Cystic Fibrosis News and Research

Latest Cystic Fibrosis News and Research

New investigational drug is well tolerated in children with nmCF

PTC Therapeutics, Inc. today announced the publication of data from a Phase 2a clinical trial of ataluren in children with nonsense mutation cystic fibrosis (nmCF) in the American Journal of Respiratory and Critical Care Medicine.

15 Nov 2010

Researchers find link between genetic defect and sticky mucus in cystic fibrosis

An imbalance of salt and water in patients with cystic fibrosis makes their lungs clog up with sticky mucus that is prone to infection. The cause of the offending imbalance is a well-known genetic error, one that blocks the molecular expressway for tiny chloride ions to move across the surface of the lungs.

15 Nov 2010

Targeted Genetics enters definitive agreements to acquire Biocontrol

Targeted Genetics Corporation, today announced that it has entered into definitive agreements related to the acquisition of Biocontrol Limited, or Biocontrol.

12 Nov 2010

Aradigm reports $1.3M net income for third quarter 2010 vs. $3.0M net loss for third quarter 2009

Aradigm Corporation today announced financial results for the third quarter and nine months ended September 30, 2010.

11 Nov 2010

Pharmaxis to present ARIDOL Bronchial Challenge Test Kit at 2010 ACAAI

Pharmaxis, a global specialty pharmaceutical company focused on therapeutic products for respiratory and immune disorders, will present ARIDOL™ (mannitol inhalation powder) Bronchial Challenge Test Kit, at the 2010 Annual Meeting of the American College of Allergy, Asthma and Immunology (ACAAI), November 11-16 in Phoenix, AZ.

11 Nov 2010

NJIT expert to discuss history of sickle cell disease at NIH symposium

The history of sickle cell disease in the 20th century and its link to race is the focus of a Nov. 16, 2010 talk at a national symposium by NJIT Associate Professor Stephen Pemberton, PhD, an expert on the history of diseases. Pemberton will speak and join a panel discussion at the National Institutes of Health (NIH), Bethesda, 100th anniversary symposium commemorating the discovery of sickle cell disease by Chicago physician James B. Herrick.

11 Nov 2010

Marker for aggressive form of idiopathic pulmonary fibrosis discovered

A diagnosis of idiopathic pulmonary fibrosis is not much better than a death sentence, given a survival rate averaging 4 to 6 years as the disease robs its victim of the ability to breathe.

11 Nov 2010

Vertex to enroll additional treatment arm in telaprevir/VX-222 Phase 2 clinical trial for HCV

Vertex Pharmaceuticals Incorporated today announced plans to enroll an additional treatment arm as part of its ongoing Phase 2 clinical trial evaluating 12-week regimens of Vertex's lead investigational hepatitis C virus (HCV) protease inhibitor, telaprevir, in combination with its lead investigational HCV polymerase inhibitor, VX-222.

10 Nov 2010

Low levels of growth hormone may contribute to growth defects in cystic fibrosis

A new study using a pig model of cystic fibrosis (CF) suggests that low levels of a growth promoting hormone at or before birth may contribute to growth defects in patients with CF.

10 Nov 2010

Discovery Labs reports net operating loss of $6.2 million for third quarter 2010

Discovery Laboratories, Inc., a biotechnology company developing its novel, synthetic, peptide-containing surfactant and related aerosolization technologies as first in class therapies for severe respiratory diseases, today provides an update on key pipeline and business initiatives and a financial update for the third quarter ended September 30, 2010.

9 Nov 2010

Synergy receives $244,479 grant under QTDP program to develop plecanatide for gastrointestinal disorders

Synergy Pharmaceuticals, Inc. a developer of new drugs to treat gastrointestinal disorders (GI) and diseases, announced today that it has been awarded a $244,479 grant under the Qualifying Therapeutic Discovery Project (QTDP) program to aid the Company in its development of plecanatide currently in clinical development to treat gastrointestinal disorders.

8 Nov 2010

Inspire third quarter net loss decreases to $7.6 million

Inspire Pharmaceuticals, Inc. announced today financial results for the third quarter ended September 30, 2010, reporting a net loss of $7.6 million or ($0.09) per share, an improvement from the third quarter of 2009 during which the Company incurred a net loss of $8.5 million or ($0.12) per share.

4 Nov 2010

DARA awarded $0.5M grant under Patient Protection and Affordable Care Act for advancing KRN5500 and DB959

DARA BioSciences announced today grant approvals by the Internal Revenue Service under the Patient Protection and Affordable Care Act of 2010 for KRN5500 and DB959 in the amount of approximately $500,000 for the calendar year 2009.

