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Hemophilia B Topic Center

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News
Study Finds No Treatment-Related Adverse Events in Boys Treated With Purified FIX Concentrate
05/09/2024
Jolynn Tumolo
Purified factor IX (FIX) concentrate was effective in treating and preventing bleeding episodes in real-world pediatric patients 6 years and younger with hemophilia B, according to a study published in the Journal of Blood Medicine.
Purified factor IX (FIX) concentrate was effective in treating and preventing bleeding episodes in real-world pediatric patients 6 years and younger with hemophilia B, according to a study published in the Journal of Blood Medicine.
Purified factor IX (FIX)...
05/09/2024
First Report Managed Care
News
Rebalancing Coagulation Pathways and Human Factor 9 Gene Knock-In Strategy Identified for Hemophilia B
04/25/2024

Grace Taylor

Grace Taylor
A study published in Molecular Therapy Nucleic Acids highlights a treatment strategy for treating hemophilia B by combining rebalancing coagulation pathways and human factor 9 (hF9) gene knock-in (KI) using a lipid nanoparticle and...
A study published in Molecular Therapy Nucleic Acids highlights a treatment strategy for treating hemophilia B by combining rebalancing coagulation pathways and human factor 9 (hF9) gene knock-in (KI) using a lipid nanoparticle and...
A study published in Molecular...
04/25/2024
First Report Managed Care
News
Antibody Test Determines Eligibility for Hemophilia Gene Therapy
12/11/2023
Jolynn Tumolo
The US Food and Drug Administration’s recent approval of the AAV5 DetectCDx test marked the agency’s first-ever approval of a companion diagnostic test for a gene therapy product.
The US Food and Drug Administration’s recent approval of the AAV5 DetectCDx test marked the agency’s first-ever approval of a companion diagnostic test for a gene therapy product.
The US Food and Drug...
12/11/2023
First Report Managed Care
News
Patient Education on AAV Vectors for Hemophilia Essential to Prevent ‘Buyer’s Remorse’
11/10/2023
Jolynn Tumolo
Having long served as the model disorders for the development of gene therapy, hemophilia A (HA) and hemophilia B (HB) are currently benefitting from the role with the emergence of adeno-associated viral (AAV) vectors, according to a review...
Having long served as the model disorders for the development of gene therapy, hemophilia A (HA) and hemophilia B (HB) are currently benefitting from the role with the emergence of adeno-associated viral (AAV) vectors, according to a review...
Having long served as the model...
11/10/2023
First Report Managed Care
News
Changing Trends in Hemophilia Treatment
10/27/2023
Julie Gould
“With other novel therapies (including gene therapy) entering the US market, it is important to document the variability in existing prescribing practices among broad-based cohorts of clinicians,” explain study authors.
“With other novel therapies (including gene therapy) entering the US market, it is important to document the variability in existing prescribing practices among broad-based cohorts of clinicians,” explain study authors.
“With other novel therapies...
10/27/2023
First Report Managed Care
News
Gene Therapy Superior to Factor IX Replacement for Hemophilia B
04/17/2023
Jolynn Tumolo
Findings from a phase 3 trial suggest gene therapy was superior to prophylactic factor IX for decreasing bleeding in patients with hemophilia B.
Findings from a phase 3 trial suggest gene therapy was superior to prophylactic factor IX for decreasing bleeding in patients with hemophilia B.
Findings from a phase 3 trial...
04/17/2023
First Report Managed Care
News
For Most Patients, Factor Replacement Likely Unnecessary Up to 25 Years After Gene Therapy for Hemophilia B
03/02/2023
Jolynn Tumolo
Predictive analyses suggest a majority of patients with hemophilia B likely would achieve durable clotting factor levels after a single infusion of etranacogene dezaparvovec.
Predictive analyses suggest a majority of patients with hemophilia B likely would achieve durable clotting factor levels after a single infusion of etranacogene dezaparvovec.
Predictive analyses suggest a...
03/02/2023
First Report Managed Care
Ajeet Gajra, MD, FACS
Videos
Survey Assesses Community Oncologists/Hematologists’ Perceptions of Gene Therapy for Hematological Disorders
01/21/2022
Ajeet Gajra, MD, FACS, discusses survey results analyzing community oncologists/hematologists’ perceptions of gene therapies and barriers to adoption or integration into clinical practice.
Ajeet Gajra, MD, FACS, discusses survey results analyzing community oncologists/hematologists’ perceptions of gene therapies and barriers to adoption or integration into clinical practice.
Ajeet Gajra, MD, FACS, discusses...
01/21/2022
Journal of Clinical Pathways
From Journal of Clinical Pathways
First Report
Decreased Medication, Utilization Costs Identified With Emicizumab-kxwh Therapy for Hemophilia
08/31/2020

