Uniting for accessible care at the Rare Blood Disease Summit

A columnist shares her takeaways from Genentech's recent event

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by Jennifer Lynne |

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Last week, I was privileged to attend Genentech‘s Rare Blood Disease Summit at its campus in San Francisco. I’ve been proud to serve on this biotechnology company’s Women in Hemophilia advisory board for the past two years.

The invitation-only summit was a small but powerful gathering, bringing together the leaders of nonprofit organizations and the Women in Hemophilia board members for discussion and collaboration. An overarching topic was the imperative initiative to improve access to care for underserved communities. They include women and those facing language and financial barriers that hinder their ability to access the critical care they need.

Among the nonprofit organizations present were Hope for Hemophilia, the Hemophilia Federation of America (HFA), and the National Bleeding Disorders Foundation (formerly known as the National Hemophilia Foundation).

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Shellye Horowitz, a former columnist for Hemophilia News Today and a summit presenter, has taken on the role of director of education at HFA, where she’s emerged as a passionate advocate for women and others living with mild hemophilia. Her contributions to the field are genuinely inspiring.

Shellye delivered an enlightening presentation addressing the special challenges women and those with mild hemophilia face. Her compelling talk made a significant impact on me. I’m excited to delve into its insights in an upcoming column.

Most people in the room had a deeply personal connection to hemophilia or another rare blood disease. Many women shared stories of their sons affected by hemophilia and recounted their own experiences with undiagnosed or untreated bleeding symptoms. Several told me they’ve given up on receiving a diagnosis, much less treatment. These stories always leave me frustrated and sad.

Hemlibra and women

Amid the challenges, however, I was heartened to learn about the groundbreaking clinical trials being led by Genentech. Hemlibra (emicizumab-kxwh), a medication that has significantly improved the lives of many grappling with hemophilia A, offers hope. Instead of traditional IV infusions, Hemlibra is administered as a more convenient subcutaneous injection. Importantly, clinical trials for Hemlibra now welcome women with hemophilia A and severe von Willebrand disease.

Another memorable moment was the opportunity to meet the incredibly inspiring Brandi Lewis. Brandi is a dedicated columnist for PNH News, another website published by BioNews. She and her mother courageously opened up about Brandi’s battles with two rare blood disorders: PNH, or paroxysmal nocturnal hemoglobinuria, and aplastic anemia. Brandi’s commitment to raising awareness and her ability to share her story so eloquently left a lasting impression on me and many others at the event.

The summit underscored the vital need for collaboration and innovation in the pursuit of accessible, effective treatments for everyone affected by rare blood disorders. Together, we can strive for a future where no one’s suffering goes unnoticed or untreated.

Note: Hemophilia News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or another qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Hemophilia News Today or its parent company, BioNews, and are intended to spark discussion about issues pertaining to hemophilia.


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