Abstract: Background: This is the second article related to a review of the literature based on data from national cystic fibrosis (CF) registries up to June 2008 and covering a total of 115 studies. It focuses on two topics: neonatal screening (NS) and nutritional status, with particular reference to growth.Methods: Ten papers meeting the inclusion criteria were found on the topic of NS and its impact on the course of the disease, and were analyzed according to a dedicated grid. The issue of nutrition was addressed by 14 studies, analyzed according to similar criteria.Results: Most of the studies report benefits of early diagnosis by NS, albeit to variable degrees. The benefits were assessed in terms of better nutritional status and growth, but also in terms of lower overall morbidity rate as compared to subjects diagnosed by symptoms. The main biases of these studies, which partly undermine the validity of their results, are also analyzed.A part of our analysis on nutrition/growth is dedicated to the identification of the most suitable parameters to define malnutrition: in children older than two years the body mass index percentile (BMIp) appears to be the most sensitive and significantly associated with respiratory function. Better nutritional status and satisfactory growth appear to be associated with better lung function and lower risk of death. The relationship between nutritional status and socio-economic status is also of interest.Conclusions: CF registry studies support the outcome of cohort observational studies i.e. that pre-symptomatic early diagnosis is beneficial, especially in terms of nutritional status and growth. Studies on nutrition indicate that good nutritional status is associated with better respiratory function and prognosis. Regarding methods, the need emerged to manage potential biases of this kind of non randomized studies, resorting to suitable statistical techniques, such as matching and stratification and, above all, to multivariate methods able to provide estimates adjusted for the main covariates tested.

Gilead Sciences, Inc. (Nasdaq:GILD) announced that the U.S. Food and Drug Administration (FDA) has granted marketing approval for Cayston(R)(aztreonam for inhalation solution) as a treatment to improve respiratory symptoms in cystic fibrosis (CF) patients with Pseudomonas aeruginosa (P. aeruginosa)…

Aradigm Corporation (OTCBB:ARDM) (the “Company”) announced that it dosed the first patient in the U.S…

Aradigm Corporation (OTCBB:ARDM) (the “Company”) announced that it dosed the first patient in the U.S…

We have a new antibiotic in the arsenal to help fight the life-threatening infections associated with cystic fibrosis.

Yesterday, the FDA approved an important new inhaled antibiotic called Cayston^® (aztreonam for inhalation solution) for the treatment of CF. The drug was made possible by significant support from the Cystic Fibrosis Foundation, including an early $1 million investment to help develop the therapy.

Developed by Gilead Sciences, Inc., Cayston offers a much-needed antibiotic alternative for CF patients who battle recurrent lung infections and develop resistance to existing antibiotics.

Cayston is administered with a new device called Altera that allows patients to take the medicine in less than five minutes, a fraction of the time required for other inhaled antibiotics.

This shortened delivery time reduces the burden on patients, who – on average — have a treatment regimen of three to four hours per day.

Cayston will be available by the end of next week. Patients interested in learning more about Cayston should consult their physicians.

The approval of Cayston demonstrates that our drug development model, fueled by donors and volunteers like you, is working and making a real difference in the lives of people with cystic fibrosis.

To help continue our progress in the fight against CF, please consider making a tax-deductible donation to the CF Foundation today.

Thank you for all you do.

Sincerely,

Robert J. Beall, Ph.D.
President and CEO
Cystic Fibrosis Foundation
800-FIGHT-CF
info@cff.org
www.cff.org

Third Annual Cystic Fibrosis Country Concert

Please Save the Date for the 3rd Annual CF Concert & Motorcycle Ride on Sunday AUG 22,2010 at the American Legion Post 781 Grounds in Mountain Top,Pa (approx 1mile off of the Nuangola Exit on 81 or 3 miles from RT 309)… Concert Starting at 1pm.. General Admission to CONCERT only $5.00 Donation/Children under 12 FREE.. In addition to the GREAT BANDS, the Concert will also feature:

• the National Guard, Nascar
• a Classic Car Show Sponsord by RACE Car club
• Messages by McCain School of Mesages
• Free Petting Zoo for the kids
• Basket Raffles/Tricky Trays
• Good Food at Reasonable Prices
• Great Sponsors & vendors with free giveaways…

Beer,Soda & Water only $1.00…So much to do we can’t fit it on the page CONCERT WILL FEATURE an Up CLose and Personal Concert Sponsored by FROGGY 101

Farmer's Daughter
Crystal Martinez	Big Carl & the Mudpond Boys

…and more to be added in the next few weeks.

