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Cystic Fibrosis - Liposomal Tobramycin Receives Second Orphan Drug Designation Within Weeks

... for infections of the respiratory tract in cystic fibrosis patients has received a second orphan ... pathogens that can cause lethal infections in cystic fibrosis patients. For Axentis Pharma AG of ... of the respiratory tract in patients with cystic fibrosis. Only three months ago, the FDA granted ...

Transave Receives $2.2 Million Cystic Fibrosis Foundation Award for Development of ARIKACE(TM)

... MONMOUTH JUNCTION, N.J., June 25 /PRNewswire/ -- cystic Fibrosis Foundation Therapeutics Inc., the nonprofit affiliate of the cystic Fibrosis Foundation, has provided a $2.2 million ... aims to treat Pseudomonas lung infections in cystic fibrosis (CF) and non-CF bronchiectasis patients. ...

New Legislation Seeks to Boost Participation in Clinical Trials for Rare Diseases

... populations. This is particularly true for cystic fibrosis (CF). More than 30 promising CF drugs ... allows more people with rare diseases - including cystic fibrosis - to participate in clinical trials," ... Robert J. Beall, Ph.D., president and CEO of the cystic Fibrosis Foundation. "Cystic fibrosis is a ...

Pharmaxis Announces Positive Results of Phase 3 Cystic Fibrosis Trial

... Phase III trial of Bronchitol(TM) in people with cystic fibrosis. The study represents one of the largest clinical trials conducted in cystic fibrosis. The primary endpoint of the ... is the leading cause of death for people with cystic fibrosis and this deterioration now averages 1-2% ...

Cystic Fibrosis - Orphan Drug Designation for Innovative Treatment Against Lung Infections by Axentis Pharma AG

... for infections of the respiratory tract in cystic fibrosis patients has received orphan drug ... of the respiratory tract in patients with cystic fibrosis that is delivered directly to the site ... of the respiratory tract in patients with cystic fibrosis. The orphan drug designation is ...

Pharmaxis Announces Milestone in Phase III Trial for Cystic Fibrosis

... the effectiveness of Bronchitol in people with cystic fibrosis. The final subject has now ... and involved 325 randomized subjects with cystic fibrosis. The trial was a double blind, placebo ... a marketing approval for Bronchitol for treating cystic fibrosis in Europe and elsewhere. The ...

PTC Therapeutics Completes Enrollment of Pivotal Trial in Muscular Dystrophy

... of ataluren in patients with nonsense mutation cystic fibrosis (nmCF) in 2009. The United States ... orphan drug status for the treatment of DMD and cystic fibrosis due to nonsense mutations. The FDA has ... National Center for Research Resources and the cystic Fibrosis Foundation Therapeutics Inc. (the ...

Nanoemulsion Kills Highly Resistant Bacteria Found in Patients With Cystic Fibrosis

... chronic illness and death among individuals with cystic fibrosis. The findings are significant ... are the primary cause of death in persons with cystic fibrosis, and there are simply no effective ... antibiotics serve as frontline therapy against cystic fibrosis-related bacterial infections. But ...

PTC Announces Data Showing That PTC124 Causes Statistically Significant Improvements in Chloride Channel Function in Cystic Fibrosis Patients

... the chloride channel function of children with cystic fibrosis (CF) caused by a particular genetic ... improvements in the function of the cystic fibrosis transmembrane conductance regulator ... and head of the department of pediatrics and cystic Fibrosis Center at Hadassah University Hospital. ...

Mpex Pharmaceuticals Presents New Data on MP-376 in Cystic Fibrosis

... Investigational eFlow Nebulizer System for use in cystic fibrosis (CF) and chronic obstructive pulmonary ... results are being presented at the North American cystic Fibrosis Conference (NACFC) and ICAAC/IDSA ... of bacterial exacerbations in patients with cystic fibrosis and COPD. The company has also built ...

