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Teen Explains What Life is Like in a Coma
Teen with cystic fibrosis describes life in a coma. (Source: ABC News: Health)
Source: ABC News: Health - July 16, 2015 Category: Consumer Health News Tags: Health Source Type: news

Scientists grow 'mini bile ducts' to help discovery of new drugs
An experimental cystic fibrosis drug has been shown to prevent the disease’s damage to the liver, thanks to a world-first where scientists grew mini bile ducts in the lab.  (Source: Medical Research Council General News)
Source: Medical Research Council General News - July 14, 2015 Category: Research Source Type: news

Stem cells might heal damaged lungs
Collectively, such diseases of the airways as emphysema, bronchitis, asthma and cystic fibrosis are the second leading cause of death worldwide. More than 35 million Americans alone suffer from chronic respiratory disease. Scientists have now proposed a new direction that could, in the future, lead to the development of a method for alleviating some of their suffering. The study's findings show how it might be possible to use embryonic stem cells to repair damaged lung tissue. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - July 14, 2015 Category: Science Source Type: news

Scientists create 'mini bile ducts' to discover new drugs that could prevent the need for liver transplantation
An experimental cystic fibrosis drug has been shown to prevent the disease’s damage to the liver, thanks to a world-first where scientists grew mini bile ducts in the lab.  (Source: Medical Research Council General News)
Source: Medical Research Council General News - July 13, 2015 Category: Research Source Type: news

How family friend recognised the signs of cystic fibrosis
Cystic fibrosis sufferer Charlie York, 11, from North Yorkshire, has survived major surgery to replace his failing lungs. He is pictured with his mother Sasha. (Source: the Mail online | Health)
Source: the Mail online | Health - July 13, 2015 Category: Consumer Health News Source Type: news

Setting the Scene of Life
"All the world's a stage, And all the men and women are merely players." - Shakespeare Time: Present Setting: An operating room, doctor's office Characters: Ashley (me), Mark, Radiologist, Pulmonologist, Nurses, Receptionists Call it a coping mechanism, a way for me to deal with my reality, or simply a means to save myself from exposing a deep set pain and fear by being solely focused on the present scene. Doing all in hopes of avoiding any thoughts of the next act. Of course, the next act is filled with incredible opportunity and possibility, but it's also filled with pages of uncertainty. There's an unsettle...
Source: Healthy Living - The Huffington Post - July 13, 2015 Category: Consumer Health News Source Type: news

Scientists grow 'mini bile ducts' to help discovery of new drugs
An experimental cystic fibrosis drug has been shown to prevent the disease’s damage to the liver, thanks to a world-first where scientists grew mini bile ducts in the lab.  (Source: Medical Research Council General News)
Source: Medical Research Council General News - July 13, 2015 Category: Research Source Type: news

Orkambi (lumacaftor/ivacaftor) for the Treatment of Cystic Fibrosis (CF)
Orkambi (lumacaftor/ivacaftor) is the first approved medicine to treat the underlying cause of cystic fibrosis (CF), a rare, life-threatening genetic disease. (Source: Drug Development Technology)
Source: Drug Development Technology - July 8, 2015 Category: Pharmaceuticals Source Type: news

Celtaxsys receives FDA approval to begin acebilustat’s trial of CF
Clinical-stage pharmaceutical development firm Celtaxsys has received approval from the US Food and Drug Administration (FDA) to start a Phase II clinical trial of its drug candidate, acebilustat (CTX-4430), in adult cystic fibrosis (CF) patients in … (Source: Drug Development Technology)
Source: Drug Development Technology - July 6, 2015 Category: Pharmaceuticals Source Type: news

Cystic Fibrosis News: Nonviral Gene Therapy Shows Promise, Drug Combo Approved (FREE)
By Amy Orciari Herman Edited by Susan Sadoughi, MD, and André Sofair, MD, MPH Cystic fibrosis received much attention late last week: a nonviral gene therapy conferred significant (although modest) benefits to lung function in an early trial, and the FDA approved a new combination drug therapy for the condition.In the … (Source: Physician's First Watch current issue)
Source: Physician's First Watch current issue - July 6, 2015 Category: Primary Care Source Type: news

