Research study is open to participants worldwide to advance understanding and treatments for rare disease causing blistering that may be accompanied by severe pain, itching, burning, and stinging.
Sacramento, CA and Washington, D.C., March 31, 2017—The International Pemphigus & Pemphigoid Foundation (IPPF), a NORD member organization, has launched the largest-ever study to research pemphigus and pemphigoid that causes blistering of the skin and mucosa. Pemphigus and pemphigoid currently has no cure.
“The IPPF Natural History Registry will provide a complete picture of each patient’s experience with pemphigus and pemphigoid,” said Marc Yale, Interim Executive Director, of the IPPF. “We are launching this initiative to help fill the missing link researchers and medical experts need to advance research and get to a cure.”
To help drive awareness and participation, the IPPF will provide information about the existence of the registry via email, website advertisements, flyers, the Quarterly newsletter, and at its’ Annual Patient Meeting to interested members of the pemphigus and pemphigoid community
“Our goal is to enroll as many patients, or their parents or legal guardians, as possible,” said Yale. “The success of the registry is dependent upon community participation.”
The IPPF Natural History Registry is a natural history study that consists of electronic surveys to collect infbout the patient experience and disease progression. Patients, or their caregivers or guardians, can enter information from anywhere in the world. The data is made anonymous and stored securely in an online portal called a registry. The IPPF may share the data but not your personal identifying information with individuals or institutions conducting research or clinical trials, as approved by the study’s governing board that includes scientists, doctors and patient advocates.
The IPPF is launching the study in collaboration with the National Organization for Rare Disorders (NORD), an independent charity that built its natural history study platform as part of its mission to help identify and treat all 7,000 rare diseases. Funding is supported by a cooperative agreement between NORD and the U.S. Food and Drug Administration (FDA). The FDA has praised NORD’s program as a helpful tool “that protects the security and privacy of personal information, while making valuable information available to a researcher or drug developer interested in creating a new therapy for a rare disease.”[1]
NORD President and CEO Peter L. Saltonstall said, “NORD’s natural history studies platform empowers patients and families to drive research and eliminate some of the unknowns that still exist in rare diseases. We are glad to be working with our Member Organization on this project and thank the FDA for its support and ongoing commitment to help people with rare diseases.”
Pemphigus and pemphigoid are rare autoimmune blistering disorders with an estimated U.S. annual incidence of ~2,500 and world incidence of ~40-50,000 people. These conditions are difficult to diagnose and debilitating for the patient. If extensive, blistering can lead to life-threatening fluid loss, infection, and disfigurement. PV can also cause significant damage to the skin, including nail loss and pigmentary alteration, making timeliness of intervention and treatment essential to prevention of disability.
For more information, visit https://www.pemphigus.org/.
About the IPPF
The International Pemphigus & Pemphigoid Foundation’s most important objectives are to provide patients and doctors worldwide with information about pemphigus and pemphigoid, and to provide patients and their caregivers much needed comfort and support so they can continue to live active, productive lives.
About National Organization for Rare Disorders (NORD)
An independent 501(c)(3) nonprofit organization, NORD is the leading advocacy organization representing all patients and families affected by rare diseases in the U.S. Established in 1983, NORD is committed to the identification, treatment and cure of the 7,000 rare diseases that affect 30 million Americans, or 1 in every 10 people, through programs of advocacy, education, research, and patient/family services. In addition to educational resources for patients, families, medical professionals and students available on its website (www.rarediseases.org), NORD represents 250 member organizations and collaborates with many others in specific causes of importance to the rare disease patient community.
Contact:
For IPPF: Marc Yale, [email protected], 916-922-1298 ext.7106
For NORD: Jennifer Huron, [email protected], 203-744-0100
[1] Woodcock, J. “The more we know about rare diseases, the more likely we are to find safe and effective treatments.” FDAVoice (Oct. 23, 2014)