PTC Therapeutics Announces Data Showing Six-Minute Walk Test Consistently Measures Ambulatory Function in Patients With Duchenne Muscular Dystrophy

... and the European Commission for the treatment of cystic fibrosis and DMD due to nonsense mutations. The ... Center for Research Resources and notably, the cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the cystic Fibrosis Foundation), which recently ...

Cystic Fibrosis Axentis Pharma Initiates Clinical Trial for Lung Infections

... the treatment of severe pulmonary infection in cystic fibrosis patients. The new formulation allows ... drug for the treatment of pulmonary infection in cystic fibrosis patients. The product ARB-CF0223 ­ also ... of the respiratory tract in patients with cystic fibrosis. It can be used in lower doses and also ...

Second Pivotal Phase 3 Trial In Cystic Fibrosis Begins

... Phase 3 clinical trial evaluating Bronchitol in cystic fibrosis sufferers. The Phase 3 trial is being ... seeks approval to market Bronchitol for cystic fibrosis in the United States. Pharmaxis Chief ... with the FDA and with assistance from the U.S. cystic Fibrosis Foundation. This trial follows the ...

Bronchitol Phase 3 Cystic Fibrosis Trial Completes Recruitment

... trial of Bronchitol for the treatment of cystic fibrosis has completed enrolment. The first ... we look forward to the results helping to shape cystic fibrosis clinical practice in the future." ... European Medicines Agency for Bronchitol in cystic fibrosis. Bronchitol is designed to hydrate the ...

Bronchitol Cystic Fibrosis Dose Trial Results Positive

... in Lung Function of cystic Fibrosis Patients SYDNEY, Australia, Aug. 11 ... mg and 40 mg of Bronchitol in 48 patients with cystic fibrosis at 12 centres across Canada and ... flow) and the respiratory domain of the cystic fibrosis quality of life questionnaire (CFQR). ...

PTC Therapeutics Announces $25 Million Award From Cystic Fibrosis Foundation Therapeutics for Development of PTC124

... Therapy to Address an Underlying Cause of cystic Fibrosis - SOUTH PLAINFIELD, N.J. and BETHESDA, ... /PRNewswire/ -- PTC Therapeutics, Inc. (PTC) and cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), the nonprofit affiliate of the cystic Fibrosis Foundation, today announced the ...

The Ocean Becomes a Pipeline To A Cure for Cystic Fibrosis

... the lifespan and quality of life for people with cystic fibrosis (CF), a fatal genetic disease ... has led the Southern California Chapter of the cystic Fibrosis Foundation to form a "PIPELINE TO A ... bond between the sport of surfing and those with cystic fibrosis. On July 19, 2008, ...

Mpex Initiates a Phase 2 Clinical Trial of Aerosol MP-376 in the Treatment of Chronic Bacterial Infections in Cystic Fibrosis Patients

... aeruginosa and other bacteria in patients with cystic fibrosis. MP-376 is Mpex's proprietary aerosol ... we hope to initiate in 2009." Patients with cystic fibrosis (CF) suffer from chronic infections ... of bacterial exacerbations in patients with cystic fibrosis and severe chronic bronchitis. The ...

Transave Announces Positive Phase II Results for Once-Daily Arikace(TM) in the Treatment of Cystic Fibrosis Patients Who Have Pseudomonas Lung Infections

... Data Presented at the European cystic Fibrosis Society Conference MONMOUTH JUNCTION, ... is being developed for the treatment of cystic fibrosis (CF) patients who have lung infections ... Results were presented today at the 31st European cystic Fibrosis Society (ECFS) Conference in Prague, ...

Inspire's Phase 3 CF Trial, Using PARI's Nebulizer & Compressor, Shows Positive Top-Line Results

... and PRONEB Ultra compressor for the treatment of cystic fibrosis (CF). "We are pleased to see these ... solution for the treatment of cystic fibrosis. Additional information is available at ... waste. Denufosol for the treatment of cystic fibrosis has been granted Fast Track designation ...