Encouraging results for gene therapy trial for CF
A therapy that replaces the faulty gene responsible for cystic fibrosis in patients’ lungs has produced encouraging results in a major UK trial. (Source: Medical Research Council General News)
Source: Medical Research Council General News - July 4, 2015 Category: Research Source Type: news

Medical News Today: Cystic fibrosis gene therapy trial offers hope of treatment
A trial to test the safety and effectiveness of a therapy that replaces a faulty gene in the lungs of patients with cystic fibrosis has shown encouraging results. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - July 3, 2015 Category: Consumer Health News Tags: Cystic Fibrosis Source Type: news

VIDEO: Gene therapy for cystic fibrosis
Trials suggest a new gene therapy treatment for cystic fibrosis patients could be available within five years. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - July 3, 2015 Category: Consumer Health News Source Type: news

Cystic Fibrosis More Deadly for Hispanics, Data SuggestCystic Fibrosis More Deadly for Hispanics, Data Suggest
Researchers found worse lung function in Hispanic children with CF by age 6 years and a nearly threefold greater risk of death. Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 3, 2015 Category: Consumer Health News Tags: Pulmonary Medicine News Source Type: news

Gene therapy breakthrough for cystic fibrosis
ConclusionThis RCT showed that a new non-viral-based gene therapy for cystic fibrosis was able to produce “modest” benefits in lung function compared to a placebo. The treatments were given once a month for a year. The study had many strengths, including its double-blind randomised design, recruiting adequate numbers to demonstrate real differences between groups, and using pre-specified outcomes and sub-analysis. This means we can be confident in the reliability of the findings presented. Although the findings of this study are encouraging, there are always limitations. These include: This study was relativel...
Source: NHS News Feed - July 3, 2015 Category: Consumer Health News Tags: Genetics/stem cells Heart/lungs Source Type: news

Encouraging results for gene therapy trial for CF
A therapy that replaces the faulty gene responsible for cystic fibrosis in patients’ lungs has produced encouraging results in a major UK trial. (Source: Medical Research Council General News)
Source: Medical Research Council General News - July 3, 2015 Category: Research Source Type: news

First trial of gene therapy for cystic fibrosis to show beneficial effect on lung function
For the first time gene therapy for cystic fibrosis has shown a significant benefit in lung function compared with placebo, in a phase 2 randomized trial. The technique replaces the defective gene response for cystic fibrosis by using inhaled molecules of DNA to deliver a normal working copy of the gene to lung cells. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - July 3, 2015 Category: Science Source Type: news

Vitamin E dose recommendations may be too high for Cystic Fibrosis patients, finds new research
ConclusionsOur results suggest that the CF-specific vitamin E recommendations are higher than necessary to prevent deficiencies. Therefore, vitamin E dosages of 50% of the recommendations could be used, at least initially. We found no evidence that higher serum α-tocopherol levels had protective effects on pulmonary function in paediatric patients with CF. Moreover, the finding of higher serum α-tocopherol levels in patients with lower pulmonary function casts doubt on the hypothesis that vitamin E has therapeutic benefits. Janna W. Woestenenk View the full paper here   vitamin eCystic Fibrosisα-toco...
Source: The Nutrition Society - July 3, 2015 Category: Nutrition Authors: Cassandra Ellis Source Type: news

Gene therapy treatment for cystic fibrosis may be possible by 2020, scientists say
Although results of first trial were ‘modest and variable’, second bigger trial aims to combine gene therapy with other treatments for longer term benefitsA treatment to help those with cystic fibrosis may be available within five years, say scientists who who have been working for decades to develop a gene therapy for the disease.The results of a year-long trial showed only a small and variable improvement in the lung capacity of those children and adults who took part, but scientists now believe they have proved gene therapy is possible in treating cystic fibrosis. With more work, they say that by the end of ...
Source: Guardian Unlimited Science - July 3, 2015 Category: Science Authors: Sarah Boseley Health editor Tags: Medical research Science Health UK news Imperial College London University of Oxford University of Edinburgh Source Type: news

Gene therapy shows promise in cystic fibrosis research
‘Patients who received the treatment showed a benefit in tests of lung function’ (Source: The Irish Times - Health)
Source: The Irish Times - Health - July 3, 2015 Category: Consumer Health News Source Type: news

