CSL Behring reports positive Phase III PROLONG-9FP trial data of IDELVION to treat haemophilia B
Australian CSL's US biotherapeutics subsidiary CSL Behring has reported positive results from its Phase III PROLONG-9FP clinical development programme of IDELVION to treat haemophilia B. (Source: Drug Development Technology)
Source: Drug Development Technology - December 6, 2016 Category: Pharmaceuticals Source Type: news

Gene Therapy Shows Promise in Hemophilia B (CME/CE)
(MedPage Today) -- No infusions, major bleeds in preliminary trial (Source: MedPage Today Meeting Coverage)
Source: MedPage Today Meeting Coverage - December 5, 2016 Category: Journals (General) Source Type: news

After one dose of gene therapy, hemophilia B patients maintain near-normal levels of clotting factor
Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor IX in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental gene therapy in a clinical trial, patients with hemophilia produced near-normal levels of clotting factor IX, allowing them to stop clotting factor infusions and to pursue normal activities of daily life without disabling bleeding episodes. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - December 3, 2016 Category: Science Source Type: news

After gene therapy, hemophilia B patients maintain near-normal levels of clotting factor
(Children's Hospital of Philadelphia) Researchers are reporting the highest and most sustained levels to date of an essential blood-clotting factor in patients with the inherited bleeding disorder hemophilia B. After receiving a single dose of an experimental gene therapy in a clinical trial, patients with hemophilia produced near-normal levels of clotting factor IX, allowing them to stop clotting factor infusions and to pursue normal activities of daily life without disabling bleeding episodes. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 3, 2016 Category: Global & Universal Source Type: news

CRISPR used for first time to correct clotting in newborn and adult mice
CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. For the first time, researchers have developed a dual gene therapy approach to deliver key components of a CRISPR/Cas9-mediated gene targeting system to mice to treat hemophilia B. This disorder is also called factor IX deficiency and is caused by a missing or defective clotting protein. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 30, 2016 Category: Science Source Type: news

Penn scientists use CRISPR for first time to correct clotting in newborn and adult mice
(University of Pennsylvania School of Medicine) CRISPR/Cas9, a powerful genome editing tool, is showing promise for efficient correction of disease-causing mutations. For the first time, researchers have developed a dual gene therapy approach to deliver key components of a CRISPR/Cas9-mediated gene targeting system to mice to treat hemophilia B. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 30, 2016 Category: Global & Universal Source Type: news

Researchers develop bioresorbable capsule for treating hemophilia
Researchers from the Cockrell School of Engineering at the University of Texas at Austin have developed a biodegradable capsule to deliver a protein therapy for hemophilia. The research, published in the International Journal of Pharmaceutics, could provide a cheaper, less painful alternative to injections or infusions. The capsule contains micro and nanoparticles which carry a protein therapy to treat hemophilia B. The disease is caused by a missing or defective factor IX, a clotting protein, in the blood. Access to the protein therapy is limited due to its cost and the complications brought on by needle-based drug d...
Source: Mass Device - November 28, 2016 Category: Medical Equipment Authors: Sarah Faulkner Tags: Drug-Device Combinations Pharmaceuticals Research & Development University of Texas at Austin Source Type: news

First-ever capsule to treat hemophilia developed
In the near future, hemophiliacs could be able to treat their disease by simply swallowing a capsule. Thanks to a new breakthrough, treatment for hemophilia can now be administered via a biodegradable system, a capsule, giving people affected by the hereditary bleeding disorder hope for a less expensive, less painful treatment option than conventional injections or infusions. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 28, 2016 Category: Science Source Type: news

CHMP Backs Lonoctocog Alfa (Afstyla) for Hemophilia A CHMP Backs Lonoctocog Alfa (Afstyla) for Hemophilia A
A European Medicines Agency committee has recommended marketing authorization for lonoctocog alfa for the prevention and treatment of bleeding in patients of all ages with hemophilia A.International Approvals (Source: Medscape Hematology-Oncology Headlines)
Source: Medscape Hematology-Oncology Headlines - November 12, 2016 Category: Cancer & Oncology Tags: Hematology-Oncology News Alert Source Type: news

