The Latest Medical Research to Find Treatments and Cures for Hemophilia

the PicnicHealth Team
May 12, 2020
Blog post originally written by the AllStripes community team. AllStripes was acquired by PicnicHealth in 2023.

Many PicnicHealth users have serious blood disorders like hemophilia that impact nearly all aspects of their lives. While many people with hemophilia have learned to carefully manage their condition, there continues to be a need for research into new therapies and treatments for their symptoms, as well as a hope that someday there may be a cure for hemophilia.

What is Hemophilia?

Hemophilia is a disease characterized by the blood’s inability to clot correctly. People with hemophilia have blood that lacks enough of the necessary blood-clotting proteins or factors that stop them from bleeding in the event of an injury. Internal bleeding is a severe problem for hemophiliacs, especially if the bleeding takes place around the knees, ankles, and elbows. This can lead to severe damage in the internal organs, as well as the delicate tissues of the body, leading to what could be fatal situations.  

Hemophilia cannot be “caught,” and it’s not contagious. It’s a genetic disorder, which means that people who have the genetic disposition to the disease are more likely to develop it. Genetic testing can determine if a person has a higher risk of developing the condition, especially if they have family members who have already been diagnosed with the disease. Throughout the years, the medical world has gotten better in diagnosing the disease, and have taken steps to improve the treatment provided to the people with it.

Advancements in Treatment for Hemophilia

For decades, researchers have worked to develop more novel and effective means to treat hemophiliacs. They have improved ways to mimic or replace the missing clotting proteins. 

Emicizumab is one of the humanized bispecific antibodies that have been developed to treat hemophilia this way. It mimics the activity of the protein FVIII, binding itself to activated coagulation FIX and factor X. It was approved by the FDA as hemophilia treatment in 2017 to treat patients with Hemophilia A. About 25-40% of patients with this type of hemophilia can no longer receive FVIII as the therapy itself, and emicizumab enables them to have a lifesaving treatment that they need. According to doctors, the drug makes a patient previously suffering from severe hemophilia seem more as though they had a milder form of it.

Turoctocog alfa pegol is another recently approved drug. Also known as N8-GP or Esperoct, it extends the lifespan of FVIII during prophylaxis. This leads to a reduction of bleeding episodes and on-demand treatment. After evaluating Hemophilia A patients who have taken the procedure, the drug proved that it is an effective, fixed-dosed regimen for adults and adolescents with hemophilia, resulting in improved quality of life.

Another new development is a promising form of gene therapy. Gene therapy has been one of the conventional means of treating hemophilia, as it introduces genetic material to cells to make up for an abnormal gene or a missing much-needed protein in the body. The new gene therapy uses genetically reprogrammed cells derived from hemophilia A patients that have been grafted onto hemophiliac mice in preliminary studies. The reprogrammed cells restored proper blood levels of FVIII and improved clotting. Should the treatment prove similarly beneficial to humans, it won’t just be an effective hemophilia treatment, but also be useful as a long-term blood protein replacement.

This work by researchers has led to more significant strides in treating hemophilia. Hopefully treatments like these can help people with this disease can experience better clotting, improved quality of life, and a better outlook on their future with fewer risks.

PicnicHealth also supports research on hemophilia by structuring real-world evidence datasets from anonymized medical records of real people living with hemophilia in the real world. Visit to sign up for a free PicnicHealth account and contribute to research if you have hemophilia.


the PicnicHealth Team

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It’s important to have all of your loved one’s medical records together in one spot. This makes it much easier for you and your loved one’s physicians to accurately map the patient’s medical journey and more easily share information between doctors. Fortunately, tools exist to make record management and access simple. A free resource like PicnicHealth helps you collect and organize all of this information. PicnicHealth’s intuitive timeline allows you to pinpoint data across the medical history, eliminating your need for keeping heavy binders filled with paper records or keeping track of multiple software portal logins.

Review the Medical Records to be an Informed Advocate

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When you’re juggling appointment times and insurance claims, putting a robust support system together might not strike you as the most urgent task. Investing the time to cultivate relationships with people can turn to in times of need will pay dividends. The next time you need a last-minute ride or just someone to listen, you won’t be on your own.

There are many condition-specific support groups and support groups for caregivers generally in person or online. In addition to the encouragement and empathy they provide, support groups can be a helpful source of tips, resources, and recommendations for navigating caregiving.


Stay organized.

The backbone of effective caregiving is organization. Keep medical information, appointment schedules, and medication lists in order. Use a planner or a digital service like PicnicHealth to stay on top of your responsibilities. This attention to detail can prevent future complications and reduce day-to-day stress.


Explore treatments and clinical trials.

We’ve seen incredible breakthroughs in treatment over the past couple of years, powered by patients and their caregivers participating in research. Stay in the loop about the latest in medical advancements and available resources that could benefit your loved one. Whether it’s a new therapy option or a community service that aids independence, being informed can make a world of difference in the quality of care you provide.


Make time for self-care.

It may seem self-centered to focus on self-care—but when you feel good, you can be a better caregiver. Whether it’s exercise, a mindfulness practice, a soak in the bath, or just time to rest when you need it, carve out those moments in the day when you can unwind, reset, and stay healthy mentally and physically. Think of it as building up your reserves of kindness, patience, and understanding—which can only benefit your loved one. No one can pour from an empty cup.

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LC-FAOD Odyssey: A Preliminary Analysis, presented at INFORM 2021

Data from real-world medical records:

(from 13 patients with LC-FAOD)

16 yrs old

Median age at enrollment

38% Female

15 providers / patient

7.5 years of data / patient

Data from patient-reported outcome (PRO) survey

(from 13 patients with LC-FAOD)

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