11 billion haemophilia drug market dominated by Shire took another blow with the Irish company winning a preliminary injunction over its Swiss rival's medication. (Source: Reuters: Health)
Source: Reuters: Health - July 10, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Positive phase III results for Roche ’s emicizumab in haemophilia A published in The New England Journal of Medicine
Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that data from HAVEN 1, a phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with haemophilia A with inhibitors, were published in The New England Journal of Medicine (NEJM). (Source: World Pharma News)
Source: World Pharma News - July 10, 2017 Category: Pharmaceuticals Tags: Featured Roche Business and Industry Source Type: news

Positive phase III results for Roche ’s emicizumab in haemophilia A published in The New England Journal of Medicine
Roche today announced that data from HAVEN 1, a phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with haemophilia A with inhibitors, were published in The New England Journal of Medicine (NEJM). (Source: Roche Media News)
Source: Roche Media News - July 10, 2017 Category: Pharmaceuticals Source Type: news

Positive phase III results for Roche ’s emicizumab in haemophilia A published in The New England Journal of Medicine
Roche today announced that data from HAVEN 1, a phase III study evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) in adults and adolescents with haemophilia A with inhibitors, were published in The New England Journal of Medicine (NEJM). (Source: Roche Investor Update)
Source: Roche Investor Update - July 10, 2017 Category: Pharmaceuticals Source Type: news

Roche, Shire court fight underscores high stakes in hemophilia
ZURICH (Reuters) - Roche's bid to muscle in on Shire's share of the $11 billion hemophilia drug market took a new, contentious turn this weekend when the British drugmaker won a court injunction against how the Swiss drugmaker talks about its new medicine. (Source: Reuters: Health)
Source: Reuters: Health - July 10, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Disruptive technology for the treatment of hemophilia
(Children's Hospital Los Angeles) An international team of hematologists including Guy Young, MD, of Children's Hospital Los Angeles, has found that in patients with hemophilia A with inhibitors, a novel therapy called emicizumab, decreases incidence of bleeding episodes by 87 percent. Results of this multicenter phase III study called HAVEN 1, will be presented at the International Society of Thrombosis and Hemostasis and published in the New England Journal of Medicine on July 10. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 10, 2017 Category: International Medicine & Public Health Source Type: news

Shire submits IND application for SHP654 to treat haemophilia A
Irish-based Shire has submitted an investigational new drug (IND) application to the US Food and Drug Administration (FDA) for SHP654, also designated as BAX 888, an investigational factor VIII (FVIII) gene therapy to treat haemophilia A. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - July 9, 2017 Category: Pharmaceuticals Source Type: news

Shire gets injunction against Roche over hemophilia drug
ZURICH (Reuters) - Pharmaceutical group Shire said on Sunday it had obtained a preliminary injunction in a Hamburg court against rival Roche over its hemophilia drug emicizumab, alleging incomplete and misleading statements surrounding the treatment. (Source: Reuters: Health)
Source: Reuters: Health - July 9, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Economic burden of haemophilia  B high in the US
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - June 30, 2017 Category: Drugs & Pharmacology Source Type: news

New Roche haemophilia drug prevents bleeds but questions remain
ZURICH (Reuters) - Roche's investigational haemophilia drug emicizumab cut the bleed rate by 87 percent in patients with resistance to standard therapy compared with those who received another treatment, the Swiss company said on Monday. (Source: Reuters: Health)
Source: Reuters: Health - June 26, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Roche says new hemophilia drug cuts bleed rate by 87 percent
ZURICH (Reuters) - Roche's investigational hemophilia drug emicizumab cut the bleed rate by 87 percent in patients who had developed resistance to standard treatment, compared with those who instead got bypassing agents, the Swiss company said on Monday. (Source: Reuters: Health)
Source: Reuters: Health - June 26, 2017 Category: Consumer Health News Tags: healthNews Source Type: news

Roche ’s emicizumab showed positive results in phase III studies (HAVEN 1 and HAVEN 2) in haemophilia A with inhibitors
Roche today announced positive data from the primary analysis of the phase III HAVEN 1 study in adults and adolescents and interim analysis of the phase III HAVEN 2 study in children evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) for the treatment of haemophilia A with inhibitors to factor VIII. (Source: Roche Media News)
Source: Roche Media News - June 26, 2017 Category: Pharmaceuticals Source Type: news

Roche ’s emicizumab showed positive results in phase III studies (HAVEN 1 and HAVEN 2) in haemophilia A with inhibitors
Roche today announced positive data from the primary analysis of the phase III HAVEN 1 study in adults and adolescents and interim analysis of the phase III HAVEN 2 study in children evaluating once-weekly subcutaneous emicizumab prophylaxis (preventative) for the treatment of haemophilia A with inhibitors to factor VIII. (Source: Roche Investor Update)
Source: Roche Investor Update - June 26, 2017 Category: Pharmaceuticals Source Type: news

International team to evaluate new advances in hemophilia treatment
(McMaster University) The CoreHEM project will publish an 'effectiveness guidance document' and accompanying peer reviewed article providing recommendations for patient-important outcomes for clinical studies. This is expected by early 2018. Use of these measures will help patients and clinicians to make better treatment decisions, payers to make better policy decisions, and companies to have better predictability in how their studies will be assessed. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 20, 2017 Category: International Medicine & Public Health Source Type: news

