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What would you say if you had 15 seconds to advocate for pemphigus and pemphigoid?

Would you tell them about the statistics? Would you talk about the person you know who is struggling with their disease? The sister or brother, friend or co-worker who struggles to find treatments and support? Or would you talk about your own diagnosis and how its existence has changed your world?

Global Genes is proud to launch the #15ForRARE Campaign—a social media video movement highlighting the individual voice in the rare community. Through participation in this campaign, #15ForRARE video activists (YOU!) are entered into a contest to win two sets of tickets to this year’s RARE Patient Advocacy Summit, and Tribute to Champions of Hope Gala. A Prize valued at $1,100. There will be two winners- each able to bring a guest.

Now is your time to speak up and tell the world why you “Care About RARE.” This movement is for patients, advocates, and industry alike. Through our individual voices we will unite and bring attention to a community that so deserves it.

Here’s how you get involved and get entered into our contest:Advocate for Rare

• Film yourself “selfie” style answering the prompt “I care about rare because…”
•Video must be no longer than 15 seconds
• Video must be uploaded to our contest entry form, found here.
• Video must be posted on at least one social media platform (Facebook,Twitter, Instagram, Youtube, Vimeo, Vine) with the hashtag #15ForRARE and#GlobalGenes (Don’t forget to use #healourskin too!)

And that’s it!

Each place you post your entry is an extra entry in the contest. The winner will be selected at random from the pool of applicants. Two winners will get two free entries to Global Genes annual RARE Patient Advocacy Summit and our Tribute to Champions of Hope Gala (airfare and accommodations not included, no cash prize offered). If you have already purchased tickets and win the contest, you will be reimbursed for their cost.

Global Genes will be using the videos submitted to us for cause marketing as well as will feature them at their RARE Patient Advocacy Summit!

My name is Todd Kuh and prior to December of 2010, I was a healthy, active person who had a passion for bicycle racing.  After being diagnosed in December of 2010, I was a Pemphigus patient and no longer an athlete.  My physician informed me shortly after my diagnosis that I would not race again.  He explained that physical and emotional stress must be carefully monitored and controlled from that moment on as stress could trigger the disease.  As I sat in the exam room I heard “Chronic disease”, “no known cure”, “painful”, and “potentially life threatening” and wondered how I would tolerate this new reality. 

On my way from the physician’s office that afternoon to the pharmacy to purchase my bag of medications (trying to figure out what just happened to my life), I decided to take one last bike ride.  As I rode that afternoon, still feeling strong from the countless hours of training, I suddenly realized that I would never feel this way again.

I always believed that I was blessed to be able to ride and race my bike and never took a single ride for granted.  Throughout my medical treatment, I leaned on friends, family and lessons learned from training and racing to provide me with the positive attitude I needed to overcome this disease.  Although I practiced a healthy lifestyle prior to diagnosis, I implemented a revised health and meditation program that would maximize the treatment I received.

After nearly three years of intensive treatment under the careful watch of Dr. Sergei Grando at the University of California, Irvine, School of Medicine, through healthy lifestyle choices and with the incredible support from friends and family, I’m in remission.

Dr. Grando again cautioned me of the factors that could contribute to relapse, which included physical stress.  He assured me that should I choose to return to training and bicycle racing, my remission would most certainly be brief.

I realized that a healthy lifestyle incorporates exercise, which of course would be moderate in my case.  With Dr. Grando’s approval, I began cycling again.  As the months passed and my fitness slowly began to return, I realized that I was capable of doing more than I or Dr. Grando believed I could.  However, I battled with the constant fear that my exercise program would trigger a relapse, which created unnecessary stress.

I connected with the IPPF and spoke with Victoria, a Pemphigus patient who told me about her running and physical activities.  We shared our stories and were surprised and encouraged to learn that we both successfully live moderately active lifestyles while in remission.  We agreed that exercise contributes to a variety of health benefits in addition to our sustained remission.   My conversation with Victoria simply changed my life and gave me confidence that I could live an active lifestyle, which further contributed to my emotional well being.  I’m certainly much slower and I don’t go very far, but I cherish every pedal stroke!

