Tag Archives: Progeria Research Foundation

2014 Advocacy Awards Dinner

trofeoOn March 12, Research!America honored extraordinary leaders in medical and health research advocacy during the 2014 Annual Advocacy Awards at the Andrew W. Mellon Auditorium in Washington, DC.

We extend our congratulations to the honorees: Reps. Frank Wolf (R-VA) and Chaka Fattah (D-PA); actress Glenn Close and her family for their work to end the stigmas and misunderstandings surrounding mental illness; Leroy Hood, MD, PhD, president of the Institute for Systems Biology; Kathy Giusti, founder and CEO of the Multiple Myeloma Research Foundation (MMRF); Reed Tuckson, MD, managing director of Tuckson Health Connections; and The Progeria Research Foundation (PRF).

While much has been done to advance research, we have a long way to go.

13130132595_bc0c860475_o“Few out there seem to connect the dots to understand that federal funding is essential to develop the foundation of knowledge which is essential for American enterprise in developing the products and therapies that make our lives longer, healthier, and happier,” said Research!America Chair and former Member of Congress The Hon. John E. Porter in remarks at the Dinner. “There’s nothing more important to our future than investments in science, research, innovation and technology.”

And we agree! Contact your representatives and tell them to make research funding a higher priority.

Distinguished guests included current and former members of Congress and administration officials. Sen. Angus King (I-ME), Rep. David Price (D-NC), Rep. Nita Lowey (D-NY) and Rep. Scott Peters (D-CA), joined the celebration. Research!America board members,  The Hon. Mike Castle, The Hon. Kweisi Mfume and The Hon. Patrick Kennedy also attended the event along with former Congresswoman Mary Bono , former HHS Secretary The Hon. Dr. Louis Sullivan, NIH Director Dr. Francis Collins, NSF Acting Director Dr. Cora Marrett, and PCORI Executive Director Dr. Joe Selby.

Click here to see photos.

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International Rare Disease Day 2014: Joining Together for Better Care

The last day in February marks the annual International Rare Disease Day, first celebrated in 2008 by European coordinators the European Organization for Rare Diseases (EURORDIS).  For the past five years, the United States has participated in this effort thanks to the hard work of the National Organization for Rare Disorders (NORD) and other committed alliances and patient advocacy groups.

Rare diseases are classified in the U.S. as illnesses affecting fewer than 200,000 individuals – about 0.005% of Americans.  These patients and their families often struggle to be heard and there are major gaps in research on treatments and cures due to the small percentage of the population impacted by these diseases.  However, advocacy groups across the nation, often coordinated by patients and their families, are leading the way to increase the voice of those with rare diseases and to ensure research on these illnesses receives well-deserved funding.  Three examples include:

  • The Progeria Research Foundation has been fighting to increase awareness and support of Progeria since 1999.  Hutchinson-Gilford Progeria Syndrome (HGPS, Progeria) is a disorder that causes accelerated aging that eventually leads to pre-mature death due to heart disease.  Drs. Leslie Gordon and Scott Berns, along with their son Sam who passed away in January from the syndrome, have been vocal advocates on Capitol Hill and were featured in the recent HBO special “Life According to Sam.”  Funding raised by PRF contributed to the 2012 discovery of the first-ever treatment for children with the lethal disease.  The Progeria Research Foundation will be honored by Research!America with the Paul G. Rogers Distinguished Organization Advocacy Award on March 12, 2014. Continue reading →

A Weekly Advocacy Message from Mary Woolley: Promising process; disappointing progress

Dear Research Advocate:

The omnibus appropriations bill about to become law demonstrates that bipartisanship and pseudo-regular order is achievable. We won’t know for sure if we have true “regular order” until Congress proceeds through the FY15 appropriations process in a timely manner — something that hasn’t happened for many years. The importance of regular order is that the public’s interests are heard from in hearings, and every Member of Congress participates in priority-setting instead of only having the opportunity to cast a single up-or-down vote. Regular order is worth working toward, since at least one priority we all care about did not fare well in the omnibus.

