News & Perspectives

2022 Summer Internships and Young Adult Advocacy Summit on Location in Washington, DC

January 21, 2022

Summer 2022 is gearing up to be an exciting time here at HFA for young adults and college students in our bleeding disorders community. We will be kicking off the summer in Washington, DC with a 10-week paid internship for two eligible college students and ending the summer with an exciting Young Adult Advocacy Summit […]

HFA Hosts Mental Health First Aid Training

January 20, 2022

Hemophilia Federation of America (HFA) is pleased to offer free聽Mental Health First Aid聽training through an internationally renowned program by the聽National Council for Behavioral Health聽as part of HFA鈥檚 Mental Health and Wellness Initiative. Participants who complete both sections of the course will receive a Mental Health First Aid certificate indicating they have been trained in recognizing […]

Barriers to Diagnosis: Women and X-linked Diseases

January 18, 2022

On Tuesday, February 8 from 7:00 – 8:30 pm ET, HFA will present 鈥淏arriers to Diagnosis: Women with X-linked Diseases鈥 in partnership with Remember the Girls and the National Organization for Rare Diseases (NORD). Register Now A ubiquitous belief among medical professionals is that all X-linked diseases are recessive, meaning a person would need two […]

State of the States: Fall 2021

December 29, 2021

Prohibiting copay accumulators remains a state priority for 2022 Legislation to prohibit health plans from depriving consumers of any benefit from third-party assistance with cost-sharing obligations has already been pre-filed or drafted in at least a dozen state legislatures for the coming session (as well as the District of Columbia), with pending bills in four […]

Washington Wire: December 2021

December 29, 2021

As we close out 2021, we want to reflect on HFA鈥檚 policy agenda: how our policy priorities shaped our advocacy efforts over the past year and what we expect and plan for 2022. HFA鈥檚 policy agenda always centers on access 鈥 access to treatments and care, access to quality health coverage, access to relief from […]

HFA Launches 2022 Educational Series with Presentation on Joint Replacements for Patients with Bleeding Disorders

December 20, 2021

WASHINGTON, DC, December 20, 2021 —聽The Hemophilia Federation of America (HFA) offers monthly educational webinars for patients, caregivers and healthcare providers and is launching their 2022 series on January 18 at 7:00 pm ET with Dr. James Luck, Jr.,聽Director of Surgical Services at the Orthopaedic Institute for Children at UCLA Health. Dr. Luck will discuss聽joint […]

Freeline Presents Long-Term Follow-Up Data from Phase 1/2 B-AMAZE Trial in Hemophilia B at the 2021 ASH Annual Meeting and Announces Early Initiation of Phase 1/2 B-LIEVE Dose-Confirmation Trial

December 20, 2021

B-AMAZE long-term follow up data demonstrate sustained FIX expression up to 3.5 years; potential for expression levels in the normal range with relatively low vector doses B-LIEVE trial to confirm dose and immune management for planned Phase聽3 pivotal trial launched one quarter ahead of latest guidance LONDON, Dec. 13, 2021 (GLOBE NEWSWIRE) — Freeline Therapeutics […]

Pfizer and Sangamo Announce Updated Phase 1/2 Results Showing Sustained Bleeding Control in Highest Dose Cohort Through Two Years Following Hemophilia A Gene Therapy

December 20, 2021

NEW YORK & BRISBANE, Calif.–(BUSINESS WIRE)–Pfizer Inc.聽and Sangamo Therapeutics, Inc. (Nasdaq: SGMO), a genomic medicines company, today announced updated follow-up data from the Phase 1/2 Alta study of giroctocogene fitelparvovec, an investigational gene therapy for patients with moderately severe to severe hemophilia A. The Alta study data, in patients with severe hemophilia A, are being […]

Sigilon Therapeutics Announces Strategic Reprioritization

December 14, 2021

CAMBRIDGE, Mass., Dec. 13, 2021 (GLOBE NEWSWIRE) — Sigilon Therapeutics, Inc. (NASDAQ: SGTX), a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living TherapeuticsTM platform, today announced a strategic reprioritization to enable the Company to focus on MPS-1 and diabetes. 鈥淭here have been key learnings in our Phase 1/2 […]

uniQure and CSL Behring Announce Primary Endpoint Achieved in HOPE-B Pivotal Trial of Etranacogene Dezaparvovec Gene Therapy in Patients with Hemophilia B