Gene therapy for cystic fibrosis shows encouraging trial results
A therapy that replaces the faulty gene responsible for cystic fibrosis in patients’ lungs has produced encouraging results in a major UK trial. Cystic fibrosis (CF) is the commonest lethal inherited disease in the UK, affecting around 10,000 people nationally and over 90,000 worldwide. Patients’ lungs become filled with thick, sticky mucus making them vulnerable... (Source: NIHR Evaluation, Trials and Studies News)
Source: NIHR Evaluation, Trials and Studies News - July 3, 2015 Category: American Health Source Type: news

Cystic fibrosis hope as new gene therapy improves condition
A new trial shows that genetic therapy can improve cystic fibrosis (Source: Telegraph Health)
Source: Telegraph Health - July 3, 2015 Category: Consumer Health News Tags: cystic fibrosis drug cure health lancet gene therapy symptoms Source Type: news

Cystic fibrosis: Gene therapy offers hope to patients after successful trials
A glimmer of hope for the thousands of families affected by cystic fibrosis has emerged from a clinical trial showing that gene therapy produced a modest but significant improvement among children suffering from the most common inherited disease of white Europeans. (Source: The Independent - Science)
Source: The Independent - Science - July 3, 2015 Category: Science Tags: Science Source Type: news

Gene therapy offers breakthrough for cystic fibrosis sufferers
Scientists from Oxford University and Imperial College London have developed a treatment which repairs the faulty CFTR gene by adding a healthy gene on top. (Source: the Mail online | Health)
Source: the Mail online | Health - July 3, 2015 Category: Consumer Health News Source Type: news

Vertex's $259,000 Cystic Fibrosis Drug Approved
Vertex’s cystic-fibrosis drug Orkambi, which treats the most common mutation of the disease, received U.S. regulatory approval on Thursday. (Source: WSJ.com: Health)
Source: WSJ.com: Health - July 3, 2015 Category: Pharmaceuticals Tags: PAID Source Type: news

Cystic Fibrosis: New Hope for Gene Therapy? (CME/CE)
(MedPage Today) -- Early results suggest stabilization in lung function (Source: MedPage Today Pulmonary)
Source: MedPage Today Pulmonary - July 3, 2015 Category: Respiratory Medicine Source Type: news

Cystic fibrosis gene therapy boost
A gene therapy has stabilised and slightly improved cystic fibrosis in some of 136 patients in a trial. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - July 3, 2015 Category: Consumer Health News Source Type: news

Gene Therapy for Cystic Fibrosis Shows Some Promise in Study
Lung function benefit was slight, but even keeping lungs stable would be improvement, experts say (Source: Pulmonary Medicine News - Doctors Lounge)
Source: Pulmonary Medicine News - Doctors Lounge - July 3, 2015 Category: Respiratory Medicine Authors: webmaster at doctorslounge.com Tags: Pulmonology, Research, News, Source Type: news

Gene therapy offers hope to cystic fibrosis patients after successful trials
A glimmer of hope for the thousands of families affected by cystic fibrosis has emerged from a clinical trial showing that gene therapy produced a modest but significant improvement among children suffering from the most common inherited disease of white Europeans. (Source: The Independent - Science)
Source: The Independent - Science - July 2, 2015 Category: Science Tags: Science Source Type: news

For first time, gene therapy shows promise in cystic fibrosis
LONDON (Reuters) - Scientists conducting a major trial of a therapy that replaces the faulty gene responsible for cystic fibrosis say the treatment has showed significant benefit for the first time in patients' lung function. (Source: Reuters: Health)
Source: Reuters: Health - July 2, 2015 Category: Consumer Health News Tags: healthNews Source Type: news

Gene Therapy for Cystic Fibrosis Shows Some Promise in Study
Lung function benefit was slight, but even keeping lungs stable would be improvement, experts say Source: HealthDay Related MedlinePlus Pages: Cystic Fibrosis, Genes and Gene Therapy (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - July 2, 2015 Category: Consumer Health News Source Type: news

Gene Therapy for Cystic Fibrosis Improves Lung FunctionGene Therapy for Cystic Fibrosis Improves Lung Function
Liposome-based delivery of the CFTR gene sequence improved lung function in patients with cystic fibrosis treated monthly for a year. Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 2, 2015 Category: Consumer Health News Tags: Pulmonary Medicine News Source Type: news

