Bleeding and Clotting Disorders Archives

Gene Therapy for Hemophilia B: One Infusion, No More Weekly Treatments

Posted on March 6, 2026March 6, 2026 by Jennifer Glover

Key Takeaways:

Gene therapy for hemophilia B targets the root cause of the condition — the faulty gene — rather than replacing the missing clotting factor from outside the body. A single intravenous infusion delivers a working copy of the factor IX gene to the liver, enabling the body to produce its own clotting factor.
Cure 4 The Kids Foundation completed Nevada’s first Hemgenix infusion in 2024 and is one of only a handful of centers on the West Coast to have administered this treatment. To date, C4K has successfully completed three Hemgenix infusions.
Gene therapy is not a cure. The underlying genetic condition remains, and a person with hemophilia B can still pass the gene to their children. Long-term monitoring is essential.
Hemgenix is currently approved for adults only (18 and older). Pediatric gene therapy trials are in development but not yet available for children.
New treatment options are expanding rapidly. Beyond gene therapy, the FDA approved fitusiran (Qfitlia) in 2025 — a subcutaneous injection given once every two months that works for both hemophilia A and B, approved for patients aged 12 and older.

What Does Gene Therapy for Hemophilia Look Like in Practice?

Imagine living your entire life tethered to a treatment schedule. Every week — sometimes multiple times a week — you need an intravenous infusion of a clotting factor just to prevent a bleed that could send you to the hospital. You plan your work around it. Your vacations. Your daily routine. It has been this way since you were a child.

Now imagine a single infusion that could change all of that.

That’s not a hypothetical. It happened right here in Nevada. And it happened at C4K.

What is Gene Therapy for Hemophilia B?

Gene therapy for hemophilia B is a one-time intravenous treatment that delivers a working copy of the factor IX gene to the liver using a modified, harmless virus as a carrier. Once in place, the liver cells begin producing factor IX on their own — the clotting protein the body was never able to make before. Unlike traditional replacement therapy, which requires lifelong intravenous infusions, gene therapy addresses the genetic root cause of the bleeding disorder in a single treatment.

Why Was Nevada’s First Gene Therapy Infusion for Hemophilia a Milestone?

In 2024, Cure 4 The Kids Foundation completed Nevada’s first successful infusion of Hemgenix®, an FDA-approved gene therapy for adults with hemophilia B. C4K is one of only five centers on the West Coast that has administered this treatment — with just two additional centers currently trained to do so. Nationally, the number of sites approved to provide Hemgenix remains extremely limited, making access to this therapy a significant barrier for many patients across the country. To date, C4K has successfully completed three Hemgenix infusions.

The treatment was administered in our infusion suite under the supervision of Dr. Aimee Foord, director of Cure 4 The Kids Foundation’s Bleeding and Clotting Disorders Clinic, and Dr. Joseph Lasky, our medical director.

Our first patient was a 39-year-old man from Arizona who had lived with hemophilia B his entire life, requiring regular factor IX infusions to prevent dangerous bleeding episodes. After a single Hemgenix infusion, his body began producing its own factor IX.

More than a year later, he has maintained normal factor levels without a single additional infusion. Our subsequent patients have seen similarly promising results.

While this is a fantastic medical milestone, for this patient, it means so much more: It’s a fundamentally different life.

What Is Hemophilia, and Why Does It Require Lifelong Treatment?

To understand why gene therapy matters, it helps to understand what hemophilia is at its most basic level.

Hemophilia is a genetic bleeding disorder. People with hemophilia B are missing a working copy of the gene that tells the body how to make factor IX — a protein the blood needs to form clots. Without it, even a minor injury can lead to prolonged, sometimes dangerous bleeding. Internal bleeding, particularly around joints, can cause chronic pain and permanent damage over time.

For decades, the standard treatment has been replacement therapy: regular intravenous infusions of the missing clotting factor. It works, but it’s a lifelong commitment. Depending on severity, a patient may need infusions multiple times per week — every week — for the rest of their life.

How Does Hemgenix Gene Therapy Work?

Gene therapy takes a completely different approach. Instead of replacing the missing protein from outside the body, it addresses the root cause: the faulty gene itself.