Roche Hemophilia Drug Stumbles in Trials
Four patients experienced serious adverse events during a clinical trial of Roche's experimental hemophilia med, ACE910. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - November 3, 2016 Category: Pharmaceuticals Source Type: news

Adverse events in trial dent hopes for Roche hemophilia drug
LONDON (Reuters) - Four patients have suffered serious adverse events in a clinical trial of an experimental hemophilia medicine from Roche, the Swiss group said on Wednesday, clouding prospects for its potential blockbuster product. (Source: Reuters: Health)
Source: Reuters: Health - November 2, 2016 Category: Consumer Health News Tags: healthNews Source Type: news

Swissmedic approves Sobi ’s Alprolix to treat haemophilia B
The Swiss Agency for Therapeutic Products, Swissmedic, has approved Sobi ’s Alprolix (eftrenonacog alfa) for the treatment of haemophilia B. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - October 31, 2016 Category: Pharmaceuticals Source Type: news

Device inspired by MUSSELS plugs its own puncture wound after an injection
Scientists from Jeonju University, Seoul, said those most likely to benefit from the invention are people with whose blood does not clot properly such as people with haemophilia and diabetes. (Source: the Mail online | Health)
Source: the Mail online | Health - October 3, 2016 Category: Consumer Health News Source Type: news

Falling and fall risk in adult patients with severe haemophilia - Rehm H, Schmolders J, Koob S, Bornemann R, Goldmann G, Oldenburg J, Pennekamp P, Strauss AC.
The objective of this study was to define fall rates and to identify possible fall risk factors in adult patients with severe haemophilia. PATIENTS, MATERIAL, METHODS: 147 patients with severe haemophilia A and B were evaluated using a standardized test ba... (Source: SafetyLit)
Source: SafetyLit - September 30, 2016 Category: Global & Universal Tags: Economics of Injury and Safety, PTSD, Injury Outcomes Source Type: news

Tapping evolution to improve biotech products
Scientists can improve protein-based drugs by reaching into the evolutionary past, a new paper proposes. As a proof of concept for this approach, the research team showed how " ancestral sequence reconstruction " or ASR can guide engineering of the blood clotting protein known as factor VIII, which is deficient in the inherited disorder hemophilia A. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - September 26, 2016 Category: Science Source Type: news

What 1989 And The Golden Girls Tell Us About Medicine Today
Today, 1989 may be most associated with Taylor Swift: It is the album that won her a second Grammy for Album of the Year. Not only that, it happens to be the year Swift was born--such a long, long time ago! People under 35 have no personal memory of 1980s pop culture, which is ironic since Swift's album in part pays homage to it. In the real 1989 (no offense to Swift and the 10 co-producers who made the album), all sorts of revolutions took place: Mr. Gorbachev tore down that pesky wall, for example. America's greatest antagonist, the Soviet Union, collapsed in 1989. Brazil conducted its first democrat...
Source: Healthy Living - The Huffington Post - September 23, 2016 Category: Consumer Health News Source Type: news

Pharma companies air concerns over plans to cap prices
If finalised, the regulator will be able to cap prices of several essential medicines, including those to treat poisoning, cardiovascular diseases and haemophilia. (Source: The Economic Times Healthcare and Biotech News)
Source: The Economic Times Healthcare and Biotech News - September 8, 2016 Category: Pharmaceuticals Source Type: news

Amid takeover rumors, Biogen gives details on hemophilia spinoff
As acquisition rumors continue to swirl around Biogen (Nasdaq: BIIB), the drug maker disclosed more details about its planned hemophilia spinoff. The company will be called Bioverativ and trade under the symbol BIVV on the Nasdaq Stock Market. The speciality drug company will focus on the discovery, research, development and commercialization of treatments for hemophilia and other blood disorders and be led by John Cox, Biogen’s former ex ecutive vice president of Pharmaceutical Operations and… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - August 15, 2016 Category: Biotechnology Authors: Jason deBruyn Source Type: news