MOTOR CYCLE RIDE INFO:

$20 per person per RIDER..

$10 per person for PASS

Both include a delicious PIG ROAST LUNCH at conclusion of ride at the American Legion MT Top… Sign-In starts at 9:30am -11:30 at the KMART parking lot on RT 309 WB.. Donuts, coffee and water provided by Dunkin Donuts WB/& Paiges Family… Ride leaves promptly from KMART parking lot at 11:30 with a 65 mile ride that will go 309 to 437 through Freeland and the Conyingham Valley and the ride concludes at the American Legion Post 781 where your lunch and a great day awaits you… First 200 to arrive at legion will also recieve Thank you bags…

Hope To See You There!

As prenatal and embryonic genetic testing has become more widespread in the U.S., the number of infants with certain inheritable diseases — such as cystic fibrosis, Tay-Sachs and familial dysautonomia — has declined, according to an Associated Press review of research and interviews with genetics experts, the AP/Seattle Times reports…

An inexpensive, fast, accurate DNA test that reveals a person’s risk of developing certain diseases is expected to become a reality, thanks to technology developed at the University of Edinburgh. Scientists have developed a method of pinpointing variations in a person’s genetic code at critical points along the DNA chain. The technique could be used to analyse DNA in a drop of saliva…

In a study that could lead to new therapeutic targets for patients with the cystic fibrosis, a research team from the University of California, San Diego School of Medicine has identified a defective signaling pathway that contributes to disease severity…

As you may know, last week we announced encouraging results from Vertex Pharmaceuticals Incorporated‘s Phase 2a trial of VX-809, one of the first investigational drugs aimed at treating the underlying causes of cystic fibrosis. Learn more about this exciting advancement.The remarkable progress of VX-809 is a reminder of why it is so important to remove barriers that prevent people with CF from participating in clinical trials. Passing the “Improving Access to Clinical Trials Act of 2009” will allow people with CF who are on public benefits to participate in important research without losing their health coverage. We continue to work with Congressional supporters to chart the best path forward for the bill. There are two options to pass the bill:

Option #1: On its own

Non-controversial, bi-partisan and no-cost legislation like the clinical trials bill may be considered under special procedural rules. Thanks to your hard work, 110 members of the House, and eight members of the Senate have signed on as co-sponsors, meeting the requirements to provide the bill the opportunity to advance on its own.

Option #2: As part of a health care reform bill

Despite the shift away from health reform in past weeks, our sponsors – several of whom are key negotiators on health reform – continue to consider it as an option and the best opportunity to move the bill sooner rather than later.

Read more about what H.R. 2866 would do.

The bill has two viable opportunities because you made it clear to your members of Congress that this is a priority for you and should be for them as well.

Ask your members of Congress to keep the “Improving Access to Clinical Trials Act” on their agenda until it passes.

Looking to do more to help fight CF? Sign up for a GREAT STRIDES walk!This spring, take steps to cure cystic fibrosis by signing up for a GREAT STRIDES walk. Join tens of thousands of co-workers, friends and family come together each year as one community for one cause…to help find a cure for CF. In 2009, nearly $35 million was raised to support vital CF programs. Register online today!

Thank you for your support!

Mary B. Dwight
Vice President for Government Affairs
Cystic Fibrosis Foundation
(800) FIGHT CF
publicpolicy@cff.org

MAKE AN IMPACT ON CF

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To learn more about the Cystic Fibrosis Foundation, visit www.cff.org.

As part of a steadfast commitment to finding a cure for cystic fibrosis, BJ’s Restaurants, Inc. donated $521,000 to the Cystic Fibrosis Foundation in 2009 for research, care and education programs. BJ’s has been involved with the Foundation since 1998 and is one of its largest and most loyal corporate supporters…

Solvay Pharmaceuticals, Inc…

This week, we gained important new ground in our work to advance the development of new cystic fibrosis treatments.