Hemgenix works by delivering a functional copy of the factor IX gene directly to the patient’s liver cells using a modified, harmless virus as a carrier. Once the gene is in place, the liver cells begin producing factor IX on their own. The goal is for the body to take over — making the clotting factor it was never able to produce before.

It’s a single, one-time intravenous infusion. Not a daily medication. Not a weekly treatment. One infusion.

Is Gene Therapy a Cure for Hemophilia?

Gene therapy is a remarkable advancement, but it’s important to understand what it is and what it isn’t.

It is not a cure. Hemgenix does not fix or replace the original faulty gene. It adds a working copy. The underlying genetic condition remains, and a person with hemophilia B can still pass the gene to their children. Long-term monitoring is essential.

It is currently approved for adults only. Hemgenix is approved for adults aged 18 and older with hemophilia B who use factor IX prophylaxis or who have a history of serious bleeding episodes. Pediatric gene therapy trials are in development, but this treatment is not yet available for children.

Results can vary. Five-year follow-up data from the Phase 3 HOPE-B study, published in the New England Journal of Medicine, showed that Hemgenix reduced adjusted annualized bleeding rates by 63% through five years post-infusion, with mean factor IX activity levels sustained at 36.1%. In the trial, 94% of patients eliminated factor IX prophylaxis and remained free of continuous prophylaxis through four years. But individual responses differ, and ongoing research continues to track long-term outcomes.

It requires ongoing monitoring. After gene therapy, patients need regular follow-up to track factor levels, liver function, and overall response to treatment. At C4K, our team provides that long-term monitoring and support.

Where Is Gene Therapy for Bleeding Disorders Heading?

Hemgenix was the first gene therapy approved for hemophilia B (2022), and Roctavian became the first approved for hemophilia A in 2023. These approvals represented a new era in bleeding disorder treatment — one that targets the genetic root of these conditions rather than managing symptoms. (Note: BioMarin announced in early 2026 that it is withdrawing Roctavian from the market after commercial challenges. While this is a setback for hemophilia A gene therapy specifically, multiple next-generation gene therapies for hemophilia A are in active clinical development.)

The landscape is evolving rapidly. New therapies are also expanding options for patients. In March 2025, the FDA approved fitusiran (Qfitlia), a different kind of treatment that works by rebalancing the body’s clotting system rather than replacing the missing factor. It’s given as a subcutaneous injection once every two months and is approved for adults and pediatric patients aged 12 and older with hemophilia A or B — offering patients a less burdensome prophylactic option with as few as six injections per year.

Research is also underway to develop gene therapies for children, to improve the durability of factor expression over time, and to explore gene-based treatments for other bleeding disorders beyond hemophilia. The science is moving fast — and C4K is committed to staying at the leading edge of what’s available for our patients.

What Does a Life Without Weekly Infusions Look Like?

For someone who has never lived with a bleeding disorder, it’s hard to understand what weekly factor infusions mean in practice. It’s not just the time in a chair. It’s the planning. The anxiety. The constant awareness that without your next infusion, an ordinary injury could become an emergency.

For a child growing up with hemophilia, it shapes everything — what sports they play, whether they go on the school camping trip, how their parents navigate every bump and bruise with a calculation most families never have to make.

Gene therapy doesn’t erase the diagnosis. But for the patients it works for, it can remove the most burdensome part of living with it. And that changes a life.

How Can I Learn More About Gene Therapy for Hemophilia B?

If you or a family member is living with hemophilia B and you want to learn more about whether gene therapy may be an option, our Bleeding and Clotting Disorders Clinic is here to help. Dr. Aimee Foord and our hematology team can walk you through what’s available, what the process looks like, and what to expect.

To schedule a consultation, call us at (702) 732-1493.

At C4K, we believe every patient deserves access to the most advanced care available — right here in Nevada. No patient is ever turned away for financial reasons. That is our promise.

Frequently Asked Questions

Is gene therapy a cure for hemophilia B?

