July 23rd 2025
BioMarin's Roctavian gene therapy shows long-term effectiveness in treating severe hemophilia A, reducing bleeding and improving quality of life for patients.
Careful Consideration Needed for Management of Acquired (Autoimmune) Hemophilia
May 22nd 2025Patients diagnosed with acquired (autoimmune) hemophilia, a serious and rare bleeding disorder, have high rates of hospital readmissions because of infections and bleeding, as well as high death rates especially among older patients.
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Hemgenix Gene Therapy Shows Long-Term Efficacy, Safety in Hemophilia B Patients
December 21st 2023Men with severe or moderately severe hemophilia B continue to display low bleeding rates and very little use of replacement therapies for at least three years following a single dose of the approved gene therapy hemgenix.
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FDA Approves Adzynma for Rare Blood Clotting Disorder cTTP
November 27th 2023CTTP is rare hereditary blood clotting disorder stemming from a disease-causing mutation in the ADAMTS13 gene. This gene plays a crucial role in producing the ADAMTS13 enzyme, responsible for regulating blood clotting.
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Diversity Gaps in U.S. Rare Disease Trials and the Inequalities in Hemophilia Clinical Studies
November 17th 2023Harsha Rajasimha, MD, founder and executive chairman of IndoUSrare, spoke to MHE about the current disparities in diversity and ethnicity in rare disease clinical trials in the U.S., as well as what he's seeing in clinical trials for diseases like hemophilia, in specific.
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Gene Therapy for Hemophilia As a ‘Paradigm Shift’
September 28th 2023Roctavian for hemophilia A and Hemgenix for hemophilia B could spare some patients from treatment that involves intravenous infusions of clotting factors several times a week. But a hemophilia expert cautions that the high-priced gene therapies aren’t a cure and that the reprieve won’t last a lifetime.
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Safeguarding Hemostasis and Adherence in Hemophilia A: Specialty Pharmacy Strategies
August 30th 2023Specialty pharmacies can help patients with hemophilia A maintain hemostasis and ensure patient safety through medication therapy management, utilizing regional care coordinators and direct pharmacist outreach, and by maintaining consistent provider communication.
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Gene Therapy Performance Guarantees Will “Improve Access”
August 17th 2023Joe Pugliese, president and CEO of the patient advocacy group Hemophilia Alliance, discusses recent announcements by manufacturers of two ultra-expensive gene therapies for hemophilia that they will offer payers partial or full reimbursement if patients fail on their therapies.
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Counseling Patients With Hemophilia A: Medication Storage and Administration Techniques
August 16th 2023Counseling for patients with hemophilia A should include medication storage requirements, the importance of maintaining sterile injections, and administration considerations, such as how to use blunt fill needles.
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Balancing Aspects That Influence Hemophilia A Treatment Decisions
August 16th 2023Diverse factors can influence treatment decisions for patients with hemophilia A, such as the evolving landscape of insurance coverage, challenges related to vein access and medication storage, and mode of administration.
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