Edan Stanley

Edan Stanley
Emicizumab-kxwh, which was approved by the FDA in November 2017 to prevent or reduce the frequency of bleeding episodes in patients with hemophilia (HEM) A and FVIII inhibitors, was found to be associated with decreased medication and...
Emicizumab-kxwh, which was approved by the FDA in November 2017 to prevent or reduce the frequency of bleeding episodes in patients with hemophilia (HEM) A and FVIII inhibitors, was found to be associated with decreased medication and...
Emicizumab-kxwh, which was...
08/31/2020
First Report Managed Care
Formulary Frontlines®
Innovations in Managing Hemophilia
08/26/2019

Eileen Koutnik-Fotopoulos

Eileen Koutnik-Fotopoulos
Although hemophilia is designated an orphan disease by the FDA because it affects fewer than 200,000 people in the United States, it imposes a significant financial and burden on patients, caregivers, and society.
Although hemophilia is designated an orphan disease by the FDA because it affects fewer than 200,000 people in the United States, it imposes a significant financial and burden on patients, caregivers, and society.
Although hemophilia is...
08/26/2019
First Report Managed Care
News
ICER Report Finds Hemophilia Biosimilar Cost-Effective
01/29/2018
A recent cost-effectiveness report from ICER examined the value of Hemlibra (emicizumab-kxwh; Genentech), a biosimilar that treats hemophilia A.
A recent cost-effectiveness report from ICER examined the value of Hemlibra (emicizumab-kxwh; Genentech), a biosimilar that treats hemophilia A.
A recent cost-effectiveness...
01/29/2018
First Report Managed Care

Newsfeed

News
Study Finds No Treatment-Related Adverse Events in Boys Treated With Purified FIX Concentrate
05/09/2024
Jolynn Tumolo
Purified factor IX (FIX) concentrate was effective in treating and preventing bleeding episodes in real-world pediatric patients 6 years and younger with hemophilia B, according to a study published in the Journal of Blood Medicine.
Purified factor IX (FIX) concentrate was effective in treating and preventing bleeding episodes in real-world pediatric patients 6 years and younger with hemophilia B, according to a study published in the Journal of Blood Medicine.
Purified factor IX (FIX)...
05/09/2024
First Report Managed Care
News
Rebalancing Coagulation Pathways and Human Factor 9 Gene Knock-In Strategy Identified for Hemophilia B
04/25/2024