No. Gene therapy for hemophilia B adds a working copy of the factor IX gene but does not fix or replace the original faulty gene. The underlying genetic condition remains, and a person with hemophilia B can still pass the gene to their children. However, for many patients, gene therapy significantly reduces or eliminates the need for routine factor IX infusions.

Who is eligible for Hemgenix gene therapy?

Hemgenix is currently FDA-approved for adults aged 18 and older with hemophilia B who use factor IX prophylaxis therapy or who have a history of current or life-threatening bleeding episodes. Eligibility also depends on testing for factor IX inhibitors and liver health. Your hematologist can help determine if you’re a candidate.

How long do the effects of Hemgenix last? is von Willebrand disease diagnosed?

Five-year follow-up data from the HOPE-B clinical trial showed that factor IX activity levels remained sustained at a mean of 36.1%, and 94% of patients remained free of continuous prophylaxis through four years. Research is ongoing to track outcomes beyond five years.

Where can I get gene therapy for hemophilia B in Nevada?

Cure 4 The Kids Foundation in Las Vegas is the only center in Nevada to have administered Hemgenix gene therapy for hemophilia B. C4K is one of a limited number of centers on the West Coast offering this treatment. To learn more, contact the Bleeding and Clotting Disorders Clinic at (702) 732-1493.

Is gene therapy for hemophilia available for children?

Not yet. Hemgenix is currently approved only for adults aged 18 and older. Pediatric gene therapy trials are in development, and C4K is committed to offering the latest treatments as they become available.

What other new treatments are available for hemophilia besides gene therapy?

In March 2025, the FDA approved fitusiran (Qfitlia), a subcutaneous injection given once every two months for patients aged 12 and older with hemophilia A or B. Unlike gene therapy or factor replacement, Qfitlia works by rebalancing the body’s clotting system. Your hematologist can help you understand which treatment options are right for you.

About the Author: Annette Logan-Parker brings over 30 years of experience in pediatric oncology to her role as Founder and Chief Advocacy & Innovation Officer at Cure 4 The Kids Foundation. She has dedicated her career to improving outcomes for children with cancer and ensuring equitable access to cutting-edge treatments for all families.

She Wasn’t Clumsy. She Wasn’t Dramatic. She Had a Bleeding Disorder No One Tested For.

Posted on March 4, 2026March 4, 2026 by Jennifer Glover

Key Takeaways:

Bleeding disorders in girls are common but commonly missed. Von Willebrand disease is the most common bleeding disorder, affecting boys and girls equally — but girls are far less likely to be diagnosed.
Girls wait an average of 16 years for a diagnosis. Heavy periods, easy bruising, and prolonged nosebleeds are often dismissed as normal rather than recognized as symptoms of a bleeding disorder.
An estimated 2 million women in the U.S. may have an undiagnosed bleeding disorder. Many families don’t realize their bleeding is abnormal because other women in the family share the same undiagnosed condition.
Early diagnosis changes everything. It makes surgeries safer, periods treatable, and protects against life-threatening complications during pregnancy and childbirth.
You are not overreacting. If your daughter’s symptoms concern you, ask your pediatrician for a referral to a hematologist. At C4K, no child is ever turned away for financial reasons.

Why Bleeding Disorders in Girls Go Undiagnosed — and What Parents Can Do

She bruised easily. Everyone said she was clumsy.

Her nosebleeds wouldn’t stop. Everyone said she’d grow out of it.

Her periods left her doubled over and anemic. Everyone said that’s “just how it is for some girls.”

She wasn’t clumsy. She wasn’t dramatic. She had von Willebrand disease. And no one tested her for 16 years.

If this sounds familiar — if you’re reading this and thinking about your own daughter, your niece, or even yourself — you’re not alone. And you’re not imagining it.

What Are Bleeding Disorders, and Why Are Girls More Likely to Be Missed?

Bleeding disorders are conditions that affect the blood’s ability to clot properly. They include hemophilia, von Willebrand disease, rare factor deficiencies, platelet disorders, and more. Every March, during Bleeding Disorders Awareness Month, the bleeding disorders community comes together to raise awareness about these conditions. Since 2016, when the U.S. Department of Health and Human Services expanded what was previously Hemophilia Awareness Month, March has recognized the full spectrum of inheritable bleeding disorders. The red tie is the symbol of this community, representing the blood ties that bind us together.

