PRESS
Push to Cure Rare Diseases
March 10, 2010
NIH-Funded Research Study
March 8, 2010
250 Million People Worldwide Estimated to Suffer From Rare Disease
March 8, 2010
GENE THERAPY REVERSES EFFECTS OF LETHAL CHILDHOOD MUSCLE DISORDER IN MICE
February 28, 2010
CHI SUPPORTS RESEARCH AND HOPE FOR PATIENTS OF RARE DISEASES
February 25, 2010
RARE DISEASE ADVOCATES UNITE TO TRANSLATE SLOGAN OF GLOBAL GENES PROJECT IN TIME FOR WORLD RARE DISEASE DAY!
February 25, 2010
reco® jeans SUPPORTS CHILDREN WITH RARE DISEASES
February 23, 2010
MILLIONS AROUND WORLD TO OBSERVE RARE DISEASE DAY ON SUNDAY
February 23, 2010
GLOBAL GENES PROJECT TO RAISE AWARENESS FOR MILLIONS OF CHILDREN LIVING WITH RARE DISEASE
February 1, 2010
GALAPAGOS TO FOCUS ON RARE DISEASES IN STRATEGIC SHIFT
January 26, 2010
THE PATIENT ASCENDANT
January 18, 2010
FUTURE OF NEWBORN SCREENING ENVISIONED: PROCEEDINGS NOW VIEWABLE ONLINE
January 7, 2010
HUNTING NEWBORN TESTS FOR SUPER-RARE GENE DISEASES
January 5, 2010
THE LONELINESS OF FIGHTING A RARE CANCER
January 5, 2010
DONATE GAMES CHARITY CONNECTS COMMUNITIES WORLDWIDE
December 21, 2009
DONATEGAMES TURNS USED VIDEO-GAMES INTO FUNDING FOR MEDICAL RESEARCH TO HELP KIDS
November 25, 2009
CHILDREN'S RARE DISEASE NETWORK RECEIVES LIFE TECHNOLOGIES FOUNDATION GRANT
November 17, 2009
BABY Z CURED OR RARE DISEASE IN 3 DAYS
November 11, 2009
SOCIAL NETWORKING SAVIORS: TWITTER, FACEBOOK USED IN EFFORT TO HELP SAVE A BABY'S LIFE
October 29, 2009
U.S. AND EUROPEAN RARE DISEASE ORGANIZATIONS SIGN STRATEGIC ALLIANCE
October 28, 2009
RARE DISEASE CENTER HOSTS SYMPOSIUM ON NEW STRATEGIES
October 27, 2009
RARE FIND
October 23, 2009
NEW FDA GROUPS FOR RARE, NEGLECTED DISEASES COULD SPEED PATH TO MARKET
October 12, 2009
ARNOLD NATIVE TO RUN ACROSS SAHARA DESERT
August 18, 2009
CAMP SUNDOWN SHINES IN THE BRONX
August 13, 2009
RESEARCHERS IDENTIFY NEW FUNCTION FOR PROTEIN MISSING IN DUCHENNE MUSCULAR DYSTROPHY
August 4, 2009
AMERICANS STRUGGLE TO PAY FOR HEALTHCARE: STUDY
June 22, 2009
DEAL REACHED TO CUT DRUG COSTS
June 20, 2009
ONE GIRL'S HOPE, A NATION'S DILEMMA
June 14, 2009
IN RARE DISEASE, A FAMILIAR PROTEIN DISRUPTS GENE FUNCTION
May 26, 2009
NEW INSTITUTE WILL STUDY RARE DISEASE
May 20, 2009
UNC-DUKE STUDY: IMPAIRED BRAIN PLASTICITY LINKED TO ANGELMAN SYNDROME LEARNING DEFICITS
May 10, 2009
TO MAKE PROGRESS IN RARE CANCERS, PATIENTS MUST LEAD THE WAY
May 9, 2009
MO. VOTES TO ADD 5 DISEASES TO NEWBORN SCREENINGS
May 9, 2009
SIGNATURE GENOMIC LABORATORIES DETECTS CHROMOSOME ABNORMALITIES IN INDIVIDUALS WITH PALLISTER-KILLIAN SYNDROME WITHOUT INVASIVE SKIN BIOPSY
May 7, 2009
MIRACLE FOR MATTHEW
May 5, 2009
SHRINKING BABY MAGGIE AGNEW BAFFLES DOCTORS
May 4, 2009
"PERSPECTIVES" PODCAST SERIES
Consumer Group Sues Over Health Insurance Increases
Podcast: March 8, 2010
The Burrill Report (March 8, 2010): Consumer Group Sues Over Health Insurance Increases (.MP3,10.81 Mb)