PTC Therapeutics Announces Data from Additional Clinical Studies of PTC124 in Cystic Fibrosis Confirming Activity

... - Data Presented at the 31st European cystic Fibrosis Conference - SOUTH PLAINFIELD, N.J. ... promising new data from two studies of PTC124 in cystic fibrosis (CF). Results from an Israeli Phase ... improvements in the function of the cystic fibrosis transmembrane conductance regulator ...

Discovering How Video Games Can Motivate Healthy Behaviors: More than $2 Million Awarded to 12 Research Teams Across U.S.

... - Breath Biofeedback Video Game for Children with cystic Fibrosis will explore whether a breath biofeedback video game can improve cystic fibrosis patients' self-administration of inhaled ... user group. In addition to potentially helping cystic fibrosis patients self-manage their condition and ...

PTC Therapeutics Announces Data Supporting Cough Frequency as a New Outcome Measure in Evaluating Treatments for Cystic Fibrosis

... - Findings Show cystic Fibrosis Patients Cough More Than 600 Times Per ... M.D., head of the Department of Pediatrics and cystic Fibrosis Center, Hadassah University ... adults and children. Patients with CF lack the cystic fibrosis transmembrane conductance regulator ...

Cystic Fibrosis Foundation Announces Positive Early Results for Phase 2 Clinical Trial of VX-770 - an Oral Compound to Treat CF

... BETHESDA, Md., March 27 /PRNewswire/ -- The cystic Fibrosis Foundation announced today that VX-770, ... improvements in several key indicators of cystic fibrosis, including lung function, nasal ... sweat chloride is a key clinical indicator of cystic fibrosis. The "sweat test" is the traditional ...

PARI Nebulizer & Compressor Used Exclusively in Inspire's Phase 3 CF Trial

... second Phase 3 clinical trial for a potential new cystic fibrosis treatment. PARI reusable nebulizers ... of the disease. Denufosol for the treatment of cystic fibrosis has been granted Fast Track designation ... patients with asthma, chronic lung disease, and cystic fibrosis. PARI's worldwide vision is to improve ...

PTC Therapeutics Announces Publication of Preclinical Data in PNAS

... and Restores Protein Function in cystic Fibrosis Model SOUTH PLAINFIELD, N.J., Feb. 4 ... mutations, was active in a preclinical model of cystic fibrosis (CF). These results support and add to ... cases of most inherited diseases, such as cystic fibrosis (10%) and Hurler's syndrome (70%). ...

Cystic Fibrosis Foundation Reports Upward Trend for Key Health Outcomes

... Md., Jan. 28 /PRNewswire-USNewswire/ -- The cystic Fibrosis Foundation reported today that key ... "Across the country, we are seeing great care for cystic fibrosis get even better," said Bruce C. ... and quality improvement purposes. About the cystic Fibrosis Foundation ...

Pediatric Pulmonary Expert Comments on Study of Lung Transplants for Cystic Fibrosis

... a study of lung transplantation for children with cystic fibrosis, a leading pediatric pulmonologist calls ... than ever in referring children with cystic fibrosis for lung transplantation, with careful ... of the child's illness," said Allen. He said that cystic fibrosis centers must provide appropriate social ...

PTC Therapeutics Announces Additional Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy

... Neurology Society meeting and North American cystic Fibrosis Conference, further support our belief ... the initiation of longer-term studies in DMD and cystic fibrosis (CF) as well as additional proof of ... of PTC124 in nonsense-mutation-mediated cystic fibrosis (CF) and Duchenne muscular dystrophy ...

PTC Therapeutics Announces Pharmacokinetic and Safety Results from Phase 2 Study of PTC124 in Duchenne Muscular Dystrophy

... subjects, including patients with both DMD and cystic fibrosis. The safety profile has consistently ... of PTC124 in nonsense-mutation-mediated cystic fibrosis (CF) and Duchenne muscular dystrophy ... from the Muscular Dystrophy Association (MDA), cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), ...