At C4K, our Bleeding and Clotting Disorders Clinic, led by Dr. Aimee Foord, treats patients of all ages across this full spectrum. We see firsthand what these conditions look like in children — and we see how often they go unrecognized, especially in girls.

Most people hear “bleeding disorder” and think of hemophilia in boys. But the most common bleeding disorder isn’t hemophilia — it’s von Willebrand disease, and it affects boys and girls equally. The difference? Girls are far less likely to be diagnosed.

Here’s why that matters:

An estimated 2 million women in the U.S. may have an undiagnosed bleeding disorder. That’s not a rare condition. That’s a common one that’s commonly missed.
As many as 1 in 10 women with heavy periods may have a bleeding disorder — but most are never tested.
On average, women wait 16 years from the onset of symptoms to receive a diagnosis. They report an average of six separate bleeding symptoms before anyone connects the dots.
Fewer than 10% of adolescent girls with heavy menstrual bleeding are screened for von Willebrand disease, despite more than 15 years of screening guidelines from the American College of Obstetricians and Gynecologists.

Why Are Bleeding Disorders in Girls Dismissed as Normal?

Because girls’ bleeding symptoms look different — and they’re easier to dismiss.

When a boy with hemophilia bleeds into a joint, it’s visible and alarming. When a teenage girl soaks through a pad every hour, misses school every month, and is chronically exhausted, she’s told: “That’s just how it is for some women.” Or: “You’ll regulate eventually.” Or: “Take some ibuprofen.”

Research confirms what many families already feel: women with inherited bleeding disorders often wait well over a decade — about 14 years on average — for a diagnosis, compared to under 10 years for men, even when bleeding begins at similar ages. The problem isn’t that girls aren’t showing symptoms. It’s that their symptoms are being normalized.

There’s another layer to this that we see in our clinic: many families don’t recognize heavy periods as abnormal because other women in the family have the same experience, unknowingly sharing the same undiagnosed condition. Mothers, daughters, aunts, sisters. Generation after generation told their bleeding is normal.

As Dr. Nidhi Bhatt at St. Jude Children’s Research Hospital has noted, adolescents often don’t feel comfortable talking about their bleeding — and that silence is one of the major reasons heavy menstrual bleeding goes undetected. St. Jude created the EAGER Clinic (Empowering Adolescents with Gynecology and Hematology Resources and Care) to address exactly this gap — bringing hematology, gynecology, and nursing together for adolescent patients. Their data reflects what we see nationally: only 1 in 3 women with heavy menstrual bleeding ever seek care for it.

What Are the Signs of a Bleeding Disorder in Girls?

Every child is different, and heavy bleeding doesn’t automatically mean a bleeding disorder. But these are signs worth discussing with your pediatrician or a hematologist:

Periods that last longer than 7 days
Soaking through a pad or tampon in an hour or less
Passing blood clots larger than a quarter
Missing school, sports, or social activities because of her period
Chronic fatigue or a diagnosis of iron-deficiency anemia
Easy bruising or frequent nosebleeds
Excessive bleeding after dental work, surgery, or an injury
A family history of heavy bleeding, frequent nosebleeds, or hysterectomies due to heavy periods

That last one matters more than many people realize. Von Willebrand disease is inherited. If the women in your family have always had “bad periods,” it’s possible there’s an undiagnosed bleeding disorder running through the family line.

What Happens When a Bleeding Disorder Goes Undiagnosed?

Delayed diagnosis isn’t just frustrating. It causes real harm.

Women who experienced multiple bleeding events before their diagnosis were nearly three times more likely to undergo a hysterectomy — a surgery that, in many cases, could have been avoided with earlier treatment.

Studies show that 74% of women with von Willebrand disease reported excessive bleeding from multiple sites. Twenty-five percent had undergone a hysterectomy, compared to just 9% in control groups. And rates of depression and anxiety in patients with VWD exceed 60%.

