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Revolutionary Single-Dose Gene Therapy Offers New Hope for Adults with Hemophilia B
11/08/2024
A groundbreaking gene therapy for hemophilia B has shown remarkable success in reducing bleeding episodes and improving the quality of life for adult patients, according to a recent international phase III clinical trial.
A groundbreaking gene therapy for hemophilia B has shown remarkable success in reducing bleeding episodes and improving the quality of life for adult patients, according to a recent international phase III clinical trial.
A groundbreaking gene therapy...
11/08/2024
First Report Managed Care
News
New 4F-PCC Proves Effective in Rapid Reversal of Anticoagulation for Urgent Surgery, Clinical Trial Finds
09/10/2024
An investigational 4-factor prothrombin complex concentrate (4F-PCC) is as effective and safe as an FDA-approved 4F-PCC for rapidly reversing vitamin K antagonist (VKA) anticoagulation in patients requiring urgent surgery, according to a...
An investigational 4-factor prothrombin complex concentrate (4F-PCC) is as effective and safe as an FDA-approved 4F-PCC for rapidly reversing vitamin K antagonist (VKA) anticoagulation in patients requiring urgent surgery, according to a...
An investigational 4-factor...
09/10/2024
First Report Managed Care
News
Fitusiran Prophylaxis Shows Promise for Protection Against Bleeding for Patients With Hemophilia A or B
06/05/2024
A phase 3 study evaluated the efficacy and safety of fitusiran in males with hemophilia A or B who were receiving bypassing agent (BPA)/clotting factor concentrate (CFC) prophylaxis.
A phase 3 study evaluated the efficacy and safety of fitusiran in males with hemophilia A or B who were receiving bypassing agent (BPA)/clotting factor concentrate (CFC) prophylaxis.
A phase 3 study evaluated the...
06/05/2024
First Report Managed Care
News
Study Finds No Treatment-Related Adverse Events in Boys Treated With Purified FIX Concentrate
05/09/2024
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
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
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
“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
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
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

