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  • Vertex: The Best Of The Companies Investing In Cystic Fibrosis
    02/21/12
    Imagine assembling a child's first bike. After hours of diligently following the instructions, you send your little one off, excited to try it out. Unbeknownst to you, the instructions contained an error and the bike was not assembled correctly. You watch your little one take a tumble off the malfunctioning bike, thankful for helmets, knee pads and band-aides, and vow to fix the problem. Conventional wisdom says one should find the instructions, notify the company to fix their instructions for good and rebuild the bike. For some reason, however, that is not possible at this time. Read More »
  • Clinical trial shows no cystic fibrosis benefits from clarithromycin
    02/17/12
    Despite hopes that oral clarithromycin could improve lung function in cystic fibrosis (CF) patients thanks to its immunomodulatory properties, a randomised, double-blind trial has failed to show any benefit, backing up results from two smaller studies.Over 60 CF patients were given either a placebo or 500mg of oral clarithromycin twice daily for five months, with a one-month washout. The trial was primarily designed to identify any change in lung function (FEV1 and FVC), although other endpoints included quality of life, weight, number of pulmonary exacerbations, sputum inflammatory content and nasal potential difference. Read More »
  • Drug Bests Cystic-Fibrosis Mutation
    02/8/12
    In a blinded clinical trial, neither the patient nor the clinician should know who is receiving placebo and who the active drug. But during a trial of Kalydeco (ivacaftor), a cystic-fibrosis treatment approved by the US Food and Drug Administration on 31 January, Drucy Borowitz says it was sometimes easy to tell the difference. Read More »
  • FDA approves Vertex's cystic fibrosis drug
    01/31/12
    The Food and Drug Administration said on Tuesday Kalydeco was effective in treating people with the genetic disease, which impairs the lungs and digestive system. Read More »
  • Early Cystic Fibrosis Lung Disease Detected by Bronchoalveolar Lavage and Lung Clearance Index
    01/27/12
    The lung clearance index (LCI) is a sensitive non-invasive marker of early lung disease in young children with cystic fibrosis (CF), according to a new study from Australian researchers. Read More »
  • Liver Transplant Stabilizes Lung Function in Cystic Fibrosis
    01/19/12
    For a select group of patients with cirrhosis due to cystic fibrosis liver disease, liver transplantation can stabilize long-term lung function and nutritional status and reduce the need for intravenous antibiotics, according to a study published online Jan. 6 in the American Journal of Transplantation. Read More »
  • Protein Folding and Disease: The Path from Bench to Bedside
    01/16/12
    Cell biologists are exploring and exploiting protein-folding pathways as a source of drug targets for diseases involving disruption of protein homeostatis, or proteostasis.Such diseases affect proteome expression, protein function, and proteome maintenance. The “American Society for Cell Biology” (ASCB) annual meeting featured presentations that illustrated how managing the protein fold can modify local proteostasis activity to intervene in protein folding, degradation, and function across a range of human diseases including cancer, cystic fibrosis, and neurodegenerative diseases. Read More »
  • Researchers Produce a New Viral Vector with the Potential to Treat Cystic Fibrosis
    01/13/12
    Cystic Fibrosis (CF) is a very common genetic disorder that currently has no cure and few effective treatments. The disease is caused by a defective recessive gene, which was identified in 1989. According to the Cystic Fibrosis Foundation, 1 in 31 Americans carry the CF gene, which is more common in Caucasians of European descent, but can appear in any race or nationality. Read More »
  • Tiny Worm Points to Big Promise
    01/10/12
    Two related studies from Northwestern University offer new strategies for tackling the challenges of preventing and treating diseases of protein folding, such as Alzheimer’s, Parkinson’s and Huntington’s diseases, amyotrophic lateral sclerosis (ALS), cancer, cystic fibrosis and type 2 diabetes.To do its job properly within the cell, a protein first must fold itself into the proper shape. If it doesn’t, trouble can result. More than 300 diseases have at their root proteins that misfold, aggregate and eventually cause cellular dysfunction and death. Read More »
  • Vertex Announces Key 2012 Business Objectives as Company Prepares for Planned Global Launch of KALYDECO in Cystic Fibrosis
    01/9/12
    Preparations for approval and launch of KALYDECO(TM) ongoing; additional studies of KALYDECO planned for mid-2012----Nine new medicines in development for serious diseases; multiple proof-of-concept and later-stage studies planned for 2012- Read More »
  • New hope in cystic fibrosis fight as Scots patients first to test drug
    01/2/12
    Patients in Scotland are set to be the first to test a new drug to treat the devastating effects of cystic fibrosis.Research company NovaBiotics, based in Aberdeen, has been developing a treatment known as Lynovex for the past 18 months to tackle the symptoms of the disease and also keep patients healthier for longer. Read More »
  • Inhaled Dry Powder Mannitol Improves Lung Function in CF
    12/21/11
    Adding the inhaled dry powder mannitol to standard therapy for cystic fibrosis produces sustained improvement in lung function for up to 52 weeks, according to a study published online Dec. 16 in the American Journal of Respiratory and Critical Care Medicine. Read More »
  • Pulmatrix Presents Data on iSPERSE for Inhaled Combination Drug Therapies, Including Triple Drug Combinations
    12/19/11
    LEXINGTON, Mass., Dec 19, 2011 (BUSINESS WIRE) -- Pulmatrix, a clinical stage biotechnology company discovering and developing a new class of therapies for the prevention, treatment and control of respiratory diseases, today announced that its iSPERSE(TM) inhaled drug platform demonstrated multi-drug delivery capability and superiority over conventional lactose blending for an effective therapeutic dose of the active ingredients in Advair(R), salmeterol and fluticasone Read More »
  • Long-term inhaled dry powder mannitol improves lung function in CF
    12/16/11
    Adding inhaled dry powder mannitol to standard therapy for cystic fibrosis produced sustained improvement in lung function for up to 52 weeks, according to a new study. Along with the treatment's efficacy and good safety profile, the convenience and ease of administration of mannitol treatment may improve adherence with therapy in these patients.In the double-blind study, which was supported by Pharmaxis Limited, 318 patients were randomized to treatment with 400 mg bid inhaled mannitol or 50 mg bid inhaled mannitol (control group) for 26 weeks, followed by an additional 26 weeks of open-label active treatment. A 50 mg dose was chosen as the control because it was felt it would not be clinically effective, based on an earlier dose escalation study. Mannitol was given on top of a background of typical concomitant therapy such as recombinant human deoxyribonuclease and inhaled antibiotics. Read More »
  • New Guideline on Newborn Screening for Cystic Fibrosis Issued
    12/14/11
    A new guideline published by the Clinical and Laboratory Standards Institute (CLSI) seeks to clarify the best practices for screening newborns for cystic fibrosis. Read More »