Promising Cystic Fibrosis Compound on Track for Development

... Therapy Made Possible by Early Investment by cystic Fibrosis ... drug in treating the basic defect in cystic fibrosis. This product was identified as part of a $1.7 million award to Parion from cystic Fibrosis Foundation Therapeutics, Inc. -- the CF ...

TriLink BioTechnologies, Inc. Announces Supply Agreement With Osmetech Molecular Diagnostics

... Diagnostics to supply reagents for Osmetech's recently launched eSensor(R) cystic Fibrosis Carrier Detection (CFCD) System and the eSensor(R) XT-8 System ... 4800 platform and the eSensor(R) XT-8 platform, presently available for cystic Fibrosis Carrier Detection and Warfarin Sensitivity testing, respectively. ...

Mpex Pharmaceuticals Announces Initiation of a Phase 2 Clinical Trial of Aerosol MP-376 in COPD

... trials for potential use as a maintenance treatment in patients with cystic fibrosis. About COPD There are more than 9 million patients with COPD ... therapy for the prevention of bacterial exacerbations in patients with cystic fibrosis and COPD. The company has also built a discovery and development ...

Pharmaxis Concludes Special Protocol Assessment with FDA for Bronchitol Phase 3 Trial

... problem. Our bronchiectasis program follows closely behind our work in cystic fibrosis where a phase 3 clinical trial is expected to soon close ... as a treatment to improve mucus clearance in the lungs of patients with cystic fibrosis, bronchiectasis and other acute and chronic pulmonary conditions. ...

Hydrox Inc., Cardinal Health Issue Nationwide Recall of Alcohol-Free Mouthwash For Hospitals

... such as weakened immune systems or chronic lung diseases, particularly cystic fibrosis, may be more susceptible to infections with B. cepacia. B cepacia ... at all to serious respiratory infections, especially in patients with cystic fibrosis. The recall includes the following products: Description ...

PTC Therapeutics Announces Initiation of Phase 2b Registration-Directed Clinical Trial of PTC124 in Duchenne/Becker Muscular Dystrophy

... PTC. The trial builds on the results we have achieved to date in DMD and cystic fibrosis (CF) and constitutes a major step forward in establishing the ... trials in nonsense-mutation-mediated Duchenne muscular dystrophy (DMD) and cystic fibrosis (CF). It is estimated that 13% of the cases of DMD and 10% of ...

Genzyme Genetics Announces Launch of Carrier Testing and Prenatal Diagnosis for Spinal Muscular Atrophy

... breathing, swallowing, head and neck control, walking and crawling. After cystic fibrosis, which is routinely screened for in the general population, SMA ... and prenatal diagnosis, Genzyme offers a broad carrier test menu including cystic fibrosis testing with 97 mutations, fragile X testing and 11 carrier tests ...

Lung Transplants in Cystic Fibrosis Patients With Life-Threatening Bacteria Sparks Debate at ISHLT Meeting

... Satellite Symposium 3: The Challenges of Lung Transplantation in cystic Fibrosis (CF) at the International Society of Heart and Lung ... and Redha R. Souilemas, MD, European Georges Pompidou Hospital, Paris. cystic Fibrosis, Burkholderia Cenocepacia and Lung Transplantation Patients ...

Nobel Prize-Winning Discovery

... potential of this discovery, using knock-out and knock-in mice to study cystic fibrosis. This disorder devastates multiple organ systems in one out ... genes, among other functions. Eventually, this could mean knocking out cystic fibrosis and other inherited disorders. Without Evans, Smithies and ...

PARI's eFlow Included in Gilead's NDA Submission for Aztreonam Lysine for Inhalation

... by eFlow, is an investigational therapy in development for people with cystic fibrosis (CF) who have pulmonary Pseudomonas aeruginosa (P. aeruginosa) ... and has not yet been determined safe or efficacious in humans. About cystic Fibrosis Today, more than 30,000 people in the United States have CF. CF ...
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