Sangamo Therapeutics And Pfizer Announce That SB-525 Investigational Hemophilia A Gene Therapy Receives Orphan Medicinal Product Designation From The European Medicines Agency
Companies also Announce Phase 1/2 Clinical Trial Evaluating SB-525 in Adults with Severe Hemophilia A is Now Open for Enrollment RICHMOND, Calif., June 7, 2017 -- (Healthcare Sales & Marketing Network) -- Sangamo Therapeutics, Inc. (NASDAQ: SGMO) and P... Biopharmaceuticals, Regulatory Sangamo Therapeutics, Pfizer, Hemophilia, Gene Therapy (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - June 7, 2017 Category: Pharmaceuticals Source Type: news

FDA Clears Rebinyn for Hemophilia B FDA Clears Rebinyn for Hemophilia B
The coagulation factor IX product is for the treatment and control of bleeding episodes and the perioperative management of bleeding in adults and children with hemophilia B.News Alerts (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - June 1, 2017 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news

FDA Approves Rebinyn (Coagulation Factor IX (Recombinant), GlycoPEGylated) for Patients with Hemophilia B
PLAINSBORO, N.J., May 31, 2017 /PRNewswire/ -- Novo Nordisk today announced that the U.S. Food and Drug Administration (FDA) has approved the Biologics License Application for Rebinyn (Coagulation Factor IX (Recombinant), GlycoPEGylated) for the... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - May 31, 2017 Category: Drugs & Pharmacology Source Type: news

Hemophilia Center offers new clinic for girls
The Hemophilia Center of Western New York has opened a new clinic aimed at growing awareness and diagnosis of bleeding disorders among girls and women. The new Specialized Hematology Experts (SHE) Clinic is the brainchild of Shilpa Jain, a pediatric hematologist, and Shaveta Malik, a gynecologist and expert in reproductive health who treats pediatric and adult patients. It’s one of just a handful of clinics across the country focused on girls a nd women with bleeding and clotting disorders. Though… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - May 22, 2017 Category: Biotechnology Authors: Tracey Drury Source Type: news

Hemophilia Center offers new clinic for girls
The Hemophilia Center of Western New York has opened a new clinic aimed at growing awareness and diagnosis of bleeding disorders among girls and women. The new Specialized Hematology Experts (SHE) Clinic is the brainchild of Shilpa Jain, a pediatric hematologist, and Shaveta Malik, a gynecologist and expert in reproductive health who treats pediatric and adult patients. It’s one of just a handful of clinics across the country focused on girls a nd women with bleeding and clotting disorders. Though… (Source: bizjournals.com Health Care News Headlines)
Source: bizjournals.com Health Care News Headlines - May 22, 2017 Category: Health Management Authors: Tracey Drury Source Type: news

Swedish Orphan Biovitrum AB - Elocta(R) Approved in the Kingdom of Saudi Arabia for the Treatment of Haemophilia A
STOCKHOLM, May 18, 2017 -- (Healthcare Sales & Marketing Network) -- Swedish Orphan Biovitrum AB (publ) (Sobi™) today announces that the Saudi Food & Drug Authority (SFDA) in the Kingdom of Saudi Arabia has approved Elocta® (efmoroctocog alfa), a reco... Biopharmaceuticals, Regulatory Swedish Orphan Biovitrum, Sobi , Elocta , haemophilia A (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - May 18, 2017 Category: Pharmaceuticals Source Type: news

First In Vivo Human Genome Editing to Be Tested in New Clinical Trial
Sangamo Therapeutics will use zinc finger nucleases to introduce the gene for a missing clotting factor into the livers of men with hemophilia B. (Source: The Scientist)
Source: The Scientist - May 18, 2017 Category: Science Tags: News & Opinion,News Analysis,The Scientist Source Type: news

What Causes Anemia?
Discussion One of the most common problems in pediatrics is anemia. It is defined as “a lower than normal value for the related measurements of hemoglobin, hematocrit, and number of red blood cells”, usually 2 standard deviations below the normal for age. Normal hematological values change with age. For a discussion of which values are used click here. The most common type of anemia in childhood is iron deficiency which is commonly caused by inadequate stores (e.g. premature infant), inadequate intake (e.g. poor nutrition) or blood loss (e.g. menses). Anemia screening is recommended at age 9-12 months, and for...
Source: PediatricEducation.org - May 15, 2017 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Sangamo Therapeutics and Pfizer announce collaboration for Hemophilia A gene therapy
Sangamo Therapeutics, Inc. (Nasdaq: SGMO) and Pfizer Inc. (NYSE: PFE) have announced an exclusive, global collaboration and license agreement for the development and commercialization of gene therapy programs for Hemophilia A, including SB-525, one of Sangamo's four lead product candidates, which Sangamo expects will enter the clinic this quarter. (Source: World Pharma News)
Source: World Pharma News - May 11, 2017 Category: Pharmaceuticals Tags: Featured Pfizer Business and Industry Source Type: news

Pfizer strikes deal with Sangamo, broadening hemophilia pipeline
NEW YORK (Reuters) - U.S. drugmaker Pfizer Inc said on Wednesday it reached an agreement with Sangamo Therapeutics Inc for rights to its gene therapy programs to treat the rare blood-clotting disorder hemophilia A, under which Pfizer will pay $70 million upfront. (Source: Reuters: Health)
Source: Reuters: Health - May 10, 2017 Category: Consumer Health News Tags: healthNews Source Type: news