Author Archives: Noelle Madsen

 

15FORRARE-web-banner

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

H.R. 6, The 21st Century Cures Act of 2015, continues to gain broad support in the United States House of Representatives. The legislation will; increase the development of research through the National Institutes of Health, improve patient-focused drug development, accelerate the approval process for new medical therapies, provide guidance on Precision Medicine, modernize the clinical trial process, expedite patient access, and streamline data and health information.

The IPPF urges you to email the following Congressional Representatives and thank them for their support in co-sponsoring H.R. 6, the 21st Century Cures Act of 2015:

New Cosponsor: Rep. Jim Costa [D-CA16]

New Cosponsor: Rep. Timothy Walz [D-MN1]

New Cosponsor: Rep. Ben Luján [D-NM3]

New Cosponsor: Rep. Christopher Gibson [R-NY19]

New Cosponsor: Rep. Bill Johnson [R-OH6]

New Cosponsor: Rep. Bill Flores [R-TX17]

New Cosponsor: Rep. Julia Brownley [D-CA26]

New Cosponsor: Rep. Lois Frankel [D-FL22]

New Cospo nsor: Rep. Rodney Davis [R-IL13]

New Cosponsor: Rep. Cheri Bustos [D-IL17]

New Cosponsor: Rep. Joseph Kennedy [D-MA4]

New Cosponsor: Rep. Ann Wagner [R-MO2]

New Cosponsor: Rep. Joaquin Castro [D-TX20]

New Cosponsor: Rep. Barbara Lee [D-CA13]

New Cosponsor: Rep. John Sarbanes [D-MD3]

New Cosponsor: Rep. Robert Latta [R-OH5]

New Cosponsor: Rep. Steve Scalise [R-LA1]

New Cosponsor: Rep. Gregg Harper [R-MS3]

New Cosponsor: Rep. Pete Olson [R-TX22]

New Cosponsor: Rep. Adam Kinzinger [R-IL16]

New Cosponsor: Rep. Mike Pompeo [R-KS4]

New Cosponsor: Rep. Chris Collins [R-NY27]

New Cosponsor: Rep. Mimi Walters [R-CA45]

New Cosponsor: Rep. Rick Allen [R-GA12]

Click on the link to go to your Representatives website and contact them. Please use this sample letter to assist in writing your email.

If you have any questions or would like to be more involved in IPPF advocacy, please contact marc@pemphigus.org

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

The International Pemphigus & Pemphigoid Foundation (IPPF) and the entire pemphigus and pemphigoid community would like to thank the House Energy and Commerce Committee for its non-partisan approval yesterday of the 21st Century Cures Act by a vote 51-0.

This legislation has important significance to our community as it will help modernize and personalize healthcare, support disease research, encourage innovation, and improve patient access to treatment. The bill, a year-long in the making, was championed by committee Chairman Fred Upton (R-MI) and Representative Diana DeGette (D-CO).

H.R. 6, the 21st Century Cures Act, has been sent to the House or Senate for full consideration. The IPPF urges you to contact your Representatives and ask them to support this legislation.

The Senate Health, Education, Labor & Pensions Committee (HELP) is currently working on it’s own version of the initiative called America’s Leadership in Medical Innovation for Patients, led by Chairman Lamar Alexander (R-TN). The IPPF will keep you aware of the latest developments as they occur but we also encourage you to reach out to the following Senators to express the urgency in creating legislation that will benefit you and all rare disease patients.

Republicans by Rank
Lamar Alexander (TN)
Michael B. Enzi (WY)
Richard Burr (NC)
Johnny Isakson (GA)
Rand Paul (KY)
Susan Collins (ME)
Lisa Murkowski (AK)
Mark Kirk (IL)
Tim Scott (SC)
Orrin G. Hatch (UT)
Pat Roberts (KS)
Bill Cassidy, M.D. (LA)

Democrats by Rank
Patty Murray (WA)
Barbara A. Mikulski (MD)
Bernard Sanders (I) (VT)
Robert P. Casey, Jr. (PA)
Al Franken (MN)
Michael F. Bennet (CO)
Sheldon Whitehouse (RI)
Tammy Baldwin (WI)
Christopher S. Murphy (CT)
Elizabeth Warren (MA)

For information on 21st Century Cures, go to: http://energycommerce.house.gov/markup/full-committee-vote-21st-century-cures-act

For information on the Senate Health, Education, Labor & Pensions Committee (HELP) go to: http://www.help.senate.gov/

If you would like to get more involved in pemphigus & pemphigoid advocacy, please contact Marc Yale, Peer Health Coach marc@pemphigus.org.

Thank you for your support!

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/

 

Rare Disease Day (RDD) is a unique global advocacy effort to bring recognition of rare diseases as an international health challenge.

On March 2, 2015, rare disease patients, caregivers and advocates, rare disease organization, legislators, and industry representatives gathered at the California State Capitol. Will Zrnchik, IPPF CEO, welcomed the crowd and introduced Peter Saltonstall, President and CEO of the National Organization for Rare Disorders. Peter said, “NORD has been working … to find a therapy, make sure the pathways are clear, and to make sure that the incentives are there for industry to want to develop drugs for small populations.” California Assembly Member Katcho Achadjian (D-36) co-sponsored CA House Resolution 6 recognizing February 28, 2015 as Rare Disease Day in California.