VX-809, a potential CF drug developed by Vertex Pharmaceuticals, showed encouraging results in a preliminary Phase 2a clinical trial.  VX-809 is one of the first investigational drugs aimed at treating the underlying causes of CF.

The oral therapy was tested in patient volunteers who have the most common cystic fibrosis mutation, Delta F508.

Results from the trial showed VX-809 was well-tolerated and, in a subset of CF patients, reduced sweat chloride levels ? a key indicator of CF.

VX-809 is the direct result of one of the Foundation?s largest investments in drug development.  This gives us confidence that our innovative approach to science leads to progress.

Learn more about this study.

Without your support, this milestone would simply not be possible.  Your contributions help us build hope for the future and continue to advance vital cystic fibrosis research.

If you have CF, you can also help by participating in a clinical trial. Visit the Foundation?s Web site to learn more about clinical trials and to find out how you can get involved.

Thank you for your commitment to the fight against CF. Together, we are making a difference!

One of the Cystic Fibrosis Foundation’s most active volunteers will carry the Olympic Torch today in Calgary as it makes its way to Vancouver and the Opening Ceremony for the 2010 Olympic Winter Games. Liz Burns, incoming board president for the Foundation’s Georgia Chapter, has helped raise more than $600,000 for CF research, care and education programs…

Johns Hopkins University researchers have created biodegradable nanosized particles that can easily slip through the body’s sticky and viscous mucus secretions to deliver a sustained-release medication cargo…

Abstract: Cystic fibrosis (CF) is a life-shortening disease with significant morbidity. Despite overall improvements in survival, patients with CF experience frequent pulmonary exacerbations and declining lung function, which often accelerates during adolescence. New treatments target steps in the pathogenesis of lung disease, such as the basic defect in CF (CF Transmembrane Conductance Regulator [CFTR]), pulmonary infections, inflammation, and mucociliary clearance. These treatments offer hope but also present challenges to patients, clinicians, and researchers. Comprehensive assessment of efficacy is critical to identify potentially beneficial treatments. Lung function and pulmonary exacerbation are the most commonly used outcome measures in CF clinical research. Other outcome measures under investigation include measures of CFTR function; biomarkers of infection, inflammation, lung injury and repair; and patient-reported outcomes. Molecular diagnostics may help elucidate the complex CF airway microbiome. As new treatments are developed for patients with CF, efforts should be made to balance treatment burden with quality of life. This review highlights emerging treatments, obstacles to optimizing outcomes, and key future directions for research.

Using disinfectants could help superbug bacteria become resistant not only to the disinfectant itself but to antibiotics, even if they have not been exposed to them, according to a new study from Ireland: the findings could be important step in the fight to prevent superbugs spreading in hospitals…

Boston University School of Medicine’s (BUSM) Pulmonary Center has received a two-year $1.4 million grant from the National Heart, Lung, and Blood Institute to derive stem cells and lung progenitors from patients with lung disease…

An increase in the number of screened carriers for cystic fibrosis (CF) was associated with a decrease in the number of children born with CF in northeast Italy, according to a study in the December 16 issue of JAMA. Some studies have suggested that there has been a progressive decrease in the incidence of newborns with CF in some areas…

Researchers have made an important discovery about the secret life of the defective protein that causes cystic fibrosis: while scientists already knew that CFTR protein regulates the acid-alkali balance in cells, what they didn’t know, until this study revealed it, was what turns that ability on and off…

A team of researchers studying the protein that, when defective or absent, causes cystic fibrosis (CF) has made an important discovery about how that protein is normally controlled and under what circumstances it might go awry…

How does a cell manage to identify and degrade the diverse types of defective proteins and thus protect the body against serious diseases? The researchers Sabine C. Horn, Professor Thomas Sommer, Professor Udo Heinemann and Dr. Ernst Jarosch of the Max Delbrück Center for Molecular Medicine (MDC) Berlin-Buch, Germany, have now found a crucial piece in this puzzle…