Anthem Blue Cross, California’s biggest for-profit health insurance company, has been on the hot seat lately. Its plan to raise premiums for individuals by as much as 39 percent landed the CEO of its parent company, Wellpoint, in front of a Congressional committee and reignighted efforts in Washington to pass healthcare reform legislation. Now the advocacy group ConsumerWatchdog has filed a class-action lawsuit against Anthem over the rate increases saying they violate California law. We spoke to Jerry Flanagan, healthcare policy director for ConsumerWatchdog, about his group’s lawsuit, what Anthem’s actions mean for reform, and why the proposed healthcare legislation wouldn’t have prevented Anthem from instituting the rate increases in the first place.
TAILORING TREATMENTS TO KIDS
Podcast: January 11, 2010
'Perspectives' Podcasts presented by Children's Rare Disease Network >>
In treating pediatric patients, doctors and hospitals are often limited by available devices or drugs that have not been optimized for the treatment of children. Don Lombardi saw the absence of appropriate devices and drug formulations for kids as a significant opportunity to effect positive change. Three years ago he founded the Institute for Pediatric Innovation in Cambridge Massachusetts to create new ways to foster, streamline, and stimulate development and commercial adoption of new medical products for children. We spoke to Lombardi about his organization, why there aren’t more devices and drugs designed specifically to meet the needs of children and the deadly toll that results.
BIOTECHS ADJUST TO THE NEW NORMAL
Podcast: January 18, 2010
Biotechs Adjust to the New Normal (.mp3, 16.34MB)
Chris Starr has lived through biotech windows, cycles, and bubbles. An industry veteran, today he is CEO and co-founder of Raptor Pharmaceuticals, which works on improving existing drugs through its targeting platform and formulation expertise. Like many executives, Starr has had to prioritize his pipeline and find ways to raise money during tough times. We talked to Starr about the changing environment for biotechs, how he’s adapted, and as times improve, whether or not there’s a new normal.
CURE THE PROCESS
Rare disease researcher turned advocate wants the FDA to change its approach to how it reviews new drugs for rare diseases.
Podcast: January 4, 2010
'Perspectives' Podcasts presented by Children's Rare Disease Network
For 18 years, Emil Kakkis saw first hand the challenges of developing treatments for rare diseases and bringing them to market. First as a researcher, and than as chief medical officer of the biotech company BioMarin, he worked to develop Aldurazyme, an enzyme replacement therapy for a rare genetic disorder known as MPS I. In 2009, he formed The Kakkis EveryLife Foundation in the hopes of expanding access to treatment for patients with rare diseases. To that end, the Foundation has initiated the CureTheProcess campaign to focus on increasing the predictability of the regulatory pathway for treatments of rare diseases. We spoke to Kakkis about what changes are needed in the way rare disease treatments are developed and regulated, how to transform the drug development process to meet the needs of more patients with rare diseases, and why a clear pathway for orphan treatments will shorten and reduce the financial risk associated with development of rare disease therapeutics and attract new investment into the area.