3 Nov 2010

FDA grants orphan drug designation to Discovery's KL4 surfactant for CF treatment

Discovery Laboratories, Inc. today announced that the Office of Orphan Products Development of the United States Food and Drug Administration (FDA) has granted orphan drug designation to Discovery's KL4 surfactant for the treatment of cystic fibrosis (CF).

1 Nov 2010

Vertex reports superior SVR rates with telaprevir-based combination therapy in Phase 3 studies for hepatitis C

Vertex Pharmaceuticals Incorporated today announced new data from its Phase 3 studies of people with genotype 1 chronic hepatitis C who have not been treated previously. In these studies, the majority of people achieved superior sustained viral response (SVR or viral cure) rates with a telaprevir-based combination regimen, compared to current therapies, regardless of race/ethnicity or stage of liver fibrosis (factors known to limit response to current hepatitis C treatments).

1 Nov 2010

Nanomedicine Center for Nucleoprotein Machines receives $16.1 million NIH award

The Georgia Tech-led Nanomedicine Center for Nucleoprotein Machines has received an award of $16.1 million for five years as part of its renewal by the National Institutes of Health (NIH). The eight-institution research team plans to pursue development of a clinically viable gene correction technology for single-gene disorders and demonstrate the technology's efficacy with sickle cell disease.

29 Oct 2010

New iPSC cell lines could help in treating patients with lung diseases

A team of researchers from Boston University's Center for Regenerative Medicine and the Pulmonary Center have generated 100 new lines of human induced pluripotent stem cells (iPSC) from individuals with lung diseases, including cystic fibrosis and emphysema. The new stem cell lines could possibly lead to new treatments for these debilitating diseases.

29 Oct 2010

Vertex initiates OPTIMIZE Phase 3b study in patients infected with genotype 1 HCV

Vertex Pharmaceuticals Incorporated today announced the initiation of a Phase 3b study called OPTIMIZE that will evaluate twice-daily (BID) dosing of a telaprevir-based combination regimen in people chronically infected with genotype 1 hepatitis C virus (HCV) who have not been treated previously. This is the first Phase 3 study to evaluate twice-daily dosing of a protease inhibitor for the treatment of hepatitis C. OPTIMIZE will not include a control arm of pegylated-interferon and ribavirin alone.

26 Oct 2010

DARA BioSciences to raise $1.4 million in registered direct offering

DARA BioSciences, Inc., a development-stage pharmaceutical company, today announced that it has entered into a definitive agreement with several institutional investors to sell 612,667 units, with each unit consisting of one of the Company's common shares and 0.50 warrants to purchase one share of common stock, for gross proceeds of approximately $1.4 million, before deducting placement agent fees and estimated offering expenses, in a "registered direct" offering.

25 Oct 2010

Pharmaxis announces combined results of Bronchitol Phase III trial for cystic fibrosis

Pharmaceutical company Pharmaxis today announced significant results of pooled data from its two large scale six month Phase III trials of Bronchitol (inhaled mannitol) in people with cystic fibrosis.

25 Oct 2010

Cystic Fibrosis News and Research

Latest Cystic Fibrosis News and Research

New investigational drug is well tolerated in children with nmCF

Researchers find link between genetic defect and sticky mucus in cystic fibrosis

Targeted Genetics enters definitive agreements to acquire Biocontrol

Aradigm reports $1.3M net income for third quarter 2010 vs. $3.0M net loss for third quarter 2009

Pharmaxis to present ARIDOL Bronchial Challenge Test Kit at 2010 ACAAI

NJIT expert to discuss history of sickle cell disease at NIH symposium

Marker for aggressive form of idiopathic pulmonary fibrosis discovered

Vertex to enroll additional treatment arm in telaprevir/VX-222 Phase 2 clinical trial for HCV

Low levels of growth hormone may contribute to growth defects in cystic fibrosis

Discovery Labs reports net operating loss of $6.2 million for third quarter 2010

Synergy receives $244,479 grant under QTDP program to develop plecanatide for gastrointestinal disorders

Inspire third quarter net loss decreases to $7.6 million

DARA awarded $0.5M grant under Patient Protection and Affordable Care Act for advancing KRN5500 and DB959

FDA grants orphan drug designation to Discovery's KL4 surfactant for CF treatment

Vertex reports superior SVR rates with telaprevir-based combination therapy in Phase 3 studies for hepatitis C

Nanomedicine Center for Nucleoprotein Machines receives $16.1 million NIH award

New iPSC cell lines could help in treating patients with lung diseases

Vertex initiates OPTIMIZE Phase 3b study in patients infected with genotype 1 HCV

DARA BioSciences to raise $1.4 million in registered direct offering

Pharmaxis announces combined results of Bronchitol Phase III trial for cystic fibrosis

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Cystic Fibrosis News and Research

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