For teenage girls, the impact shows up in missed school days, withdrawal from activities they love, chronic exhaustion, and the quiet erosion of confidence that comes from being told — over and over — that nothing is wrong when they know something is.

How Does Early Diagnosis Change a Girl’s Life?

When we identify a bleeding disorder early, everything shifts.

Surgeries become safer because the care team knows what they’re working with. Injuries are managed appropriately. Heavy periods can be treated — not dismissed. And the young woman at the center of it finally has answers instead of doubt.

Early diagnosis also protects her future. Women with bleeding disorders face increased risks during pregnancy and childbirth. Knowing about a bleeding disorder before that moment — not during a hemorrhage in the delivery room — can be lifesaving.

Where Can Families Get Help for Bleeding Disorders in Nevada?

At Cure 4 The Kids Foundation, our Bleeding and Clotting Disorders Clinic provides comprehensive evaluation and treatment for children and adults with bleeding disorders. Dr. Aimee Foord and our hematology team specialize in the conditions that are most often missed — especially in girls and young women.

If your daughter’s symptoms sound like what we’ve described here, we want you to know: you are not overreacting. You are not imagining it. And you don’t have to figure it out alone.

To schedule an appointment or request a referral, call us at (702) 732-1493.

No child is ever turned away for financial reasons. That is our promise.

This Month and Beyond

Bleeding Disorders Awareness Month is about more than wearing a red tie — though we hope you’ll do that too. It’s about expanding who we think of when we think of bleeding disorders.

It’s the teenage girl who can’t make it through a school day. It’s the mother who assumes her heavy periods are normal because her own mother had the same thing. It’s the young woman heading into surgery without anyone thinking to check her clotting factors first.

They deserve answers. They deserve care. And at C4K, they’ll find both.

If you think your daughter might have a bleeding disorder, don’t wait. Talk to your pediatrician. Ask for a referral. Or call us directly.

Because she’s not clumsy. She’s not dramatic. And she deserves to know what’s really going on.

Frequently Asked Questions

Can heavy periods be a sign of a bleeding disorder?

Yes. As many as 1 in 10 women with heavy menstrual bleeding may have an underlying bleeding disorder such as von Willebrand disease. Signs include periods lasting longer than 7 days, soaking through a pad or tampon in an hour or less, passing large blood clots, chronic fatigue, and iron-deficiency anemia. If your daughter is missing school or activities because of her period, it’s worth discussing with a pediatrician or hematologist.

What is von Willebrand disease?

Von Willebrand disease (VWD) is the most common inherited bleeding disorder, affecting up to 1% of the population. It occurs when the blood lacks sufficient von Willebrand factor, a protein that helps blood clot. VWD affects boys and girls equally, but girls are often diagnosed later — or not at all — because their symptoms, particularly heavy periods, are frequently dismissed as normal.

How is von Willebrand disease diagnosed?

Diagnosis typically involves blood tests that measure von Willebrand factor levels and clotting function. These are specific tests that your pediatrician can order or a hematologist can perform. Standard blood work such as a CBC does not test for von Willebrand disease, which is one reason it’s so often missed. If your daughter has symptoms, ask specifically for von Willebrand disease testing.

Is a bleeding disorder the same as hemophilia?

Hemophilia is one type of bleeding disorder, but it’s not the only one — and it’s not the most common. Von Willebrand disease, rare factor deficiencies, and platelet disorders are all part of the bleeding disorders spectrum. Hemophilia is more commonly associated with boys, which has contributed to the misconception that bleeding disorders don’t affect girls. They do.

Where can I get my daughter evaluated for a bleeding disorder in Las Vegas?

Cure 4 The Kids Foundation’s Bleeding and Clotting Disorders Clinic, led by Dr. Aimee Foord, provides comprehensive evaluation and treatment for children and adults with bleeding disorders in Southern Nevada. No child is ever turned away for financial reasons. Call (702) 732-1493 or visit cure4thekids.org to schedule an appointment or request a referral.

Congratulations Dr. Lasky!

Posted on February 14, 2019June 13, 2024 by admin

We’re proud to announce some news about Dr. Lasky! He has been promoted to director of our Bleeding and Clotting Disorders Clinic.