Grace Taylor

Grace Taylor
A study published in Molecular Therapy Nucleic Acids highlights a treatment strategy for treating hemophilia B by combining rebalancing coagulation pathways and human factor 9 (hF9) gene knock-in (KI) using a lipid nanoparticle and...
A study published in Molecular Therapy Nucleic Acids highlights a treatment strategy for treating hemophilia B by combining rebalancing coagulation pathways and human factor 9 (hF9) gene knock-in (KI) using a lipid nanoparticle and...
A study published in Molecular...
04/25/2024
First Report Managed Care
News
Antibody Test Determines Eligibility for Hemophilia Gene Therapy
12/11/2023
Jolynn Tumolo
The US Food and Drug Administration’s recent approval of the AAV5 DetectCDx test marked the agency’s first-ever approval of a companion diagnostic test for a gene therapy product.
The US Food and Drug Administration’s recent approval of the AAV5 DetectCDx test marked the agency’s first-ever approval of a companion diagnostic test for a gene therapy product.
The US Food and Drug...
12/11/2023
First Report Managed Care
News
Patient Education on AAV Vectors for Hemophilia Essential to Prevent ‘Buyer’s Remorse’
11/10/2023
Jolynn Tumolo
Having long served as the model disorders for the development of gene therapy, hemophilia A (HA) and hemophilia B (HB) are currently benefitting from the role with the emergence of adeno-associated viral (AAV) vectors, according to a review...
Having long served as the model disorders for the development of gene therapy, hemophilia A (HA) and hemophilia B (HB) are currently benefitting from the role with the emergence of adeno-associated viral (AAV) vectors, according to a review...
Having long served as the model...
11/10/2023
First Report Managed Care
News
Changing Trends in Hemophilia Treatment
10/27/2023
Julie Gould
“With other novel therapies (including gene therapy) entering the US market, it is important to document the variability in existing prescribing practices among broad-based cohorts of clinicians,” explain study authors.
“With other novel therapies (including gene therapy) entering the US market, it is important to document the variability in existing prescribing practices among broad-based cohorts of clinicians,” explain study authors.
“With other novel therapies...
10/27/2023
First Report Managed Care
News
Gene Therapy Superior to Factor IX Replacement for Hemophilia B
04/17/2023
Jolynn Tumolo
Findings from a phase 3 trial suggest gene therapy was superior to prophylactic factor IX for decreasing bleeding in patients with hemophilia B.
Findings from a phase 3 trial suggest gene therapy was superior to prophylactic factor IX for decreasing bleeding in patients with hemophilia B.
Findings from a phase 3 trial...
04/17/2023
First Report Managed Care
News
For Most Patients, Factor Replacement Likely Unnecessary Up to 25 Years After Gene Therapy for Hemophilia B
03/02/2023
Jolynn Tumolo
Predictive analyses suggest a majority of patients with hemophilia B likely would achieve durable clotting factor levels after a single infusion of etranacogene dezaparvovec.
Predictive analyses suggest a majority of patients with hemophilia B likely would achieve durable clotting factor levels after a single infusion of etranacogene dezaparvovec.
Predictive analyses suggest a...
03/02/2023
First Report Managed Care
News
ICER Report Finds Hemophilia Biosimilar Cost-Effective
01/29/2018
A recent cost-effectiveness report from ICER examined the value of Hemlibra (emicizumab-kxwh; Genentech), a biosimilar that treats hemophilia A.
A recent cost-effectiveness report from ICER examined the value of Hemlibra (emicizumab-kxwh; Genentech), a biosimilar that treats hemophilia A.
A recent cost-effectiveness...
01/29/2018
First Report Managed Care
News
FDA Approves Long-Acting Hemophilia B Treatment
03/07/2016
The FDA approved Idelvion (coagulation factor IX [recombinant], albumin fusion protein; CSL Behring) for use in children and adults with hemophilia B. This is the first coagulation factor-albumin fusion protein product and the second factor...
The FDA approved Idelvion (coagulation factor IX [recombinant], albumin fusion protein; CSL Behring) for use in children and adults with hemophilia B. This is the first coagulation factor-albumin fusion protein product and the second factor...
The FDA approved Idelvion...
03/07/2016
First Report Managed Care
News
Gene Therapy Found Effective in Hemophilia B
11/19/2014
Ten patients with severe hemophilia B have remained cured of the inherited bleeding disorder for as long as 3 years due to gene therapy.
Ten patients with severe hemophilia B have remained cured of the inherited bleeding disorder for as long as 3 years due to gene therapy.
Ten patients with severe...
11/19/2014
First Report Managed Care
News
Keratoconus Appears to be Associated With Demodex Mites
05/16/2024
Jolynn Tumolo
Keratoconus, an eye disorder that leads to gradual curvature of the cornea, may be associated with Demodex infestation, suggests a small study published in the Oman Journal of Ophthalmology.
Keratoconus, an eye disorder that leads to gradual curvature of the cornea, may be associated with Demodex infestation, suggests a small study published in the Oman Journal of Ophthalmology.
Keratoconus, an eye disorder...
05/16/2024
First Report Managed Care
News
Hospital Costs May Be Higher for Patients With Private Insurance
05/14/2024
Hannah Musick
A recent research report reveals that individuals with private health insurance pay 254% more for hospital procedures than Medicare rates, leading to potentially increased costs for millions of Americans.
