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.
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.
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 PicnicHealth.com to sign up for a free PicnicHealth account and contribute to research if you have hemophilia.
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List the names of all the doctors, hospitals, and other facilities your loved one visits regularly, along with those they have visited in the past. Try to go back as far as you can, striving for at least the last 5-10 years, but do your best. Even if you can’t remember them all, having a strong baseline can help you quickly identify gaps in records.
It is important to make sure that you are fully empowered to make decisions on behalf of your loved one with Alzheimer’s. Your relationship status with the patient may not be enough to legally give you access to your loved one's medical information. It is a good idea to talk to an expert about securing special legal status, such as Power of Attorney (POA), a legal document that allows an individual to name someone as their decision maker should they no longer be able to make decisions on their own.
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.
The better you understand your loved one's medical history, the better you can advocate on their behalf. Access and understanding of this information will help you to ask informed questions with physicians. Through regular communication backed by the data in the medical records, you can help your loved one’s care team develop a more successful care plan.
Learn more about PicnicHealth’s commitment to the Alzheimer’s community and the Alzheimer’s Association
Learn MoreIf your loved one is in the early stages of Alzheimer’s, putting a support system together might not seem like priority #1. But it’s never too soon to build a network of people that you can turn to in times of need. Cultivate connections today with the people who can be there tomorrow, or whenever you might need a hand.
You may also want to connect with other Alzheimer’s caregivers through a support group, whether it meets 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 the world of Alzheimer’s.
If your loved one is in the early stages of Alzheimer’s, putting a support system together might not seem like priority #1. But it’s never too soon to build a network of people that you can turn to in times of need. Cultivate connections today with the people who can be there tomorrow, or whenever you might need a hand.
You may also want to connect with other Alzheimer’s caregivers through a support group, whether it meets 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 the world of Alzheimer’s.
It isn’t always easy to look into the future with Alzheimer’s—but doing the legwork now will save you from stress later. If your loved one is in the early stages of illness, you can involve them in conversations about legal, financial, and long-term care planning decisions. Despite the difficulty of these topics, you’ll all feel empowered by facing them early, and you can move ahead with greater confidence.
It’s an exciting time for Alzheimer’s research, with new treatments in development and coming to market. Ask your loved one’s doctors about therapies they can try or clinical trials they can join. One easy way to participate in research is to sign up at PicnicHealth, which helps to advance Alzheimer’s science by sharing participants’ anonymous health data with some of the brightest minds in research.
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 with Alzheimer’s. And don’t forget to keep a sense of humor along the way.
Easily manage all of your loved one's medical records and contribute to ongoing Alzheimer's research with PicnicHealth.
Learn More(from 13 patients with LC-FAOD)
38% Female
15 providers / patient
7.5 years of data / patient
(from 13 patients with LC-FAOD)
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