Biogen Announces New Hemophilia Spinoff Name
The hemophilia drug business that Biogen Inc. plans to spin off as a separate company early next year will be named Bioverativ. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - August 10, 2016 Category: Pharmaceuticals Source Type: news

Shire Scraps Experimental Hemophilia Gene Therapy
Shire has discontinued development of BAX 335, an experimental gene therapy for hemophilia B, inherited earlier this year in Shire's acquisition of Baxalta. (Source: PharmaManufacturing.com)
Source: PharmaManufacturing.com - August 4, 2016 Category: Pharmaceuticals Source Type: news

The Economist explains: Why gene-therapy drugs are so expensive
ON AUGUST 3rd the British pharmaceutical company GSK said that it would charge €594,000 ($665,000) for a gene-therapy cure for ADA-SCID—a severe immune disorder that is usually fatal in the first few years of life. A child born with ADA-SCID is unable to fight off everyday infections; Strimvelis has cured this in each of the 18 children it has been tested on over 15 years. Gene therapies work by delivering correct versions of DNA, usually using a virus as a vector. Once DNA is inside the cell, it produces the protein that was missing and the fault is fixed. Scientists have been trying to develop gene therapies f...
Source: Biotechnology - August 4, 2016 Category: Biotechnology Source Type: news

Roche’s emicizumab continued to show promising safety and efficacy profile in long-term study in people with severe haemophilia A
Roche announced yesterday new data for investigational haemophilia medicine emicizumab (ACE910) at the World Federation of Hemophilia 2016 World Congress. (Source: Roche Investor Update)
Source: Roche Investor Update - July 28, 2016 Category: Pharmaceuticals Source Type: news

Roche ’s emicizumab continued to show promising safety and efficacy profile in long-term study in people with severe haemophilia A
Roche announced yesterday new data for investigational haemophilia medicine emicizumab (ACE910) at the World Federation of Hemophilia 2016 World Congress. (Source: Roche Investor Update)
Source: Roche Investor Update - July 28, 2016 Category: Pharmaceuticals Source Type: news

Hemophilia guideline recommends integrated care model
ORLANDO – An integrated care model that includes a hematologist, a specialized hematology nurse, a physical therapist, a social worker, and 24/7 access to a specialized coagulation laboratory is... (Source: Family Practice News)
Source: Family Practice News - July 27, 2016 Category: Primary Care Source Type: news

BioMarin Gene Therapy Yields Dramatic Results In Hemophilia
A new gene therapy for hemophilia, a deadly disease in which blood does not clot, may allow most patients to live normal lives. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - July 27, 2016 Category: Pharmaceuticals Authors: Matthew Herper Source Type: news

Hemophilia drugs top Medicaid spending per prescription
Medicaid’s three most expensive drugs by spending per prescription were for treatment of hemophilia, with the cost leader coming in at almost $59,000, according to an analysis by the Kaiser Family... (Source: Clinical Neurology News)
Source: Clinical Neurology News - July 23, 2016 Category: Neurology Source Type: news

Hemophilia drugs top Medicaid spending per prescription
Medicaid’s three most expensive drugs by spending per prescription were for treatment of hemophilia, with the cost leader coming in at almost $59,000, according to an analysis by the Kaiser Family... (Source: Family Practice News)
Source: Family Practice News - July 23, 2016 Category: Primary Care Source Type: news

Biogen and Sobi to release data from Phase III extension studies of haemophilia therapies
Biogen and Swedish Orphan Biovitrum (Sobi) will jointly release data of the Phase III clinical extension studies, B-YOND and ASPIRE of Elocta and Alprolix, to treat haemophilia A and haemophilia B respectively. (Source: Drug Development Technology)
Source: Drug Development Technology - July 20, 2016 Category: Pharmaceuticals Source Type: news

UNC professor identifies research priorities for National Institutes of Health
(University of North Carolina Health Care) Nigel Key, MB, ChB, FRCP, director of the UNC Hemophilia and Thrombosis Center, is lead author of an advisory opinion on research priorities to address VTE in cancer patients. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 13, 2016 Category: Global & Universal Source Type: news

