Early-phase trials of the DTX101 gene therapy in hemophilia B showed manageable safety but insufficient efficacy, leading to program discontinuation while offering critical insights for future gene therapy development.
Early-phase trials of the DTX101 gene therapy in hemophilia B showed manageable safety but insufficient efficacy, leading to program discontinuation while offering critical insights for future gene therapy development.
A 13-year follow-up study shows that a single dose of adeno-associated virus (AAV) gene therapy offers durable factor IX expression and sustained clinical benefits in patients with severe hemophilia B.
A 13-year follow-up study shows that a single dose of adeno-associated virus (AAV) gene therapy offers durable factor IX expression and sustained clinical benefits in patients with severe hemophilia B.
A Community Counts Longitudinal Surveillance Cohort study reveals that modern therapies, treatment adherence, and social factors all play pivotal roles in reducing joint bleeding in severe hemophilia.
A Community Counts Longitudinal Surveillance Cohort study reveals that modern therapies, treatment adherence, and social factors all play pivotal roles in reducing joint bleeding in severe hemophilia.
Researchers report that a single-dose gene therapy led to sustained clotting factor levels and significant reductions in bleeding and treatment burden for patients with hemophilia B.
Researchers report that a single-dose gene therapy led to sustained clotting factor levels and significant reductions in bleeding and treatment burden for patients with hemophilia B.
New research highlights the potential of emicizumab, traditionally used to treat hemophilia A (HA), as a promising alternative therapy for certain hemophilia B (HB) genotypes.
New research highlights the potential of emicizumab, traditionally used to treat hemophilia A (HA), as a promising alternative therapy for certain hemophilia B (HB) genotypes.
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.
New guidelines from the International Society on Thrombosis and Haemostasis strongly recommend prophylactic treatment over episodic for patients with severe and moderately severe hemophilia A and B, with conditional recommendations for other...
New guidelines from the International Society on Thrombosis and Haemostasis strongly recommend prophylactic treatment over episodic for patients with severe and moderately severe hemophilia A and B, with conditional recommendations for other...
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...
The Senate has done it. After weeks of behind-the-scenes wrangling and last-minute amendments, lawmakers have passed a sprawling tax-and-spending megabill that makes good on a long-standing Republican goal: making the 2017 Trump tax cuts...
The Senate has done it. After weeks of behind-the-scenes wrangling and last-minute amendments, lawmakers have passed a sprawling tax-and-spending megabill that makes good on a long-standing Republican goal: making the 2017 Trump tax cuts...
In Washington, the most consequential policies are often buried in the fine print. One such detail in the Senate Finance Committee’s latest tax and spending proposal would gradually cap provider taxes—a core funding tool states use to pull in...
In Washington, the most consequential policies are often buried in the fine print. One such detail in the Senate Finance Committee’s latest tax and spending proposal would gradually cap provider taxes—a core funding tool states use to pull in...
Over the past few weeks, I’ve been following the progress of the One Big Beautiful Bill Act (OBBBA), which started in the House with sweeping tax reforms and some eye-catching health care proposals. Now, the Senate has released its (highly...
Over the past few weeks, I’ve been following the progress of the One Big Beautiful Bill Act (OBBBA), which started in the House with sweeping tax reforms and some eye-catching health care proposals. Now, the Senate has released its (highly...
With the One Big Beautiful Bill Act (OBBBA) still in the hands of the Senate, much of the national conversation has centered on what this legislation would do to federal coverage programs. But regardless of how the federal process unfolds,...
With the One Big Beautiful Bill Act (OBBBA) still in the hands of the Senate, much of the national conversation has centered on what this legislation would do to federal coverage programs. But regardless of how the federal process unfolds,...
It’s been weeks of headline-grabbing debate over Medicaid work requirements, and the “One Big Beautiful Bill Act” remains in the Senate as they deliberate over several health care provisions. While the Medicaid provisions sparked most of the...