December 13, 2021

Press release source: uniQure and CSL Behring via Globe Newswire Largest gene therapy study in hemophilia B achieved primary endpoint of non-inferiority in annualized bleeding rate after stable Factor IX (FIX) expression, assessed at 18 months following a single dose of etranacogene dezaparvovec Etranacogene dezaparvovec also achieved secondary endpoint demonstrating statistical superiority in reduction of […]

Connect with Your Community

December 8, 2021

Blood Brotherhood Our Blood Brotherhood program is designed to empower adult men living with bleeding disorders. This nationwide network of men educates, supports and challenges each person as they strive to live healthier lives. Join us as we discuss issues facing adult men in our community. Join the Blood Brotherhood Chat Sister Space Join your […]

Over the Rainbow

December 7, 2021

鈥3, 2, 1,鈥 counting down, I held my breath and flipped the test over. There they were 鈥 two pink lines staring up at me. I paused, waiting for the influx of emotion to wash over me. It never came. As much as I had been hoping and praying for this, I couldn鈥檛 seem to […]

Dear Addy: Year-Round Advocacy

December 5, 2021

Dear Addy, I鈥檓 interested in getting involved in policy and advocacy work, but am I too late to get involved? When鈥檚 the best time of year to advocate? Signed, Too Late to Advocate? Dear Too Late to Advocate, No need to worry – you鈥檙e in luck! You鈥檙e not too late at all. Advocacy is Year-Round! […]

Dear Addy: SNF Access

December 5, 2021

Dear Addy, I am going to have a knee replacement next month and hope that I can get post-operative care in a skilled nursing facility. I’ve heard that Medicare beneficiaries like me haven鈥檛 been able to get admitted into skilled nursing facilities. Am I missing something? Signed, It鈥檚 Not a Limp, It鈥檚 A Swagger Dear […]

Dear Addy: H.R. 5801

December 4, 2021

Dear Addy, I鈥檝e been hearing a lot about co-pay accumulator adjuster programs (CAAPs) lately. What are CAAPs, how do I know if my insurance plan has one, and what can I do about them? Signed, Co-pay Confused Dear Co-Pay Confused, Those are great questions. Co-pay accumulator adjuster programs (CAAPs) are becoming increasingly common in the […]

From the CDC: New Hemophilia Key Findings Now Available

December 2, 2021

On December 1, the CDC recently released a new Key Findings summary of a recent scientific publication of women and girls who have hemophilia. The CDC reports, 鈥淗emophilia primarily affects men, but聽women can have hemophilia, too. It was once thought that only men could have hemophilia and women could only聽pass on the gene聽that causes hemophilia […]

Washington Wire: November 2021

November 30, 2021

For the second year in a row, HFA鈥檚 Patient Fly-In took place as a virtual event. Two dozen community members together with HFA staff made forty (!) Zoom visits with Congressional offices during the week of November 15th. The timing of the Fly-In 鈥 always a bit of a gamble 鈥 ended up being very […]

Sigilon Therapeutics Announces Update on SIG-001 Phase 1/2 Study in Hemophilia

November 29, 2021

Source:聽Sigilon press release Sigilon Therapeutics, Inc., a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics™ platform, reported that fibrosed spheres were observed during a retrieval procedure in a patient in its Phase 1/2 study of SIG-001 in severe or moderately severe hemophilia A. The SIG-001 trial had […]

Dear Addy: Open Enrollment Options for 2022

November 22, 2021

Dear Addy, I know Open Enrollment for 2022 health insurance began on November 1, 2021. As a person with a bleeding disorder, what do I need to know, and how should I go about choosing my options? Signed, Anxious About Insurance Dear Anxious, You are right that Open Enrollment just began. Open Enrollment is the […]

HFA Recognizes 2021 Congressional Champions & Hosts Virtual Fly-In

November 17, 2021

Due to ongoing concerns regarding public gatherings and the impact of COVID-19 on in-person legislative visits, HFA is again holding its annual Patient Fly-In and Congressional Reception experience as an online campaign. The event is taking place from November 15-19, 2021. On November 16th, grassroots bleeding disorders advocates are participating in Congressional visits by video […]