Gilead Sciences, Inc. (Nasdaq: GILD) announced that the Anti-Infective Drugs Advisory Committee of the U.S. Food and Drug Administration (FDA) has recommended that aztreonam for inhalation solution be approved for the treatment of infections due to Pseudomonas aeruginosa (P. aeruginosa) in patients with cystic fibrosis (CF)…

In an encouraging new development, a team led by Scripps Research Institute scientists has restored partial function to lung cells collected from patients with cystic fibrosis. While there is still much work to be done before the therapy can be tested in humans, the discovery opens the door to a new class of therapies for this and a host of other chronic diseases…

Genta Incorporated (OTCBB: GETA) announced that the Company will supply Ganite® (gallium nitrate injection) for a new clinical trial that will be initiated in patients with cystic fibrosis (CF) who may develop serious infections. Infection is the most common cause of death in CF patients…

Abstract: In patients with cystic fibrosis (CF), respiratory infections with the opportunistic bacterial pathogen Pseudomonas aeruginosa have a major impact on morbidity and mortality. Aminoglycosides, especially tobramycin, have been used successfully to combat these infections. Aminoglycoside penetration of bronchial secretions is poor when the antibiotic is administered intravenously. Nebulization allows direct delivery of the drug to the sites of infection within the airways, while avoiding systemic exposure. Published clinical data show that inhaled tobramycin reduces the bacterial load, improves lung function and reduces the number of hospital admissions. Inhaled tobramycin has been used successfully to eradicate P. aeruginosa in patients with early infection. Maintaining clinical benefits requires chronic tobramycin treatment, and the concept of chronic intermittent inhaled treatment (typically, alternating drug and drug-free periods of 28days) was introduced to minimize the emergence of aminoglycoside resistant P. aeruginosa strains. Other therapeutic advances include the development of different tobramycin formulations and nebulizers that reduce delivery time without compromising efficacy. An optimal treatment regimen for patients with CF with early or intermittent P. aeruginosa infections remains a high priority to maintain long-term lung health.

The Cystic Fibrosis Foundation announced today that CVS/pharmacy and Warner Home Video will partner with the Foundation this holiday season to raise critical funds for cystic fibrosis through the sale of classic family movies. The videos will be sold exclusively at CVS/pharmacy…

Scientists at the University of Pennsylvania School of Medicine discovered that a dietary antioxidant found in such vegetables as broccoli and cauliflower protects cells from damage caused by chemicals generated during the body’s inflammatory response to infection and injury…

Inspire Pharmaceuticals, Inc. (NASDAQ: ISPH) announced today patient enrollment is complete in three of its late-stage clinical trials…

Cystic fibrosis carrier testing should be offered to all couples considering pregnancy and to women in early pregnancy and their partners, according to the authors of a study published in the Medical Journal of Australia…

The American Thoracic Society has released new clinical guidelines for the treatment of exacerbations in cystic fibrosis based on a review of the literature on current clinical practices. “This is the first such comprehensive and evidence-based systematic review of best practices for pulmonary exacerbation of cystic fibrosis,” said Susanna McColley, M.D…

Phase 1/2 clinical trial results with KB001, a Humaneered™, high-affinity antibody fragment under development by KaloBios Pharmaceuticals, Inc. in cystic fibrosis patients showed an acceptable safety profile as well as trends toward reducing tissue inflammation and clearance of the bacterium…

NanoBio Corporation today announced compelling preclinical data for NB-401, a nebulized nanoemulsion-based agent that kills highly drug-resistant strains of bacteria commonly found in cystic fibrosis patients. Currently there are limited treatment options available that effectively address these resistant bacteria…

The University of North Carolina at Chapel Hill has been awarded a five-year, $6.2 million renewal grant to continue its work as part of the National Institutes of Health’s Rare Diseases Clinical Research Network (RDCRN)…

Solvay Pharmaceuticals, Inc. announced that Phase III data published in the Journal of Cystic Fibrosis showed that CREON® (pancrelipase) Delayed-Release Capsules, the most prescribed pancreatic enzyme replacement therapy (PERT) in the United States, significantly improves a key measure of fat absorption in patients with CF who suffer from exocrine pancreatic insufficiency (EPI)…