Swissmedic grants approval for Sobi’s Elocta to treat haemophilia A
Specialty healthcare company Swedish Orphan Biovitrum (Sobi) has received approval from the Swiss Agency for Therapeutic Products, Swissmedic, for its Elocta (rFVIIIFc) to treat patients affected with haemophilia A. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - June 28, 2016 Category: Pharmaceuticals Source Type: news

Hemophilia
(Source: eMedicineHealth.com)
Source: eMedicineHealth.com - June 27, 2016 Category: Journals (General) Source Type: news

Baby Boomers With Hemophilia Didn't Expect To Grow Old
A generation that survived life-threatening bleeds, the HIV epidemic and Hepatitis C now nears retirement with an illness that can mostly be safely managed at home — for about $250,000 a year. (Source: NPR Health and Science)
Source: NPR Health and Science - June 20, 2016 Category: Consumer Health News Authors: Lesley McClurg Source Type: news

‘My mum has Alzheimer’s and I have haemophilia’: why homecare matters
Omar Ramadan has severe haemophilia – a disorder that impairs the body’s ability to control blood clotting – and he cares for his mother who has Alzheimer’s. Despite all this, he’s a cheerful and positive man, and last week he took the time to travel to an event to deliver a speech to a room full of people. In the local council building in Tower Hamlets, Omar took to the floor and explained how grateful he is to the homecare workers who visit him and his mum. Before he had his homecare service set up Omar had difficulty with most daily tasks – shopping, personal hygiene, domestic jo...
Source: UNISON Health care news - June 16, 2016 Category: UK Health Authors: ellisro Tags: Article Magazine ethical care charter save care now Source Type: news

Idelvion for the Treatment of Haemophilia B
Idelvion (Coagulation Factor IX (Recombinant), Albumin Fusion Protein) is an injectable formulation developed by CSL Behring, for the treatment of haemophilia B in children and adults. (Source: Drug Development Technology)
Source: Drug Development Technology - June 14, 2016 Category: Pharmaceuticals Source Type: news

Study suggests another look at common treatments for hemophilia
Participants who received a recombinant therapy— the present standard in the United States — developed antibodies or “inhibitors” to the treatments at almost twice the rate as those whose treatments were made from human plasma, new research shows. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - June 13, 2016 Category: Science Source Type: news

Kovaltry (Anti-haemophilic Factor VII (Recombinant)) for the Treatment of Haemophilia A
Kovaltry (Anti haemophilic Factor VII (Recombinant)) is developed by Bayer as the treatment for children and adult patients with haemophilia A. (Source: Drug Development Technology)
Source: Drug Development Technology - June 8, 2016 Category: Pharmaceuticals Source Type: news

CSL Behring receives FDA approval to treat haemophilia A
CSL Behring has received approval from the US Food and Drug Administration (FDA) for the use of its recombinant factor VIII single-chain therapy, Afstyla, as a treatment for haemophilia A. (Source: Pharmaceutical Technology)
Source: Pharmaceutical Technology - May 27, 2016 Category: Pharmaceuticals Source Type: news

[Policy Forum] Paying for future success in gene therapy
Imagine a young man with hemophilia A who no longer has to self-administer factor VIII replacement; an individual with sickle cell disease who is free of chronic pain and intermittent crises; a girl functionally blind since the age of 5 who can now see; or a baby rescued from a fatal, inherited neurodegenerative disease. For decades, gene therapy has tantalized us with such futuristic scenarios. However, these goals are now coming into focus, and it is the time to consider some of the consequences of success. Authors: Stuart H. Orkin, Philip Reilly (Source: ScienceNOW)
Source: ScienceNOW - May 27, 2016 Category: Science Authors: Stuart H. Orkin Tags: Medicine Source Type: news

