# The Latest Medical Research to Find Treatments and Cures for Hemophilia By the PicnicHealth Team May 12, 2020 • 4 min read 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](https://www.mayoclinic.org/diseases-conditions/hemophilia/symptoms-causes/syc-20373327) 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](https://www.ashclinicalnews.org/spotlight/feature-articles/breakthroughs-bleeding-updates-treating-managing-hemophilia/) 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](https://www.pharmacytimes.com/news/new-treatment-for-hemophilia-a-receives-fda-approval), 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](https://hemophilianewstoday.com/2020/03/16/new-gene-therapy-hemophilia-a-shows-promise-mouse-model-study/) 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_](/content/hemophilia/index.html) _to sign up for a free PicnicHealth account and contribute to research if you have hemophilia._ ## We know that every person's story is unique and deserves to be heard. ### Create a List 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. ### Ensure You Have the Appropriate Legal Status 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. ### Gather and Organize the Medical Records in One Place 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 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 ## Together, we can make a difference. ### 1\. Build a support network. 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. ### 2\. 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. ### 3\. 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. ### 4\. 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. ## Having trouble managing your loved one's medical records? Easily manage all of your loved one's medical records and contribute to ongoing Alzheimer's research with PicnicHealth. ### 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) ## Learn more about contributing to IgAN research with PicnicHealth. ## What to Expect Before your video call: ### Schedule Your Visit Visit your to-do list on your PicnicHealth Research Dashboard or click the scheduling link sent to your email. _Note:_ _Search for “New task for the ORBIT-CIDP Study" to find the video call scheduling link._ ### Check Your Confirmation Check your email for a confirmation with your scheduled video call time and instructions. On the day of your video call: ### Join the Call Click on Video Link to join your personal video call using the link sent by email, or text message, or find it on your [research dashboard](https://app.picnichealth.com/research). ### Meet Your Nurse A Registered Nurse (RN) will guide your virtual assessment, which will last about 30 minutes. ### Complete the Physical Activity Assessment (INCAT) The nurse will guide you through questions and, if needed, physical tasks to help researchers gain a deeper understanding of CIDP. ### Complete the Movement Assessment (Optional) If you participate, a nurse will guide you through three short recorded movement activities to complete as best you can: 1. **Chair Task**: While seated with your arms crossed over your chest and hands on opposite shoulders, you’ll be asked to stand up, remain standing for 20 seconds, and then sit back down. 2. **Arm Movement Task**: While seated with your arms resting at your sides, you’ll be asked to raise both arms out to the sides until they meet above your head, then lower them back to your lap. 3. **Finger Dexterity Task**: While seated, raise your right hand with fingers extended. Touch your thumb to each fingertip in order, then reverse. Repeat with your left hand. ### Earn Compensation Receive up to $55 for your participation: - $25 for completing the Physical Activity Assessment (INCAT). - $30 for the Optional Movement Assessment. **Recording:** Your research assessment may be recorded to ensure accurate data collection. If you participate in the optional Movement Assessment, it will also be recorded. These recordings may capture your voice and responses, but identifiable information like your face, name, or background will be removed to protect your privacy. ### Opt Into the Smart Insole Study Activity Complete the opt-in survey to confirm your participation. ### Receive Your Smart Insoles Your smart insoles will be shipped to your home via FedEx and should arrive within 1 week. ### Create Your Account You’ll receive an email from Celestra Health with your account details. Follow those steps to set up your account. If you don’t see an email from Celestra Health in your inbox, **_please check your spam or junk folder._** ### Download the App After creating your account, you’ll be directed to a landing page with links to the App Store or Google Play. Use the link to download the correct version of the app for your device. ### Log In Open the app and log in using the email address and password you used when creating your account. ### Enable Permissions - For iOS users: Enable Motion & Fitness and allow access to Apple Health. - For Android users: Enable Activity Recognition permissions. ### Connect Your Insoles Turn on Bluetooth, and follow the app's instructions to connect your smart insoles. ### Enable Notifications Enable push notifications to stay updated on reminders and activity progress. ### Start Walking Sessions When you’re ready to perform a walking session, tap ‘Start’ on the Ad Hoc Walking task card in the app. ### Need Help? Should you need to contact Celestra Health support for any reason, you can submit a ticket through the Help section of the app by tapping the Submit A Ticket card and filling out the form. A Celestra Health representative will typically respond within one business day.