Newsfeed

News
Revolutionary Single-Dose Gene Therapy Offers New Hope for Adults with Hemophilia B
11/08/2024
A groundbreaking gene therapy for hemophilia B has shown remarkable success in reducing bleeding episodes and improving the quality of life for adult patients, according to a recent international phase III clinical trial.
A groundbreaking gene therapy for hemophilia B has shown remarkable success in reducing bleeding episodes and improving the quality of life for adult patients, according to a recent international phase III clinical trial.
A groundbreaking gene therapy...
11/08/2024
First Report Managed Care
News
New 4F-PCC Proves Effective in Rapid Reversal of Anticoagulation for Urgent Surgery, Clinical Trial Finds
09/10/2024
An investigational 4-factor prothrombin complex concentrate (4F-PCC) is as effective and safe as an FDA-approved 4F-PCC for rapidly reversing vitamin K antagonist (VKA) anticoagulation in patients requiring urgent surgery, according to a...
An investigational 4-factor prothrombin complex concentrate (4F-PCC) is as effective and safe as an FDA-approved 4F-PCC for rapidly reversing vitamin K antagonist (VKA) anticoagulation in patients requiring urgent surgery, according to a...
An investigational 4-factor...
09/10/2024
First Report Managed Care
News
Fitusiran Prophylaxis Shows Promise for Protection Against Bleeding for Patients With Hemophilia A or B
06/05/2024
A phase 3 study evaluated the efficacy and safety of fitusiran in males with hemophilia A or B who were receiving bypassing agent (BPA)/clotting factor concentrate (CFC) prophylaxis.
A phase 3 study evaluated the efficacy and safety of fitusiran in males with hemophilia A or B who were receiving bypassing agent (BPA)/clotting factor concentrate (CFC) prophylaxis.
A phase 3 study evaluated the...
06/05/2024
First Report Managed Care
News
Study Finds No Treatment-Related Adverse Events in Boys Treated With Purified FIX Concentrate
05/09/2024
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
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
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
“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
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
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
Using Deep Learning to Measure MS-Specific Brain Damage Beyond Aging
11/11/2024
A new study published in Neurology aimed to differentiate age-related brain changes from multiple sclerosis (MS)-specific neurodegeneration using MRI.
A new study published in Neurology aimed to differentiate age-related brain changes from multiple sclerosis (MS)-specific neurodegeneration using MRI.
A new study published in...
11/11/2024
First Report Managed Care
News
Adverse Event Data Suggest Selective β1-blockers Pose Asthma Risk
11/11/2024
The risks of asthma and asthma-like adverse events appear to be higher with selective β1-blockers than with nonselective blockers and α- and β-blockers.
The risks of asthma and asthma-like adverse events appear to be higher with selective β1-blockers than with nonselective blockers and α- and β-blockers.
The risks of asthma and...
11/11/2024
First Report Managed Care
News
RNAi Therapies Offer New Hope for Patients With Hereditary Transthyretin Amyloidosis
11/08/2024
RNA interference (RNAi)-based treatments for hereditary transthyretin amyloidosis (ATTR) have shown remarkable promise, offering new hope for patients with this rare but devastating genetic disorder.
RNA interference (RNAi)-based treatments for hereditary transthyretin amyloidosis (ATTR) have shown remarkable promise, offering new hope for patients with this rare but devastating genetic disorder.
RNA interference (RNAi)-based...
11/08/2024
First Report Managed Care
News
Revolutionary Single-Dose Gene Therapy Offers New Hope for Adults with Hemophilia B
11/08/2024
A groundbreaking gene therapy for hemophilia B has shown remarkable success in reducing bleeding episodes and improving the quality of life for adult patients, according to a recent international phase III clinical trial.
A groundbreaking gene therapy for hemophilia B has shown remarkable success in reducing bleeding episodes and improving the quality of life for adult patients, according to a recent international phase III clinical trial.
A groundbreaking gene therapy...
11/08/2024
First Report Managed Care
News
Real-World Evidence Supports Efficacy and Safety of Selexipag in Patients With Pulmonary Arterial Hypertension
11/07/2024
Real-world data shows selexipag effectively improves functional class and risk category outcomes in patients with pulmonary arterial hypertension, regardless of maintenance dose.
Real-world data shows selexipag effectively improves functional class and risk category outcomes in patients with pulmonary arterial hypertension, regardless of maintenance dose.
Real-world data shows selexipag...
11/07/2024
First Report Managed Care
News
Clinical Predictors of One-Year Seizure Freedom After SLAH
11/06/2024
A study published in Annals of Clinical and Translational Neurology aimed to predict 1-year seizure freedom for patients undergoing stereotactic laser amygdalohippocampotomy (SLAH) for mesial temporal lobe epilepsy (MTLE) using a set of...
A study published in Annals of Clinical and Translational Neurology aimed to predict 1-year seizure freedom for patients undergoing stereotactic laser amygdalohippocampotomy (SLAH) for mesial temporal lobe epilepsy (MTLE) using a set of...
A study published in Annals of...
11/06/2024
First Report Managed Care
News
Efficacy and Outcomes of Neoadjuvant Immunotherapy in Lung Cancer
11/05/2024
A meta-analysis of 43 studies suggests that neoadjuvant chemoimmunotherapy may benefit patients with resectable NSCLC and low tumor PD-L1 levels for event-free survival, showing superior outcomes compared to neoadjuvant chemotherapy.
A meta-analysis of 43 studies suggests that neoadjuvant chemoimmunotherapy may benefit patients with resectable NSCLC and low tumor PD-L1 levels for event-free survival, showing superior outcomes compared to neoadjuvant chemotherapy.
A meta-analysis of 43 studies...
11/05/2024
First Report Managed Care
News
FDA Grants Accelerated Approval for Asciminib as First-Line Treatment in Ph+CML-CP
11/01/2024
Novartis received accelerated approval by the US Food and Drug Administration (FDA) for asciminib, a medication for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+CML-CP).
Novartis received accelerated approval by the US Food and Drug Administration (FDA) for asciminib, a medication for adult patients with newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+CML-CP).
Novartis received accelerated...
11/01/2024
First Report Managed Care
News
HHS Faces Legal Action From 10 Hospitals Over 2023 Medicare Advantage Rule and Reimbursement Calculations
10/30/2024
Ten hospitals sued the US Department of Health and Human Services (HHS), claiming that they were underpaid Medicare reimbursements for serving low-income patients.
Ten hospitals sued the US Department of Health and Human Services (HHS), claiming that they were underpaid Medicare reimbursements for serving low-income patients.
Ten hospitals sued the US...
10/30/2024
First Report Managed Care
News
Impact of Immune Checkpoint Inhibitors on Multiple Sclerosis Treatment Outcomes
10/29/2024
Relapses and progression of multiple sclerosis were rare in patients treated with immune checkpoint inhibitors, according to a study presented at the American Academy of Neurology 2024 Annual Meeting.
Relapses and progression of multiple sclerosis were rare in patients treated with immune checkpoint inhibitors, according to a study presented at the American Academy of Neurology 2024 Annual Meeting.
Relapses and progression of...
10/29/2024
First Report Managed Care
FRMC Volume 21, Issue 3

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September 2024
Volume 21
Issue 3
Current Issue
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