Once I realized that I was capable of a level of physical activity beyond what I previously expected, I began to experience the calming meditative qualities that exercise provides.  Simply, moderate exercise reduces stress.  While this is certainly not news to anyone, it’s critical for Pemphigus patients.

It was at this time that Victoria and I realized that a healthy program integrating sound nutritional choices, active stress reduction and moderate exercise would greatly benefit Pemphigus and Pemphigoid patients

Chasing Down Pemphigus and Pemphigoid is a program intended to provide hope and a new perspective to the patient community, raise funding for critical research studies, awareness and patient support programs while promoting active, healthy lifestyles as a critical part of disease management.

The Chasing Down Pemphigus & Pemphigoid program is an ongoing campaign that challenges every patient to make better nutrition choices, go for a walk each day, take a yoga class, meditate, take on a new hobby or do anything that improves our health, attitude and emotional well being.  Chasing Down Pemphigus & Pemphigoid is about proving that we can do more than what was thought possible.

The IPPF is a small non-profit foundation that provides important patient programming and support.  While Chasing Down Pemphigus & Pemphigoid is first and foremost a year-round program designed to motivate and support patients in their efforts to live healthy and active lifestyles, it is also a critical fund raising campaign that assists the IPPF in continuing to provide all of the support we all greatly appreciate.

Check your mailbox and your email box in the coming months for Chasing Down Pemphigus & Pemphigoid articles on nutrition, stress reduction, exercise, support and patient stories (including YOUR story!).  Can’t wait?  Then email me at: todd@pemphigus.org or Victoria at: Victoria@pemphigus.org to get going.

Dear pemphigus & pemphigoid community,

The House of Representatives is expected to vote on H.R. 6, the 21st Century Cures Act, the week of June 15th. The IPPF is urging our patient community to show its united support for this crucial legislation by contacting your representative today and ask them to co-sponsor H.R. 6, the 21st Century Cures Act.

This bill includes many provisions that could benefit our community and the rare disease community as whole including:

  • Billions in funding for the National Institutes of Health (NIH)
  • Hundreds of millions dollars in new funding for the Food & Drug Administration (FDA)
  • The OPEN ACT, which contains incentives that could double the number of rare disease treatments
  • The CURE ACT, that requires companies to publish expanded access policies
  • Patient focused drug development
  • It’s easy. Don’t wait! Go to this link to make your voice heard: http://www.congressweb.com/KAKI/39

For those not familiar with an action alert here are some simple instructions:
1. Click on the link above
2. Fill in your street address & zip code and click “take action”
3. Fill in the rest of your personal information and a personal message (optional) about what 21st Century Cures means to you
4. Review your message and click “send”

For more information on 21st Century Cures you can go to: http://energycommerce.house.gov/cures

For questions or if you are interested in getting involved in advocacy for the pemphigus & pemphigoid community, please contact Marc Yale marc@pemphigus.org

Preparing for your doctor’s appointment can help you get the most from your visit. Taking an active role in your care will make you an empowered patient. In addition, proactively learning about your disease will improve your healthcare and treatment experience. Collecting the information needed before a doctor’s appointment can ensure that you are organized and strengthen your doctor-patient relationship. Here are ten tips on how to prepare for the visit that will assist you in feeling better when you leave the doctor’s office.

1.      Have all your questions answered. Bring a checklist, and be ready to take notes

2.      Schedule enough time & bring your prescriptions

3.      Address priorities first & clarify concerns

4.      Remember to say “Thank You”

5.      Learn what tests are needed before the visit (if any)

6.      Have copies of your medical records

7.      Get a summary of your visit when you leave

8.      Fill out medical release to get your records

9.      Be patient

10.  Be confident and share your knowledge

Sometimes it is valuable to gain a second, third, or even fourth opinion when seeking a treatment for pemphigus and pemphigoid. Additional opinions also provide an opportunity for you to learn more about your condition and it can offer some peace of mind that you are approaching your disease with the best chance for a favorable outcome.

Remember, if you have questions don’t be afraid to “Ask a Coach” because when you need us we are in your corner!

