Anonymous published Pursuit of a Cure - Spread the Word in Pursuit of a Cure 2020-08-25 12:21:44 -0400
Spread the word.
None of the incredible progress we’ve made in FOP research — identification of the FOP gene and the development of potential treatments — would be possible without the dedication of our community. And while we’ve come so far, we still have far to go.
We can search for treatments and a cure together. The IFOPA invites you to join the In Pursuit of a Cure Campaign to expand FOP research — and we hope you will invite your friends and family to join as well!
Our FOP families and communities are the best fundraisers! As we work toward our $250,000 goal, please help kick off the In Pursuit of a Cure Campaign.Want to learn more about how you can help? Join IFOPA staff for a Virtual Meetup on August 5 at either 7 am ET/11 am GMT or 4 pm ET/8 pm GMT. You can see what a personal fundraising page looks like and find out about resources available to help you invite your friends and family.Virtual Meetup MorningVirtual Meetup Afternoon
Set Up a Personal Fundraising Page
- We’ll provide you a page where you can share your FOP story, photos and why you want to see FOP research grow.
- Or we can provide a generic FOP community template for you.
- You’ll set a goal and have a unique URL to send out to your friends and family.
- You’ll get updates on who has donated to your campaign.
Invite Your Friends and Family to Our Virtual Event
We can provide you postcards or an electronic flyer to invite your friends and family to the August 26 / 27 event — In Pursuit of a Cure - An Evening of Inspiration and Possibility with the IFOPA. Email Cathryn Roys, Community Fundraising Manager, to get your postcards and/or electronic flyer.
Anonymous published Pursuit of a Cure - Get the Facts in Pursuit of a Cure 2020-08-25 12:21:30 -0400
GET THE FACTS.
Learn more about what gene therapy is and its potential in the search for a cure for FOP.
Gene therapy is an approach to treating diseases that seeks to modify or introduce genes into a patient's body. Genes are the “blueprint” for how your body functions. People with FOP have a difference in their genes that causes excess bone to grow. Gene therapy may be a way to correct that difference.
The IFOPA’s first-ever gene therapy research grant has been awarded to Jae-Hyuck Shim, PhD, Associate Professor, Department of Medicine/Division of Rheumatology Immunology and Microbiology Program at the University of Massachusetts Medical School and to Guangping Gao, PhD, Professor of Microbiology and Physiological Systems at the University of Massachusetts Medical School. Dr. Gao is also the Past President of the American Society of Gene & Cell Therapy and Director of the Horae Gene Therapy Center at the University of Massachusetts Medical School. A consortium of gene therapy experts and FOP specialists will collaborate to help guide this FOP gene therapy research.There are 2 easy ways for you to learn more about gene therapy:
Watch this video from the American Society of Gene & Cell Therapy to learn the basics of gene therapy.
- Watch this video from the American Society of Gene & Cell Therapy to learn the basics of gene therapy.
The In Pursuit of a Cure Campaign will culminate in a live international event that unites the entire FOP community on the path to treatments and a cure.CO-CHAIRSRory & Eric OttoRebecca Wallace & Kyle Wallace
Join the IFOPA and the international FOP community for an exciting evening in support of expanding FOP research, including the first-ever gene therapy grant. It’s a virtual event, so you and your family and friends can take part from the comfort of your homes!
The event will be live broadcast on Facebook and YouTube.
Select the viewing time that works best for you:
August 26 at 7 pm ET/11 pm GMT* (US, Canada, Central America & South America)
August 26 at 9 pm ET or August 27 at 1 am GMT* (US, Canada, Central America & South America)
August 27 at 5 am ET/9 am GMT* (Australia & New Zealand)
August 27 at 7 am ET/11 am GMT* (Hong Kong & Asia)
August 27 at 2 pm ET/6 pm GMT* (Europe, Russia, the Middle East and South Africa)We will leave no stone unturned in the search for treatments and a cure for FOP. The In Pursuit of a Cure Campaign will fund new ACT for FOP competitive research grants and the IFOPA’s first-ever targeted research grant in gene therapy.We hope to raise $250,000 toward new paths to treatments and a cure.
The first $50,000 in donations will be matched!* Eastern Time and Greenwich Mean Time (please convert to your local time zone)
FOP patients harbor mutations in the ALK2 protein (also known as ACVR1) that drives excessive bone morphogenetic protein (BMP) signaling, which regulates cartilage and bone development. INCB000928 is an oral investigational drug designed to target and block this disease-causing mutant FOP protein hyperactivity. In preclinical studies, INCB000928 demonstrated potency for the target kinase, selectivity, safety and strong suppression of heterotopic ossification (HO) in animal models.
