Cystic Fibrosis Concert Series

An overview of international literature from cystic fibrosis registries: 2. Neonatal screening and nutrition/growth

Mon, 01 Mar 2010 15:00:00 +0000

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.

U.S. Food And Drug Administration Approves Cayston(R) For The Improvement Of Respiratory Symptoms In Cystic Fibrosis Patients

Cystic Fibrosis News From Medical News Today — Sat, 27 Feb 2010 10:00:00 +0000

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 Doses First Patient In Its Second Phase 2b Clinical Trial Of Inhaled Liposomal Ciprofloxacin In Bronchiectasis Patients

Cystic Fibrosis News From Medical News Today — Thu, 25 Feb 2010 10:00:00 +0000

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

FDA approves new drug for CF

Country Concert for Cystic Fibrosis — Wed, 24 Feb 2010 13:08:08 +0000

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

Country Concert for Cystic Fibrosis — Sat, 20 Feb 2010 18:17:53 +0000

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!

Cases Of Some Genetic Diseases Appear To Decline As Prenatal Testing, Embryonic Screenings Increase

Cystic Fibrosis News From Medical News Today — Thu, 18 Feb 2010 12:00:00 +0000

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...

Low-Cost DNA Test To Pinpoint Risk Of Inherited Diseases

Cystic Fibrosis News From Medical News Today — Wed, 17 Feb 2010 11:00:00 +0000

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...

Defective Signaling Pathway Sheds Light On Cystic Fibrosis

Cystic Fibrosis News From Medical News Today — Mon, 15 Feb 2010 10:00:00 +0000

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...

Tell Congress to Keep Clinical Trials Bill on its Agenda

Country Concert for Cystic Fibrosis — Fri, 12 Feb 2010 08:07:30 +0000

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.

What You Can Do Today

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.

Cystic Fibrosis Foundation Receives $521,000 From BJ’s Restaurants, Inc., As Part Of Long-Term Commitment To Fight Life-Threatening Disease

Cystic Fibrosis News From Medical News Today — Mon, 08 Feb 2010 11:00:00 +0000

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...

CREON(R) (pancrelipase) Delayed-Release Capsules Significantly Improves Fat Absorption In Children With Cystic Fibrosis

Cystic Fibrosis News From Medical News Today — Sat, 06 Feb 2010 09:00:00 +0000

Solvay Pharmaceuticals, Inc...

Cystic Fibrosis Foundation Volunteer To Carry Torch For Vancouver 2010 Olympic Winter Games

Cystic Fibrosis News From Medical News Today — Mon, 18 Jan 2010 21:00:00 +0000

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...

Biodegradable Particles Can Bypass Mucus, Release Drugs Over Time

Cystic Fibrosis News From Medical News Today — Wed, 06 Jan 2010 09:00:00 +0000

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...

Measuring and improving respiratory outcomes in cystic fibrosis lung disease: Opportunities and challenges to therapy

Fri, 01 Jan 2010 16:00:00 +0000

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.

Make their dreams come true

Country Concert for Cystic Fibrosis — Thu, 31 Dec 2009 20:25:51 +0000

Disinfectants May Help Superbugs Resist Antibiotics

Cystic Fibrosis News From Medical News Today — Tue, 29 Dec 2009 10:00:00 +0000

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...

$1.4 Million Grant Received By Boston University School Of Medicine

Cystic Fibrosis News From Medical News Today — Fri, 25 Dec 2009 08:00:00 +0000

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...

Carrier Screening Associated With Decrease In Incidence Of Cystic Fibrosis

Cystic Fibrosis News From Medical News Today — Wed, 23 Dec 2009 08:00:00 +0000

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...

Cystic Fibrosis Protein’s Secret Life Revealed, US Study

Cystic Fibrosis News From Medical News Today — Tue, 22 Dec 2009 10:00:00 +0000

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...

Improved Understanding Of The Defective Protein That Causes Cystic Fibrosis

Cystic Fibrosis News From Medical News Today — Fri, 18 Dec 2009 12:00:00 +0000

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...

A Scaffold Regulating Protein Disposal Identified By MDC Researchers

Cystic Fibrosis News From Medical News Today — Mon, 14 Dec 2009 10:00:00 +0000

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 DelbrÃ¼ck Center for Molecular Medicine (MDC) Berlin-Buch, Germany, have now found a crucial piece in this puzzle...

FDA Advisory Committee Supports Use Of Aztreonam For Inhalation Solution For Patients With Cystic Fibrosis

Cystic Fibrosis News From Medical News Today — Sun, 13 Dec 2009 08:00:00 +0000

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)...

Some Function Restored To Cells From Cystic Fibrosis Patients By Scripps Research Team

Cystic Fibrosis News From Medical News Today — Mon, 07 Dec 2009 10:00:00 +0000

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 To Support Initiation Of New Clinical Trial Using Ganite(R) As Treatment For Life-Threatening Infections In Patients With Cystic Fibrosis

Cystic Fibrosis News From Medical News Today — Fri, 04 Dec 2009 09:00:00 +0000

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...

Aminoglycoside therapy against Pseudomonas aeruginosa in cystic fibrosis: A review

Felix Ratjen, Florian Brockhaus, Gerhild Angyalosi — Tue, 01 Dec 2009 20:00:00 +0000

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 To Benefit From Holiday Film Promotion

Cystic Fibrosis News From Medical News Today — Wed, 25 Nov 2009 12:00:00 +0000

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...

Exome-Sequencing Might Help Identify Genetic Cause Of Thousands Of Disorders

Cystic Fibrosis News From Medical News Today — Fri, 20 Nov 2009 13:00:00 +0000

For the first time, scientists have successfully used a method called exome sequencing to quickly discover a previously unknown gene responsible for a mendelian disorder. Mendelian disorders, such as cystic fibrosis and sickle cell disease, are the result of one or more mutations in a single gene, typically a gene that makes a protein...

Antioxidant Found In Vegetables Has Implications For Treating Cystic Fibrosis

Cystic Fibrosis News From Medical News Today — Wed, 18 Nov 2009 09:00:00 +0000

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 Completes Patient Enrollment In Three Late-Stage Clinical Trials In Cystic Fibrosis, Dry Eye And Blepharitis

Cystic Fibrosis News From Medical News Today — Fri, 06 Nov 2009 04:00:00 +0000

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

Call For Cystic Fibrosis Carrier Testing To Be Offered To All Couples Considering Pregnancy, Australia

Cystic Fibrosis News From Medical News Today — Tue, 03 Nov 2009 08:00:00 +0000

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...