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
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
The primary endpoint of the ... is the leading cause of death for people
fibrosis and this deterioration now averages 1-2% ...
Cystic Fibrosis - Orphan Drug Designation for Innovative Treatment Against Lung Infections by Axentis Pharma AG
infections of the respiratory tract in cystic
fibrosis patients has received
orphan drug ... of the respiratory tract in patients
fibrosis that is delivered directly to the site ... of the respiratory tract in
patients with cystic
The orphan drug designation is ...
Pharmaxis Announces Milestone in Phase III Trial for Cystic Fibrosis
... the effectiveness of
Bronchitol in people with cystic
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.
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
The findings are significant ... are the primary cause of death in persons
fibrosis, and there are simply no effective ... antibiotics serve as frontline therapy
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
caused by a particular genetic ... improvements in the function of the cystic
transmembrane conductance regulator ... and head of the
department of pediatrics and cystic
Fibrosis Center at Hadassah University
Mpex Pharmaceuticals Presents New Data on MP-376 in Cystic Fibrosis
... Investigational eFlow Nebulizer System for use in cystic
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
Second Pivotal Phase 3 Trial In Cystic Fibrosis Begins
... Phase 3
clinical trial evaluating Bronchitol in cystic
The Phase 3 trial is being ... seeks approval to market Bronchitol for cystic
fibrosis in the
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
The first ... we look forward to the results helping to
fibrosis clinical practice in the future."
... European Medicines Agency for Bronchitol in cystic
Bronchitol is designed to hydrate the ...
Bronchitol Cystic Fibrosis Dose Trial Results Positive
... in Lung Function of cystic
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
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
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
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
"We are pleased to see these ... solution for the treatment of cystic
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
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
patients' self-administration of inhaled ... user group. In addition to
potentially helping cystic
fibrosis patients self-manage their condition
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
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
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
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
Cystic Fibrosis Foundation Reports Upward Trend for Key Health Outcomes
... Md., Jan. 28 /PRNewswire-USNewswire/ -- The cystic
Foundation reported today that key ... "Across the country, we are seeing great care for cystic
even better," said Bruce C. ... and quality
About the cystic
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
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
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
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
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
The recall includes the following products:
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
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.
Fibrosis, Burkholderia Cenocepacia and Lung Transplantation
Nobel Prize-Winning Discovery
... potential of this discovery,
using knock-out and knock-in mice to study cystic
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
(CF) who have pulmonary Pseudomonas aeruginosa (P. aeruginosa) ... and has
not yet been determined safe or efficacious in humans.
Today, more than 30,000 people in the United States have CF. CF ...