It’s been weeks of headline-grabbing debate over Medicaid work requirements, and the “One Big Beautiful Bill Act” remains in the Senate as they deliberate over several health care provisions. While the Medicaid provisions sparked most of the...
Last week, the House of Representatives narrowly passed the One Big Beautiful Bill Act (HR 1, 119th Congress) by a single vote, sending the sweeping tax and spending reconciliation package, backed by President Trump, to the Senate. With its...
Last week, the House of Representatives narrowly passed the One Big Beautiful Bill Act (HR 1, 119th Congress) by a single vote, sending the sweeping tax and spending reconciliation package, backed by President Trump, to the Senate. With its...
It’s been another eventful week in health policy—a phrase that’s quickly becoming routine. This time, House Republicans advanced a sweeping budget reconciliation bill, designed to implement elements of the March budget resolution, with a...
It’s been another eventful week in health policy—a phrase that’s quickly becoming routine. This time, House Republicans advanced a sweeping budget reconciliation bill, designed to implement elements of the March budget resolution, with a...
It’s been a busy few days for drug pricing policy. While I’d love to dig into the details of both President Trump’s latest executive order and the newest Republican bill that would cut Medicaid spending, we don’t have the time—and this isn’t...
It’s been a busy few days for drug pricing policy. While I’d love to dig into the details of both President Trump’s latest executive order and the newest Republican bill that would cut Medicaid spending, we don’t have the time—and this isn’t...
In the span of just 9 months, the landscape of administrative law has undergone a transformation so profound that pharmaceutical executives, health care providers, and patients alike are struggling to navigate the new terrain. The Supreme...
In the span of just 9 months, the landscape of administrative law has undergone a transformation so profound that pharmaceutical executives, health care providers, and patients alike are struggling to navigate the new terrain. The Supreme...
Over the past 2 weeks, I’ve examined the growing friction between the Section 232 pharmaceutical tariff investigations and the Medicare drug pricing reforms authorized by the Inflation Reduction Act (IRA). These 2 policy agendas—industrial...
Over the past 2 weeks, I’ve examined the growing friction between the Section 232 pharmaceutical tariff investigations and the Medicare drug pricing reforms authorized by the Inflation Reduction Act (IRA). These 2 policy agendas—industrial...
According to a recent study published online in Cureus, “Increasing education about existing AHA guidelines, including available therapies and access to expert care at hemophilia treatment centers, may help improve the outcomes of patients...
According to a recent study published online in Cureus, “Increasing education about existing AHA guidelines, including available therapies and access to expert care at hemophilia treatment centers, may help improve the outcomes of patients...
Jason Rose, CEO of AdhereHealth, shares how social determinants of health, such as access to transportation or affordable housing, significantly impact medication adherence by creating barriers for patients in terms of obtaining and taking...
Jason Rose, CEO of AdhereHealth, shares how social determinants of health, such as access to transportation or affordable housing, significantly impact medication adherence by creating barriers for patients in terms of obtaining and taking...
Reducing cost-sharing for asthma medications through a preventive drug list (PDL) increases adherence to controller medications, according to a study published in JAMA Network Open.
Reducing cost-sharing for asthma medications through a preventive drug list (PDL) increases adherence to controller medications, according to a study published in JAMA Network Open.
Even though many Medicare Advantage (MA) health plan executives braced for another year of declining performance on their Star Ratings, seeing their 2024 ratings drop for the second consecutive year has been a tough pill to swallow. Ashley...
Even though many Medicare Advantage (MA) health plan executives braced for another year of declining performance on their Star Ratings, seeing their 2024 ratings drop for the second consecutive year has been a tough pill to swallow. Ashley...
Starting in 2024, Medicare Part D enrollees could benefit from a cap placed on out-of-pocket drug spending, thanks to a provision in the Inflation Reduction Act of 2022 (IRA).
Starting in 2024, Medicare Part D enrollees could benefit from a cap placed on out-of-pocket drug spending, thanks to a provision in the Inflation Reduction Act of 2022 (IRA).