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Inside the Capitol, Andrea Vergne, rare disease caregiver and advocate, told her grandson’s story living with a rare disease. Gary Sherwood, Communication Directors at the National Alopecia Areata Foundation, inspired attendees to become self-advocates and work with their elected officials. Adding to the advocacy theme, Eve Bukowski, California Healthcare Institute, echoed the need for patients to self-advocate. A special guest, California State Senator Dr. Richard Pan discussed the importance of rare disease advocacy and research.

Meanwhile, Marc Yale, IPPF Senior Peer Health Coach, and Kate Frantz, Awareness Program Manager, celebrated RDD events in Washington, DC. Marc attended NORD’s special preview of the new documentary “Banner on the Moon.” Patients, caregivers, advocacy groups and patient organizations saw Cindy Abbott’s inspiring. Cindy carries a NORD banner with her on several adventures to spread rare disease awareness. These included challenging herself to climb Mount Everest and participating in the 1,000-mile Alaskan Iditarod. Cindy’s perseverance and commitment to live life to the fullest were very inspiring.

Kate and Marc attended the American Medical Student Association’s Annual Convention. Two P/P patients, Liz Starrels and Mimi Levich, shared their P/P stories. Many students had never heard of P/P, or only briefly remembered it from their textbooks. This was a wonderful opportunity to spread awareness and encourage medical students to “Put P/P on their Radar!”

RDD 2                           RDD

Marc represented the P/P community at the Rare Disease Legislative Advocates (RDLA) conference.  There was an overview of the 21st Century Cures Initiative (http://energycommerce.house.gov/cures), which many believe is the rare disease community’s greatest hope for new and emerging treatments.

Marc also met with several Congressional Members and legislative health experts encouraging their support of the Orphan Product Extensions Now Accelerating Cures and Treatment Act of 2015, H.R. 971. This legislation would re-purpose existing drugs for rare diseases allowing patients greater access to vital treatments.

Marc RDD                                        Marc RDD 2

Marc attended a symposium celebrating rare diseases research efforts across NIH, the Federal Government and the entire advocacy community.

There are over 7,000 rare diseases. 1 in 10 Americans are affected and 95% of these diseases have no approved treatment. Rare Disease Week and Rare Disease Day is an opportunity for the Pemphigus and Pemphigoid community to be heard by advocating for yourself and others.

If you have questions about current legislation or would like information on how you can become involved, please contact marc@pemphigus.org.

Feb. 26, 2015 – SACRAMENTO, Calif. — Join rare disease patients, caregivers and other health care advocates in California on March 2, 2015 to raise important awareness for rare diseases as part of Rare Disease Day®.

During the event, patients and advocates will come together to articulate with one voice the shared message that millions of people around the world are suffering and need our help. Our patients need earlier diagnosis; safe, effective treatments; and assured access to medical care and other services.

Many important decisions related to rare diseases are made at the state level, including newborn screening; support services to help families cope with complex medical needs; an environment that promotes innovative medical research and product development; and insurance practices that assure patient access to medically-necessary therapies. The implementation of the Affordable Care Act has highlighted the increasingly important role of state policies and programs in assuring that the health care needs of Americans are addressed.

The event will be held on the North Steps of the Sacramento State Capitol at 10:00am. Speakers include Will Zrnchik, CEO, International Pemphigus & Pemphigoid Foundation, Peter Saltonstall, President and CEO, National Orginzation for Rare Disorders, and 2015 Rare Disease Day Resolution co-sponsor Assemblymember Katcho Achadjian. Immediately afterwards, people are encouraged to join speakers, patient advocacy organizations, and others to hear patient stories inside the Capitol, Conference Room #126.

The event is organized by patient advocates who have joined with the National Organization for Rare Disorders (NORD)®, a leading independent, non-profit organization committed to the identification, treatment, and cure of rare diseases, and national sponsor of Rare Disease Day in the U.S., to educate and inform the public, elected officials, legislative staff and the media.

According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 Americans. One in 10 Americans live with a rare disease—affecting 30 million people—and two-thirds of these patients are children. There are more than 7,000 rare diseases and only approximately 450 FDA-approved medical treatments. Many diseases are not being studied by medical researchers. Often, research gets funded by the families and friends of patients or by patient organizations.

Rare Disease Day is an annual awareness day celebrated around the world dedicated to elevating public understanding of rare diseases and calling attention to the special challenges faced by patients. Rare Disease Day takes place every year on the last day of February (February 28 or February 29 in a leap year)—the rarest date on the calendar—to underscore the nature of rare diseases and what patients face. It was established in Europe in 2008 by EURORDIS, the organization representing rare disease patients in Europe, and is now observed in more than 80 nations.

For more information about Rare Disease Day in the U.S., go to www.rarediseaseday.us. For information about global activities, visit www.rarediseaseday.org). To search for information about rare diseases, visit NORD’s website, www.rarediseases.org.