About the Clinical Trial
Incyte is currently conducting aPhase 1
trial with INCB000928 in healthy volunteers (e.g. people without FOP) to establish the safety of the investigational drug. ThePhase 1
trial is evaluating single and multiple ascending doses of oral INCB000928 in healthy volunteers.
- AGE: ≥ 18
- DISEASE ACTIVITY: Healthy volunteers only
- MUTATIONS: n/a
- STUDY TYPE: Interventional
- RANDOMIZED STUDY: No
- PLACEBO CONTROLLED: No
- LENGTH OF PARTICIPATION: n/a
- NUMBER OF STUDY VISITS: n/a
Phase 1, Active
Your Contribution Makes a Difference
The FOP Registry is the largest and most detailed collection of clinical and medical information about FOP and those who are living with the disease—and it’s built by people like you.
The FOP Registry collects information on how FOP affects people for an important purpose: to help clinicians and researchers better understand FOP in order to improve clinical care and speed the development and availability of disease-changing treatments, and one day, a cure.
It Takes All of Us
The best part about this database is that it's growing every day. That's because people like you from around the world, and their FOP physicians, are contributing to it.
We are grateful for the support of our sponsors: Blueprint Medicines, Ipsen and Regeneron Pharmaceuticals. Thank you to the Radiant Hope Foundation for the visionary funding that launched the FOP Registry.
Our Advisory BoardsWhat We've Learned from People in the FOP Registry
Here's just a small sample of the information uncovered that was previously not well-understood by doctors:45% of patients
in the FOP Registry report symptoms affecting their ears34% of patients
in the FOP Registry report skin problems28% of patients in
the FOP Registry report problems with sleeping, breathing and stomach/digestion
Who Can Participate?
The FOP Registry is open to anyone with FOP. The information can either be provided by the person living with FOP or their caregiver—someone who can provide detailed information about symptoms, flares and doctor visits.
You enter this information on a dedicated website. It's secure and simple, and only anonymous information (without your name or contact information) is ever shared with researchers.
Mobile App Now Available
Now you can enter your FOP Registry data on an app for the iPhone.
Ready to Get Started?
Start by going to the FOP Registry website at FOPregistry.org and click the Join Now link.
- You'll be asked to gather some information and complete the enrollment survey.
- Be sure to give yourself 30-60 minutes for this first step.
- After completing the Enrollment Survey, you'll be sent a reminder every 6 months to sign back in and fill out a new Follow-Up Survey.
- Follow-Up Surveys take less time to complete than the enrollment survey.
Your Participation Matters
The FOP Registry is the largest database on FOP in the world—because of people like you who take part and contribute their information. Data can be entered by you or a caregiver into the patient portal, or by your physician into the medical portal. Learn more about the Medical Portal Here.
By taking part in the FOP Registry, you:
- Help grow the knowledge about FOP
- Contribute to helping find effective treatments and possibly a cure
- Help doctors better understand FOP and improve patient care
- Take part in something that directly benefits the entire FOP community
The key value of the FOP Registry is that data is collected over a long period of time. That means the information shows the way FOP affects people over time. This is critical to better understanding FOP, which helps researchers, doctors and drug developers—and ultimately, people living with the disease.
Need an extra incentive to get started or complete your next survey?
Now, any FOP Registry participant who completes an entire survey (Enrollment or Follow-Up) will receive an electronic reward card worth $25. Rewards can be used online or anywhere the Visa credit card is accepted.
We know it takes time and effort to get started and to complete the surveys every 6 months. That's why we want to say thanks with this FOP Registry Rewards Program. All you have to do is log in and start contributing information. For more information on the program, visit the Registry Rewards FAQ page.
Still Not Sure?
Here's a Resource to Help You Learn More
Learn more about the FOP Registry by taking a look at this brochure available in 7 languages. See why joining the FOP Registry means directly contributing to an important research effort!VIEW BROCHURE
FAQS - Frequently Asked Questions About the FOP Registry
Find out answers to some common questions about the RegistryGO TO FAQsSTART PARTICIPATING
Contact the FOP Registry
Please contact the FOP Registry in your preferred language with any questions you may have.