The omnibus has failed to fund NIH at a level that fully reverses the impact of sequestration on the agency’s baseline funding level, much less establishes a growth trend that can fully unleash the potential inherent in the sequencing of the human genome and other research breakthroughs. As Drs. Paul Stoffels and Alan Leshner make crystal clear in an op-ed in Politico Magazine, we can’t settle for “better than sequestration.” If our nation wants to thrive, we need to grow our investment in science. Between 2010 and 2013, U.S. federal investments in science fell to less than 1% (.82%) of the economy. That’s the lowest it’s been in 50 years! As you know, this comes at a time when foreign nations are rapidly ramping up their R&D programs and taking a page from our playbook. Remember that global competitiveness in medical research is a pivotal determinant of our global economic competitiveness overall. We aren’t just talking about the future of our scientific enterprise, we are talking about the future of our economy. Are we truly willing to cede leadership in global R&D? (See also our statement cited in The Hill and other media outlets, as well as my interview today with UDC.) Continue reading →

Research!America Honors Leaders in Medical and Health Research Advocacy

Glenn Close, Dr. Leroy Hood, Dr. Reed Tuckson, Kathy Giusti and the Progeria Research Foundation to Receive 2014 Research!America Advocacy Awards

ALEXANDRIA, Va.-October 22, 2013-Research!America’s 18th annual Advocacy Awards will honor extraordinary advocates of medical and health research who are distinguished in their commitment to advancing medicine and health. The event will take place on Wednesday, March 12, 2014, at the Andrew W. Mellon Auditorium in Washington, DC, as a part of Research!America’s 25th anniversary commemoration.

The 2014 Advocacy Award winners are actress Glenn Close and her family; Leroy Hood, MD, PhD, president, Institute for Systems Biology; Kathy Guisti, founder and CEO of the Multiple Myeloma Research Foundation (MMRF); Reed Tuckson, MD, managing director, Tuckson Health Connections; and The Progeria Research Foundation (PRF). The winner of the Edwin C. Whitehead Award for Medical Research Advocacy will be named by Research!America’s Board of Directors later this year.

“This year’s honorees have transformed the lives of many individuals across the country through their remarkable achievements and advocacy for medical and health research,” said Mary Woolley, president and CEO of Research!America. “Their unwavering dedication is helping to elevate research in the national conversation and inspire a new generation of advocates.” Continue reading →

Life According to Sam

SamNext Monday, October 21 at 9:00 pm EST, HBO will debut LIFE ACCORDING TO SAM, the story of Sam Berns, a young boy with an extremely rare and highly-rapid aging disease called Progeria, and his family’s 13-year fight to find a cure.

This film tells a story not only about the love of physician parents trying to save their son – it also portrays a winding journey throughout the scientific process and the pivotal role that medical innovation plays in our lives. In the film we see children with Progeria gather in Boston from around the world – all expected to live no longer than 13 years – to receive treatment and participate in a first-of-its-kind clinical trial.

The film’s Oscar®-winning Directors Sean Fine and Andrea Nix Fine have earned multiple awards for LIFE ACCORDING TO SAM, winning the “Best of Fest” at the AFI Docs Festival in Washington, DC; the Audience Award at the Nantucket and Martha’s Vineyard Film Festivals the Best Storytelling Award at Nantucket and Best Feature Documentary at the Woods Hole and Rhode Island International Film Festivals.

The Progeria Research Foundation, a Research!America member, is leading the fight to find better treatments – and ultimately the cure – for Progeria, the rare and fatal “rapid aging” disease affecting children around the world.

You can learn more online, visit their Facebook page and follow them on Twitter.

Research into rare premature aging disorder praised as a model of “what’s right” in biomedical research

Recently, a group of scientists, clinicians and patients gathered in a suburb of Washington, DC to discuss scientific progress in the study of a rare premature aging disorder. This disease, Hutchinson-Gilford Progeria Syndrome—often referred to as HGPS or simply progeria—is one you may not have heard of, yet. But the Progeria Research Foundation and families of progeria patients have been working hard to increase awareness and raise funds for research into this rare disease that results in death at an average age of only 13. Among the list of speakers at the conference was the Director of the National Institutes of Health, Francis Collins, MD, PhD and researchers from a number of Research!America member organizations. Continue reading →