A FATHER SEES A RARE DISEASE GETS ITS DUE ATTENTION
Podcast: November 23, 2009
'Perspectives' Podcasts presented by Children's Rare Disease Network
When Brad Margus learned two of his son had Ataxia Telangiectasia, a progressive, degenerative disease commonly known as A-T, he began an ongoing effort to educate himself about the workings of biomedical research and drug development. He and his wife launched the A-T Children’s Project in 1993 to help fund and accelerate research into the rare disease. We spoke to Margus about his journey around A-T, his efforts to combat the disease, and what others parents of children with a rare disease can learn from his experiences.
PATIENTS EMBRACE VIRTUAL BIOTECHS TO DEVELOP DRUGS
FOR THEIR CONDITIONS
Podcast: October 28, 2009
'Perspectives' Podcasts presented by Children's Rare Disease Network
As a business model, the notion of the virtual biotech by some estimates has been around for as long as 25 years. But for people afflicted with a rare disease and their families, the notion of the virtual biotech is generating energy and enthusiasm around raising funding to create ad hoc teams to conduct specific drug development projects. We spoke to Jonathan Jacoby, CEO of the RARE Project, about what it takes to create a virtual biotech, what successes there have been to date, and how this reflects a broader change in the role of patients and their families in drug development.
NEW APPROACHES TO TREATMENT NEEDED IN PEDIATRIC CANCER
Podcast: July 24, 2009
'Perspectives' Podcasts presented by Children's Rare Disease Network:
Rare Disease Patient Advocates – Part Three
In this third in a series of podcasts focused on rare disease patient advocates jointly produced by The Burrill Report, the California Healthcare Institute, and the Children’s Rare Disease Network, we speak to Susan Cornelius, business advisor to The Nicholas Conor Institute for Pediatric Cancer Research. When her granddaughter struggled against cancer, she was frustrated that new technology was unavailable to her and that all doctors could offer were surgery, radiation or chemotherapy – what she characterized as cut, burn, or poison the child for a disease, in her granddaughters case, that was genetically-based. Cornelius lost her granddaughter to cancer in 2008, but now works to bring about changes to the way research is conducted.
A NEW MODEL FOR FUNDING RESEARCH
Podcast: July 17, 2009
'Perspectives' Podcasts presented by Children's Rare Disease Network:
Rare Disease Patient Advocates – Part Two
In this second in a series of podcasts focused on rare disease patient advocates jointly produced by The Burrill Report, the California Healthcare Institute, and the Children’s Rare Disease Network, we speak to Beth Anne Baber, CEO of The Nicholas Conor Institute for Pediatric Cancer Research. When Baber’s son Nicholas was diagnosed with stage 3 neuroblastoma she and her husband, both cancer researchers, fully understood the seriousness of the diagnosis. Because of the location of the tumor, radiation and surgery were not options. But as they explored treatments they discovered a startling fact. Most cancer therapies used to treat children were about 25 years old and drug companies weren’t generally pursuing new therapies for children with various cancers because the markets simply weren’t large enough. Her institute offers a new model. It seeks out industry partners to help develop specific technologies it wants to bring into the clinic while helping to fund that work.
SEARCHING FOR A CURE
Podcast: July 10, 2009
'Perspectives' Podcasts presented by Children's Rare Disease Network:
Rare Disease Patient Advocates – Part One
In this first in a series of podcasts focused on rare disease patient advocates, we speak to Debra Miller, president and founder of Cure Duchenne.
When Miller’s five-year-old son was diagnosed with Duchenne Muscular Dystrophy, she faced grim news. The progressive muscle loss caused by the disease typically leaves these children wheelchair-bound by age 10 and robs
them of their lives by 20. As she and her husband explored treatment options and ways they could become to raise money for research, they found that existing organizations were often focused on palliative care and focused on academic research rather than working with industry. Miller and her husband launched Cure Duchenne to help fund promising research at biotech companies in the hopes of seeing these therapies developed and commercialized. We spoke to Miller about her son’s story, new approaches to treating Duchenne, and the problems of funding cutting-edge research.