In May 2015, Global Genes launched their 2nd annual Cox Prize for Rare Compassion contest which encourages 1st and 2nd year medical students to get to know a rare family and write an essay based on their experiences. If the medical student does not have a rare family to work with, Global Genes will match them with a rare family in their local area.

In just a few weeks, they have had requests from over 90 medical students for help in being matched with a rare family! These students represent dozens of medical schools all over the U.S. as well as Canada and the U.K.

Global Genes is asking our help in finding rare families we can match for this program. This is a wonderful way to create awareness for your disorder within the medical education community and possibly create a relationship that could shape the med student’s future career focus. The IPPF would love to have pemphigus and pemphigoid families represented in this fantastic program! What a great way to help spread awareness to the future of the medical community about pemphigus and pemphigoid! To let Global Genes know that you are interested in being a rare family for this program please email Carrie Ostrea carrieo@globalgenes.org

Please help us spread the word about their med student matching program by going to http://globalgenes.org/cox-prize-family to see what locations are currently asking for requests. This list gets updated weekly, so please check back often to see what new cities are requested.

Thank you so much for your support! If you have any questions, please do not hesitate to ask.

Carrie Ostrea
Advocacy Director / Parent Advocate
Global Genes – Allies in Rare Disease
http://www.globalgenes.org
(949) 248-RARE x110

IPPF Awarded Abbey S. Meyer Leadership Award by NORD

Washington, D.C. – IPPF, International Pemphigus & Pemphigoid Foundation, was honored today at the Portraits of Courage gala event as the recipient of the prestigious Abbey S. Meyer Leadership Award by NORD (National Organization for Rare Disorders).

NORD hosts the Portraits of Courage Gala annually to bring together the rare disease community and to honor and celebrate the year’s outstanding achievements.  During the event, they recognize the individuals, organizations, advocates and companies that are moving the community forward in the fight against rare diseases.

The award was presented to the IPPF for the organization’s outstanding advocacy achievements on behalf of patients with rare diseases. The award, named for NORD’s founding president, is presented each year to a NORD Member Organization for demonstrating outstanding leadership and representation of its members.

We are proud to work with NORD and other member organizations to provide a voice for those living with rare diseases. Each year, NORD, a nonprofit organization that represents the 30 million Americans with rare diseases, hosts the Portraits of Courage celebration, to celebrate the pioneering achievements of individuals and organizations.

The IPPF is the pre-eminent global organization dedicated to improving the quality of life of all people diagnosed with, or affected by, pemphigus or pemphigoid.

Please call Congress TODAY and ask them to include the OPEN ACT, HR 971 (Orphan Product Extensions Now, Accelerating Cures & Treatments) in the 21st Century Cures Legislation. The OPEN ACT has the potential to double the number of approved rare disease treatments available to patients. To date, 155 patient organizations support the OPEN ACT, including NORD, Global Genes, and the Genetic Alliance. By standing together we can ensure Congress helps rare disease patients.

Click here to take action: Stand up for Rare Disease Patients TODAY

Please share this alert widely and join this event on Facebook.

Why the OPEN ACT is important: Despite advances made possible by the Orphan Drug Act, 95 percent of the 7,000 rare diseases still have no approved treatments. Biopharmaceutical companies are not repurposing major market therapies to treat rare diseases because there is no incentive for them to do so.

The OPEN ACT is bipartisan legislation that creates an economic incentive for companies to repurpose drugs for rare diseases. The OPEN ACT could:
·         Bring hundreds of treatments to rare disease patients
·         Enable access to safe, effective and affordable treatments
·         Spur biotech investment, innovation, and foster clinical research at universities while creating new jobs

Learn more at: http://curetheprocess.org/ incentivize/

 

Results So Far

I had a follow-up appointment with Dr. Williams on July 17, 2014, one month after my first infusion and two weeks after the second. She looked at me and I swear her jaw dropped. She was amazed by how well I had responded. That was a fun appointment!

Jack Sherman 7

She had consulted Dr. Anhalt shortly before my treatment. Dr. Anhalt suggested going off of azathioprine a month after my second infusion (August 1), and to start a slow prednisone taper. I asked Dr. Williams if I should stop taking azathioprine now, two weeks before we were planning. We agreed I should stop taking it. One drug down!