As one of our board-certified Pediatric Hematology-Oncology physicians, he will continue to see patients affected by childhood cancer. But he will also oversee all aspects of medical treatment for our patients affected by bleeding and clotting disorders, such as hemophilia and von Willebrand disease and other related conditions.

Since 2009, Dr. Lasky has served as principal investigator for a number of clinical trials and has participated in other research activities aimed at improving treatments for those with bleeding and clotting issues.

Congratulations Dr. Lasky!

CSL627_3001 Hemophilia A

Posted on January 18, 2019June 13, 2024 by admin

Bleeding and Clotting Disorders

Protocol:CSL627_3001

Condition or Disease: Hemophilia A

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: A Phase III Open Label, Multicenter, Extension Study to Assess the Safety and Efficacy of Recombinant Coagulation Factor VIII (rVIII SingleChain, CSL627) in Subjects with Severe Hemophilia A

Principal I investigator: Joseph Lasky III, MD

PRO15070638 Von Willebrand disease (vWD)

Posted on January 18, 2019June 13, 2024 by admin

Protocol:PRO15070638

Condition or Disease: Von Willebrand disease (vWD)

Study Type: Interventional (Clinical Trial)

Status: Opening Late 2018

Study Title: The VWD Minimize Menorrhagia (VWDMin) Trial

Principal I investigator: Joseph Lasky III, MD

ALN-AT3SC-003 Hemophilia A or B

Posted on January 18, 2019June 13, 2024 by admin

Bleeding and Clotting Disorders

Protocol:ALN-AT3SC-003

Condition or Disease: Hemophilia A or B

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: ATLAS-INH: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients with Hemophilia A or B, with Inhibitory Antibodies to Factor VIII or IX

Principal I investigator: Joseph Lasky III, MD

More Info: //clinicaltrials.gov/ct2/show/NCT03417102

ALN-AT3SC-004 Hemophilia A or B

Posted on January 18, 2019June 13, 2024 by admin

Bleeding and Clotting Disorders

Protocol:ALN-AT3SC-004

Condition or Disease: Hemophilia A or B

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: ATLAS-A/B: A Phase 3 Study to Evaluate the Efficacy and Safety of Fitusiran in Patients With Hemophilia A or B, Without Inhibitory Antibodies to Factor VIII or IX

Principal I investigator: Joseph Lasky III, MD

More Info: //clinicaltrials.gov/ct2/show/NCT03417245

1160.106 Venous thrombosis

Posted on January 18, 2019June 13, 2024 by admin

Bleeding and Clotting Disorders

Protocol:1160.106

Condition or Disease: Venous thrombosis

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: Open-label, randomized, parallel-group, active-controlled, multi-centre, non-inferiority study of dabigatran etexilate versus standard of care for venous thromboembolism treatment in children from birth to less than 18 years of age: The DIVERSITY study

Principal I investigator: Alan Ikeda, MD

More Info: //clinicaltrials.gov/ct2/show/NCT01895777

1160.108 Venous Thromboembolism, Secondary Prevention

Posted on January 18, 2019June 13, 2024 by admin

Bleeding and Clotting Disorders

Protocol:1160.108

Condition or Disease: Venous Thromboembolism, Secondary Prevention

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: Open label, single arm safety prospective cohort study of dabigatran etexilate for secondary prevention of venous thromboembolism in children from 0 to less than 18 years

Principal I investigator: Alan Ikeda, MD

More Info: //clinicaltrials.gov/ct2/show/NCT02197416

1321.7 Hemorrhage

Posted on January 18, 2019June 13, 2024 by admin

Bleeding and Clotting Disorders

Protocol:1321.7

Condition or Disease: Hemorrhage

Study Type: Interventional (Clinical Trial)

Status: Open for enrollment

Study Title: Single dose, open label, uncontrolled, safety trial of intravenous administration of idarucizumab to paediatric patients enrolled from ongoing phase IIb/III clinical trials with dabigatran etexilate for the treatment and secondary prevention of venous thromboembolism.

Principal I investigator: Alan Ikeda, MD

More Info: //clinicaltrials.gov/ct2/show/NCT02815670