Francis S. Collins, M.D., Ph.D., the new NIH director who led an international effort to map the human genome and co-discovered the gene responsible for cystic fibrosis, will address more than 3,000 leading scientists and clinicians from around the world at the 23rd annual North American Cystic Fibrosis Conference (NACFC). Held in Minneapolis from Oct…

Nationwide Children’s Hospital has been selected to serve as one of 13 special translational research centers in a network of 77 clinical trial sites that are part of the Therapeutics Development Network (TDN) of the Cystic Fibrosis Foundation. The translational research centers will lead the newest Phase I clinical trials and provide scientific direction to the entire network…

Eric Wedge, manager of the Cleveland Indians, is joining the fight against cystic fibrosis (CF), a fatal genetic disease. Wedge and 17-year old Michaela Mullet will star in a public service announcement (PSA) to be aired during a Cleveland Indians game against the Chicago White Sox on Monday evening, September 28…

Always rushed during the holidays? Get a head start this year with our 65 Roses^® Collection Store! You can shop for gifts and ? at the same time ? support the life-saving mission of the Cystic Fibrosis Foundation.

Check out the collection and order your items today! Or, you can make a tax-deductible donation to the CF Foundation, if you wish.

This special collection includes perennial favorites, such as umbrellas, and our ever-popular 65 Roses^® fleece blankets, and for the first time ever card collections! We’ve also added other new fun and exciting items to the store this year, to give you even more options as you shop.

By selecting gifts for yourself and loved ones, you will show your commitment to helping find a cure for cystic fibrosis. Each of our beautiful items is accompanied by the story of how “65 Roses” got its name and became a moving symbol of the Foundation’s dedication to children and adults with CF.

Thanks for your support!

Sincerely,

Cystic Fibrosis Foundation
6931 Arlington Road
Bethesda, MD  20814
(866) 347-2345
E-mail: DirectMarketing@cff.org
Web: www.65roses.cff.org

P.S. Please forward this message and remember to shop for a cure!

**To ensure delivery of gifts for the holidays, please order by October 10^th.

CARDIOLOGY: Abnormal heartbeats caused by changes in ion channel density Two independent studies have determined how changes in the density of different ion channels in the surface membrane of heart muscle cells can lead to life-threatening abnormal heartbeats.

A cure for cystic fibrosis, HIV-fighting ‘Trojan horses’, new pharmaceuticals from the ocean. Chemical biologists use new and innovative approaches to discover medications of the future. On 24 August, some of the field’s most prominent researchers will attend an international conference in Gothenburg, Sweden. In the field of chemical biology, chemists and biologists cooperate to investigate, and eventually control, the behavior of cells.

A faster and cheaper way to find disease genes in the human genome that is being developed by researchers in the US has passed its initial “proof of concept” test by finding previously unknown gene mutations for Freeman-Sheldon syndrome, a rare Mendelian disorder, in unrelated affected individuals. Researchers from the University of Washington (UW) in Seattle wrote about their work in a paper that appears in the 16 August issue of Nature.

We’ve reached an exciting milestone in the search for a cure.

One of our most promising potential therapies, VX-770, is moving forward in clinical trials.

This compound has the potential to be one of the first drugs ever to treat the basic genetic defect in cystic fibrosis.

VX-770 (developed by Vertex Pharmaceuticals) is already being tested in adults. This week, for the first time, Vertex initiated a clinical trial in patients aged 6 to 11. Once the studies are complete, the FDA will determine if VX-770 is safe, effective and acceptable for approval.

To find out more about VX-770, read the full story and our FAQs. You can also read a very exciting article in today’s Xconomy, quoting CF researcher Bonnie Ramsey and highlighting the work of the Cystic Fibrosis Foundation.

This milestone would not be possible without your support, and we need your help now more than ever to make this drug a reality.

If you have CF, you can help by participating in the clinical trial. Learn more by calling the Vertex clinical trials hotline at (877) 634-8789.

You can also help support this and other lifesaving research by making a donation today.

Together we can make CF stand for “cure found.”