Pfizer Giroctocogene Fitelparvovec (Hemophilia A Gene Therapy) AFFINE Phase 3 Study Update

November 5, 2021

The following is a news release from Pfizer. Read the full statement here (view PDF): We would like to share a recent update to our gene therapy clinical program for Hemophilia A (C3731003; AFFINE study) evaluating giroctocogene fitelparvovec, that we are developing with Sangamo. Following the observation of factor VIII (FVIII) levels greater than 150% […]

Washington Wire: October 2021

October 29, 2021

Policy and advocacy sessions were among the many programs on tap at HFA鈥檚 recently concluded 2021 Symposium. Thank you to all who attended! It was wonderful to 鈥渟ee鈥 you at our virtual event 鈥 even as we plan, and fervently hope, to see you in person at HFA鈥檚 2022 Symposium in San Antonio. For those […]

Dear Addy: Medicare Open Enrollment for 2022

October 29, 2021

Dear Addy, I am a Medicare beneficiary weighing my plan options for 2022. What do I need to know about choosing a plan for the coming year? Medicare Beneficiary Dear Beneficiary, Every year Medicare open enrollment runs from Oct. 15 through Dec. 7. This is the time of year when you can decide to stay […]

State of the States: Summer 2021

October 28, 2021

In this quarter鈥檚 news: advocates notch another win, securing state level protection against copay accumulator adjusters; Missourians begin enrolling in the state鈥檚 expanded Medicaid program; and ACA marketplaces report 2021 enrollment totals, rates for 2022, and other developments. North Carolina becomes 12th state to ban copay accumulator adjusters Governor Roy Cooper signed S.B. 257 into […]

Recall: Monoject Flush Prefilled Saline Syringes by Aligned Medical Solutions Recalled Due to Potential for Plunger to Draw Back After Air Has Been Expelled and Reintroduce Air Back into Syringe

October 28, 2021

Monoject Flush Prefilled Saline Syringes by Aligned Medical Solutions: Recall – Due to Potential for Plunger to Draw Back After Air Has Been Expelled and Reintroduce Air Back into Syringe Read the full press release from the FDA Issue: Aligned Medical Solutions is recalling Cardinal Health鈥檚 Monoject Flush Prefilled Saline Syringes placed into 9,378 kits. […]

Females in Research Sharing and Translation (FIRST) Project

October 11, 2021

Inherited bleeding disorders, such as hemophilia and von Willebrand disease, have long been underrecognized as causes for bleeding in females. Part of the problem with this under recognition has been the lack of female engagement in research related these bleeding disorders.     Through The Patient-Centered Outcomes Research Institute (PCORI) funded engagement project, the PRIDE […]

Dear Addy: Policy Newbie

September 30, 2021

Dear Addy, I want to become a better bleeding disorder advocate, but with so much going on in healthcare, I鈥檓 not sure which areas I should focus my on first. Does HFA have any suggestions on how to narrow my focus? Policy Newbie    

Washington Wire: September 2021

September 29, 2021

As September ends, HFA continues to closely monitor action on the 鈥淏uild Back Better鈥 Act. Congressional leaders are trying to pass this legislation, which embodies President Biden鈥檚 social infrastructure agenda, via the budget reconciliation process (i.e., a simple majority vote, almost certainly along party lines). The bill, which includes numerous health care proposals, comes with […]

Getting WIRED with Research During a Pandemic

September 28, 2021

By Whitney Armijo, Research聽Project聽Coordinator In the past two years, two Hemophilia Federation of America (HFA) projects have aimed to empower women in the bleeding disorders community to take part in research and advocate for their own health.聽 In 2019, the HFA research team created Females In Research Sharing and Translation (FIRST) to gather more details […]

Film School: Sushant Sharma Bhattarai aims to educate people about hemophilia through documentaries

September 28, 2021

By Melanie Padgett Powers, MANAGING EDITOR As a kid growing up in Nepal with hemophilia A, Sushant Sharma Bhattarai, now 22, wasn鈥檛 allowed to run around, ride bikes or play sports. Factor replacement therapy wasn鈥檛 available in his country, and the risk was just too great.聽 Later, living in California as a teenager, he still […]

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