FDA Approves Afstyla for Adults, Children With Hemophilia AFDA Approves Afstyla for Adults, Children With Hemophilia A
The US Food and Drug Administration has approved Afstyla for adults and children with hemophilia A for routine prophylaxis and on-demand treatment of bleeding episodes. FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - May 26, 2016 Category: Consumer Health News Tags: Hematology-Oncology News Alert Source Type: news

2 New Findings Offer Hope for Those with Severe Hemophilia
One pinpoints when standard therapy works best, while the other uncovers the promise of a new drug Source: HealthDay Related MedlinePlus Pages: Hemophilia, Medicines (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - May 26, 2016 Category: Consumer Health News Source Type: news

FDA Approves Afstyla (Antihemophilic Factor (Recombinant), Single Chain) for Hemophilia A
KING OF PRUSSIA, Pa. — 26 May 2016 -- CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has approved Afstyla [Antihemophilic Factor (Recombinant), Single Chain], its novel long-lasting recombinant factor VIII... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - May 26, 2016 Category: Drugs & Pharmacology Source Type: news

New findings offer hope for those with severe hemophilia
Amy Norton, HealthDay News New studies could pave the way to major changes in how doctors treat severe cases of hemophilia -- a rare genetic disorder that can cause uncontrolled bleeding. (Source: Health News - UPI.com)
Source: Health News - UPI.com - May 26, 2016 Category: Consumer Health News Source Type: news

Your NEJM Group Today: Cancer Drug Development, Hemophilia, New Jersey IM Opportunity (FREE)
By the Editors NEJM Group offers so many valuable resources for practicing clinicians. Here's what we chose for you today:NEJM Audio Interview: Rethinking Oncology Trials: Dr. Alice Shaw discusses … (Source: Physician's First Watch current issue)
Source: Physician's First Watch current issue - May 26, 2016 Category: Primary Care Source Type: news

In hemophilia A, emicizumab cuts bleeding; plasma-derived factor VIII less likely to trigger antibodies
Patients with severe hemophilia A treated with plasma-derived factor VIII, as compared with recombinant factor VIII, had a lower risk of developing neutralizing antibodies, based on a recent report... (Source: Pediatric News)
Source: Pediatric News - May 25, 2016 Category: Journals (General) Source Type: news

2 New Findings Offer Hope for Those With Severe Hemophilia
WEDNESDAY, May 25, 2016 -- Two new studies could pave the way to major changes in how doctors treat severe cases of hemophilia -- a rare genetic disorder that can cause uncontrolled bleeding. Both studies tackle a key challenge: Up to one-third of... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - May 25, 2016 Category: Journals (General) Source Type: news

Stebbins, Cocohoba leading specialty drug study with Walgreens
So-called specialty drugs can represent lifesaving advances in the treatment of serious complex conditions, such as cancers, hepatitis C, rheumatoid arthritis, multiple sclerosis, and inflammatory bowel disease. They can turn once routinely lethal diseases such as HIV and cystic fibrosis into manageable chronic conditions. They can address rare genetic conditions, such as hemophilia, or suppress immune rejection after organ transplants. (Source: UCSF School of Pharmacy News)
Source: UCSF School of Pharmacy News - May 24, 2016 Category: Universities & Medical Training Authors: Paula Joyce Source Type: news

Facial and Scalp Swelling in Children With HemophiliaFacial and Scalp Swelling in Children With Hemophilia
Two pediatric patients present with swelling of the cheek and scalp. Find out how these cases were diagnosed and learn what the treatment was. ePlasty, Open Access Journal of Plastic Surgery (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - May 24, 2016 Category: Consumer Health News Tags: Plastic Surgery & Aesthetic Medicine Journal Article Source Type: news

Hemophilia Products Do Not Raise Antibody Risk, Says EMAHemophilia Products Do Not Raise Antibody Risk, Says EMA
The evidence does not indicate that two recombinant factor VIII products from Bayer increase the risk for inhibitors that render treatment ineffective, EU regulators said in a meta-analysis. News Alerts (Source: Medscape Internal Medicine Headlines)
Source: Medscape Internal Medicine Headlines - May 13, 2016 Category: Internal Medicine Tags: Hematology-Oncology News Alert Source Type: news