News

Animal Justice on Dog Research after Ontario Ban

Gene Therapy, Industry News & Research

The government of Ontario, Canada, recently passed a ban on invasive medical research involving dogs and cats. This appears to directly affect…

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A $39 Million Grant for Hemophilia A Gene Therapy

Industry News & Research

Children’s Hospital of Philadelphia received an award of up to $38.9 million from ARPA-H for a five-year program developing personalized gene-editing therapies…

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Stimate Returns

Health and Well Being, Industry News & Research

On July 29 Ferring announced that Stimate nasal spray will return after a six-year absence following the 2020 recall. Patient availability is…

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Altuviiio Update

Health and Well Being, Industry News & Research

Altuviiio (efanesoctocog alfa) continues to impress in difficult-to-treat patients. A French real-world study reported that once-weekly Altuviiio reduced treated bleeds by about…

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Hemlibra Success

Health and Well Being, Industry News & Research

A published study in Blood Advances reports that over 60% of hemophilia A patients on Hemlibra (emicizumab) experienced no bleeding episodes. Long-term treatment with…

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A Guide to Medicaid Changes

Advocacy & Legislation, Industry News & Research

Changes to Medicaid could affect access to coverage, treatment, infusion services, and specialty care for some people with hemophilia. The Coalition for…

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Von Willebrand Disease Research Gets Active

Industry News & Research, Living with a Bleeding Disorder, Women with Bleeding Disorders

The first serious efforts to develop long-acting subcutaneous preventive treatments for VWD are taking place. VGA039 is a subcutaneous antibody designed to…

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First Mimetic on Track

Industry News & Research

Novo Nordisk’s “Mim8” (denecimig) is an investigational bispecific antibody that mimics activated factor VIII. According to findings from the multinational randomized, Phase…

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Learn More About Fibrinogen Deficiency

Industry News & Research

Fibrinogen deficiency is an ultra-rare bleeding disorder, and now NBDF has a publication to help people with this condition gain a deeper…

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HYMPAVZI® Receives Expanded Indication from FDA

Industry News & Research

The new indication for HYMPAVZI® (marstacimab-hncq) broadens access to this once-weekly non-factor prophylactic therapy that targets TFPI to improve clotting and reduce bleeding…

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CMS Rule Threatens Medicaid Protections for Bleeding Disorders

Advocacy & Legislation, Industry News & Research, Living with a Bleeding Disorder

The new rule states that having a “serious or complex medical condition,” such as bleeding disorder, will no longer automatically exempt a…

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First-Ever Recommendations Call for Routine Iron Screening in People with Bleeding Disorders

Health and Well Being, Industry News & Research, Living with a Bleeding Disorder

A new MASAC recommendation states that all people with an inheritable bleeding disorder, regardless of sex, age, or bleeding disorder severity, be…

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Next-generation Factor VIII Variants to Improve Gene Therapy for Hemophilia A

Gene Therapy, Industry News & Research

In this webinar, Ben Samelson-Jones, M.D., Ph.D., a pediatric hematologist at Children’s Hospital of Philadelphia, shared research his team is doing to…

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Discontinuation Timeline for HEMOFIL ®M and RECOMBINATE

Industry News & Research

Takeda has updated the information for how long the these treatments will be available for patients who are still using these therapies….

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NBDF-Takeda Clinical Fellows

Industry News & Research

As fewer clinicians enter the field of benign hematology, learn how the NBDF-Takeda Clinical Fellowships are working to strengthen the pipeline of…

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Crack the Case: A Hemophilia A Mystery Awaits

GLHF News, Industry News & Research, Living with a Bleeding Disorder

Hemophilia A patients with life cases. Together, we will try to explore a compelling question: can two individuals with very different experiences…

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HEMGENIX® On Hold

Industry News & Research

CSL Behring has notified the hemophilia B community of a temporary global stockout of Hemgenix. This situation is not related to the…

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New Treatment That Can Improve Hemophilia Care

Industry News & Research, Living with a Bleeding Disorder

As hemophilia treatments continue to roll out that are different than traditional factor replacement, they can offer more promising treatment but can…

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When Unexpected Bleeding Appears Later in Life

Industry News & Research, Living with a Bleeding Disorder

Acquired hemophilia usually affects people in their 70s and 80s without warning. Here’s what you need to know. National Bleeding Disorders Foundation,…

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Learn About the New Medications That Could Change Hemophilia Care

Industry News & Research, Living with a Bleeding Disorder

Anti-TFPI treatments can help people with hemophilia A or B — with or without inhibitors — to better control bleeding. Source: National…

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Von Willebrand Disease Treatment: What’s New and Next?

Industry News & Research, Living with a Bleeding Disorder

Advances include improved access to prophylaxis, multiple subcutaneous drugs in development, and research looking at better ways to personalize treatment. Source: National…

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Temporary Global Stockout of Hemgenix: A Message to the Hemophilia B Community from CSL Behring

Health and Well Being, Industry News & Research

Dear Hemophilia B Community, We are writing to share an important update on the availability of HEMGENIX® (etranacogene dezaparvovec-drlb). CSL is currently experiencing…

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Government Relations Update

Advocacy & Legislation, Industry News & Research, Living with a Bleeding Disorder

Good news for the bleeding disorders community in the House of Representative’s FY26 Labor, Health and Human Services appropriations bill, which included…

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NBDF’s Commitment to Shared Decision-Making and Evidence-Based Access in Gene Therapy

Gene Therapy, Industry News & Research

The current coverage environment can create challenges for individuals seeking to explore gene therapy as a potential treatment option, as well as…

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