Hundreds of Patients & Advocates Recognize Rare Disease Day in Massachusetts
Participants advocate for rare disease research and out-of-pocket expense caps
BOSTON, MA, February 29, 2016—More than 425 patients, advocates, caregivers and industry allies rallied today in recognition of International Rare Disease Day.
Rare Disease Day is an annual awareness day dedicated to elevating public understanding of rare diseases and calling attention to the special challenges people with rare diseases face.
“We are thrilled to celebrate Rare Disease Day, particularly with this year’s them of The Patient Voice,” said Robert K. Coughlin, President & CEO of MassBio, the life sciences trade association and co-organizer of the event. “The Massachusetts biotechnology community has long been at work discovering treatments and cures for rare diseases. We stand side by side with patients and stakeholders to advocate for policies that encourage research and ensure access to treatments and cures.”
According to the National Institutes of Health (NIH), a disease is rare if it affects fewer than 200,000 people. Nearly 1 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.
The Massachusetts Rare Disease Day celebration featured five patient or caregiver speakers:
Chris Anselmo, MBA Student and Miyoshi Myopathy Patient
Rachel Mooney, Daughter of a Parent with Posterior Cortical Atrophy (PCA)
Karen Richards, High School Student Living with Ehlers-Danlos Syndrome (EDS)
Tayjus Surampudi, College Student Living with Duchenne Muscular Dystrophy
The program was emceed by event co-organizer Katie Brandt, Caregiver & Community Resource Specialist at the MGH Frontotemporal Disorders Unit.
"For many patients and families living with a rare disease there is no treatment or cure for their condition, but that does not mean there is nothing we can do," said Brandt. "We don't have to wait a single minute to provide support and connections for families. We may not have a cure for every illness, but we have a cure for isolation and loneliness."
This year, participants advocated in support of Senate Bill 541, which would limit out-of-pocket expenses for prescription drug coverage, ensuring patients have access to the medicines they need.
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. Rare Disease Day is sponsored in the U.S. by the National Organization for Rare Disorders (NORD)®, the largest and leading independent, nonprofit organization committed to the identification, treatment, and cure of rare diseases.
For more information about Rare Disease Day in the U.S., go to www.rarediseaseday.us. For information about global activities, go to www.rarediseaseday.org). To search for information about rare diseases, visit NORD’s website, www.rarediseases.org.
Jessica Roche, MassBio, 617-758-9633, Jessica.firstname.lastname@example.org
Jennifer Huron, NORD, 203-744-0100, email@example.com