A recent research report reveals that individuals with private health insurance pay 254% more for hospital procedures than Medicare rates, leading to potentially increased costs for millions of Americans.
A recent research report reveals...
05/14/2024
First Report Managed Care
News
Study Finds No Treatment-Related Adverse Events in Boys Treated With Purified FIX Concentrate
05/09/2024
Jolynn Tumolo
Purified factor IX (FIX) concentrate was effective in treating and preventing bleeding episodes in real-world pediatric patients 6 years and younger with hemophilia B, according to a study published in the Journal of Blood Medicine.
Purified factor IX (FIX) concentrate was effective in treating and preventing bleeding episodes in real-world pediatric patients 6 years and younger with hemophilia B, according to a study published in the Journal of Blood Medicine.
Purified factor IX (FIX)...
05/09/2024
First Report Managed Care
News
Demodex Infestation Found in 9 of 10 Eye Clinic Patients Using 2-Minute Technique
05/09/2024
Jolynn Tumolo
Demodex mites were found in the eyelashes of 94% of patients examined with a simple diagnostic technique at a standard ophthalmic practice, according to a study published online in the journal Clinical Ophthalmology.
Demodex mites were found in the eyelashes of 94% of patients examined with a simple diagnostic technique at a standard ophthalmic practice, according to a study published online in the journal Clinical Ophthalmology.
Demodex mites were found in the...
05/09/2024
First Report Managed Care
News
Debate Emerges Over FDA's Plan to Regulate Laboratory Medical Tests
05/03/2024
Hannah Musick
The FDA's new regulations on laboratory-developed tests have sparked debate over access to innovative tests and patient care in the medical field, with opponents arguing that increased oversight could hinder the development of crucial new...
The FDA's new regulations on laboratory-developed tests have sparked debate over access to innovative tests and patient care in the medical field, with opponents arguing that increased oversight could hinder the development of crucial new...
The FDA's new regulations on...
05/03/2024
First Report Managed Care
Conference Coverage
What Does Value Mean to You: Value-Based Contracting Expert Insights
05/02/2024
Danielle Sposato
A panel at Asembia 2024 delved into the complexities of value-based contracting in health care, emphasizing the need for collaboration among stakeholders to address key issues and improving access to medicines for patients.
A panel at Asembia 2024 delved into the complexities of value-based contracting in health care, emphasizing the need for collaboration among stakeholders to address key issues and improving access to medicines for patients.
A panel at Asembia 2024 delved...
05/02/2024
First Report Managed Care
Conference Coverage
Expanding Pharmacists’ Role in Providing Clinical Care: Addressing Public Health Challenges
05/02/2024
Danielle Sposato
Panelists from various backgrounds discuss the critical role of pharmacists in addressing public health challenges such as HIV/AIDS and advocating for expanded clinical care and reimbursement pathways to improve health outcomes in communities.
Panelists from various backgrounds discuss the critical role of pharmacists in addressing public health challenges such as HIV/AIDS and advocating for expanded clinical care and reimbursement pathways to improve health outcomes in communities.
Panelists from various...
05/02/2024
First Report Managed Care
Conference Coverage
Addressing Cost & Accessibility for Innovative Medicines: GLP-1s and Beyond
05/02/2024
Danielle Sposato
The rapid pace of innovation, rising costs of prescription drugs, and the need for transparency to ensure access and affordability in the pharmaceutical industry are discussed in a session at Asembia 2024.
The rapid pace of innovation, rising costs of prescription drugs, and the need for transparency to ensure access and affordability in the pharmaceutical industry are discussed in a session at Asembia 2024.
The rapid pace of innovation,...
05/02/2024
First Report Managed Care
Conference Coverage
Rare Orphan, Oncology, and Specialty Drugs in Development: Review of Therapeutic Areas, Designations, and Launch Projections
05/01/2024
Danielle Sposato
The specialty pharmacy pipeline was the subject of one of many informative sessions at the annual Asembia Summit this year. Key insights were shared about current drugs in the pipeline according to their drug designations, the phase of each...
The specialty pharmacy pipeline was the subject of one of many informative sessions at the annual Asembia Summit this year. Key insights were shared about current drugs in the pipeline according to their drug designations, the phase of each...
The specialty pharmacy pipeline...
05/01/2024
First Report Managed Care
Conference Coverage
Specialty Pharmaceuticals Pipeline: Biologic and Biosimilar Approvals and Projected Developments
04/29/2024
Hannah Musick
Explore the latest trends and developments in the specialty pharmaceutical pipeline, including key legal matters, FDA issues, notable approvals, and potential biosimilar savings projected for the next decade as shared in a 2024 AMCP session.
Explore the latest trends and developments in the specialty pharmaceutical pipeline, including key legal matters, FDA issues, notable approvals, and potential biosimilar savings projected for the next decade as shared in a 2024 AMCP session.
Explore the latest trends and...
04/29/2024
First Report Managed Care

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