Since then I haven’t taken azathioprine. Better yet, I’ve been on a continual prednisone taper. I started on 25 milligrams every other day. A week later, on July 23, 2014 (three weeks after my second infusion) I took these pictures. I was completely lesion free! I was ecstatic to say the least. This far exceeded my wildest dreams!

In January 2014 I am down to 2 milligrams of prednisone, every other day! This is the lowest dosage of prednisone I have ever been on. The best news is my skin is completely void of lesions. Sure I’ve had one or two minor ones, but nothing that doesn’t clear up quickly. Pretty amazing considering where I started.

I’m not claiming remission — yet! While it’s easy to be confident about my recovery, I prefer to say I’m very optimistic about my future living with pemphigus. What I have learned over the years about this disease is things can change pretty quickly. I could end up in total remission, or I could end up needing another round of Rituximab. Either way, I believe I will be better off than had I not opted for rituximab. For that I’m very grateful!

 Continued Support and Education

Each individual is just that, an individual. These diseases aren’t like more common diseases, like Type II Diabetes. If you go to 10 doctors after a diabetes diagnoses you will probably hear the same thing and expect the same results. With pemphigus and pemphigoid being rare, ultra orphan autoimmune diseases, your results and advice will likely vary.

Even though I’m a Peer Health Coach, Marc Yale has continued to be my coach. I can’t thank him enough for his time, knowledge and support he’s given me over the years. My goal is to help patients like Marc has helped me, and share this knowledge with them each and every day. Reach out to the IPPF and use its wealth of knowledge and patient resources. If you can attend the IPPF Patient Conference, I encourage you – I implore you – to go. The information and fellowship really does make a difference!

In the end, my biggest piece of advice to you is to be proactive in you care and treatment. Work with your physicians and create a team committed to your success. Share what you learn from your coach, attending a conference, or from a conference call with your doctor. Ask them to contact the IPPF who will connect them with a P/P expert. Whatever you do, it’s your health and quality of life at stake, so make informed, educated decisions. I did and couldn’t be happier!

Good luck, and good health to you all!

Part One
Part Two

Time for the Infusion

As the treatment drew near, I had a lot of questions for Dr. Williams. She felt an oncologist was better equipped to answer them, so she scheduled a consult with one. That was a great move. The oncologist answered all my questions. He said prescribing and administering Rituxamab is an everyday occurrence for the infusion room. He said they give this treatment to leukemia and lymphoma patients who are in very poor health. Since I was in relatively good health, his concerns of complications for me were minimal. That was reassuring.

I had to do a lot of lab tests, which is common for intravenous treatments affecting the immune system. I was tested for several types of hepatitis, HIV, TB, and other infectious ailments. You can see from my “before” picture how bad my skin was.

I was treated using the Rheumatoid Arthritis Protocol (1,000 mg intravenously on days 1 and 15). My first dose was administered on June 17, 2014 and lasted 6 hours; the second on July 1, 2014, lasted 4 hours. I was relieved that other than a little jitteriness caused by a steroid drip, I had absolutely no side effects or reactions. It literally felt like I was getting a routine saline solution infusion.

Jack Sherman 4 Jack Sherman 3

When I went in for my second infusion, there was no change in my disease activity. I didn’t expect to see any changes for at least a month. To my surprise, as you can see by this photograph comparison, I was seeing signs of improvement a week after my second infusion! I was still taking 250 milligrams of azathioprine and 25 milligrams of prednisone every other day.

Jack Sherman 6 Jack Sherman 5

Stay tuned for next week’s conclusion of Jack Sherman’s Road to Rituximab Story…

Part One
Part Three

This article was originally was posted at http://www.medscape.com/viewarticle/840206

The National Organization for Rare Disorders
The Importance of Rare Disease Education
Sophia A. Walker

February 25, 2015

Recently a wise professor told my class that we medical professionals are some of the most powerful people in the world. Indeed, we have the ability to meet people at their most vulnerable, sometimes on the very worst day of their lives, and help them. “This profession,” he told us, “is such a privilege that we must never miss the opportunity to have at least done some good for every patient.” Over the past several weeks, as I have planned rare disease awareness events and begun preparing to enter the clinical years of my medical education, I find myself considering these words more frequently. However, at the end of the day, I wonder how powerful we are, really…

My interest in rare diseases originated during my senior year of high school, when I first started volunteering at the National Organization for Rare Disorders, Inc. (NORD). I was overwhelmed to discover the many obstacles experienced by patients who have rare diseases. On a technical level, any disease that affects fewer than 200,000 Americans is considered rare. Of the more than 7000 rare diseases, only approximately 350 have treatments that are approved by the US Food and Drug Administration (FDA). I found that individuals with these diseases, almost two thirds of whom are children, show great courage and perseverance in the face of significant discouragement. Although I had always wanted to be a doctor, it was not until I witnessed such unrelenting determination to overcome barriers in healthcare that I discovered my own enthusiasm for medicine.

All physicians strive to provide attentive medical care with the utmost compassion and empathy; however, as medical professionals, we must also be our patients’ most vocal advocates. Although I was not yet a physician, I still wanted to contribute to this effort. I wanted to provide a forum for the nearly 30 million Americans with rare diseases whose voices often go unheard in the medical community, and I wanted to share this passion with my peers. Every year, my fellow students and I host a Rare Diseases Awareness Event. Patients, students, clinicians, and researchers come together to share their experiences and insights regarding rare diseases. We strive to shed light on the lives of these individuals by allowing them to share their own stories, sometimes for the very first time.

Although many students may assume that we do not need to know as much about rare diseases because we are unlikely to encounter them in our practice, this is simply not the case. In fact, every one of us preparing for medical careers will see patients with rare diseases, and the extent to which we prepare ourselves for this reality will determine the impact we can have on these patients’ lives. Patients who have a rare disease face difficulty in every step of medical care, including diagnosis, treatment, and preserving quality of life. Sometimes, patients go years without receiving the correct diagnosis for their condition. Once they finally have an answer, often no treatment is available for their condition. As future physicians, we must aim to improve these prospects; the first step in doing so involves developing a keen understanding of this patient population.

The opportunities for medical students to learn about rare diseases are vast. Gaining a basic understanding of how the experience of having a rare disease is different from having a more common disease is equally essential. The National Institutes of Health (NIH) has great information related to rare diseases on its website, and the NORD website provides overviews and links to more than 200 patient organizations that provide excellent information about specific rare diseases. Students can also apply for a free NORD student membership by writing to bhollister@rarediseases.org. Once you register, you receive a monthly eNews and quarterly newsletter specifically designed for students planning healthcare careers. If you’re attending the American Medical Student Association annual convention in Washington, DC, on February 27 and 28, come to the NORD booth in the exhibit hall where patients with rare diseases will be sharing their stories.

With each speaker I listen to at a rare diseases event, with each new person I meet, I am filled once again with immense pride that our efforts, if even in a small way, have done some good. Unlike many people who are involved in advocacy efforts in this area, when I began this work, I did not have a personal connection to rare diseases. However, after years of getting to know people who have experienced these struggles, I can say that I now have several. In fact, it is the memory of the individuals I have met and the satisfaction in having contributed to raising awareness that has guided my interests, served as an influence in many decisions, and ultimately has been the driving motivation in achieving my aspirations. With every step I take moving forward in my career, rare diseases comes along with me and will continue to do so.

My passion for rare diseases advocacy has become perhaps the foremost aspect that defines me and has made me who I am. It has given me direction, has made me a leader, and continually prepares me to become one of those physicians who will do some good. A couple of years ago, one of my undergraduate professors asked me, “Are you that rare diseases girl?” He went on to say that a student who had been inspired to research rare diseases after attending my event had approached him with an interest in working in his lab. This is the reason why I raise awareness for rare diseases. If just one more person every year becomes inspired, that may eventually make all the difference in the world. It turns out that, in the end, we are all powerful together. After all, according to the NORD motto: “Alone we are rare. Together we are strong.”®