FDA Clears Combo Drug Orkambi for Cystic FibrosisFDA Clears Combo Drug Orkambi for Cystic Fibrosis
Orkambi combines the approved drug ivacaftor (Kalydeco) with lumacaftor and is indicated for patients with CF aged 12 years and older with two copies of the F508del mutation. FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - July 2, 2015 Category: Consumer Health News Tags: Pulmonary Medicine News Alert Source Type: news

FDA OKs Cystic Fibrosis Combination
(MedPage Today) -- Drug combo targets most common root cause of disease (Source: MedPage Today Pulmonary)
Source: MedPage Today Pulmonary - July 2, 2015 Category: Respiratory Medicine Source Type: news

Vertex Pharma's lung disorder therapy wins FDA nod
(Reuters) - Vertex Pharmaceuticals Inc's unique combination therapy for a rare lung disorder was given the green light by U.S. health regulators, extending the company's reach to the most common form of genetic mutation responsible for cystic fibrosis. (Source: Reuters: Health)
Source: Reuters: Health - July 2, 2015 Category: Consumer Health News Tags: healthNews Source Type: news

Orkambi Approved for Cystic Fibrosis
When disease caused by mutated gene from each parent (Source: Pulmonary Medicine News - Doctors Lounge)
Source: Pulmonary Medicine News - Doctors Lounge - July 2, 2015 Category: Respiratory Medicine Authors: webmaster at doctorslounge.com Tags: Pharmacy, Pulmonology, FDA Approvals, Source Type: news

Orkambi, a New Cystic Fibrosis Drug, Wins F.D.A. Approval
The drug is the second one brought to market by Vertex and could be effective for more patients than the first drug, Kalydeco. (Source: NYT Health)
Source: NYT Health - July 2, 2015 Category: Consumer Health News Authors: ANDREW POLLACK Tags: Cystic Fibrosis Drugs (Pharmaceuticals) Food and Drug Administration Vertex Pharmaceuticals Inc VRTX NASDAQ Biotechnology and Bioengineering Source Type: news

Orkambi Approved for Cystic Fibrosis
When disease caused by mutated gene from each parent Source: HealthDay Related MedlinePlus Pages: Cystic Fibrosis, Medicines (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - July 2, 2015 Category: Consumer Health News Source Type: news

FDA approves new treatment for cystic fibrosis
The U.S. Food and Drug Administration today approved the first drug for cystic fibrosis directed at treating the cause of the disease in people who have two copies of a specific mutation. (Source: Food and Drug Administration)
Source: Food and Drug Administration - July 2, 2015 Category: American Health Source Type: news

Cystic Fibrosis Might Be Deadlier for Hispanics, Study Suggests
Title: Cystic Fibrosis Might Be Deadlier for Hispanics, Study SuggestsCategory: Health NewsCreated: 6/30/2015 12:00:00 AMLast Editorial Review: 7/1/2015 12:00:00 AM (Source: MedicineNet Lungs General)
Source: MedicineNet Lungs General - July 1, 2015 Category: Respiratory Medicine Source Type: news

Cystic Fibrosis Might Be Deadlier for Hispanics, Study Suggests
Genetic differences may thwart response to latest treatments, researchers say (Source: Pulmonary Medicine News - Doctors Lounge)
Source: Pulmonary Medicine News - Doctors Lounge - July 1, 2015 Category: Respiratory Medicine Authors: webmaster at doctorslounge.com Tags: Pulmonology, Research, News, Source Type: news

Cystic Fibrosis Might Be Deadlier for Hispanics
Genetic differences may thwart response to latest treatments, researchers say Source: HealthDay Related MedlinePlus Pages: Cystic Fibrosis, Genes and Gene Therapy, Hispanic American Health (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - June 30, 2015 Category: Consumer Health News Source Type: news

ProQR begins Phase Ib trial of QR-010 to treat patients with cystic fibrosis
Dutch biotech firm ProQR Therapeutics has initiated a Phase Ib clinical trial (PQ-010-001) of QR-010, a new investigational RNA therapeutic designed to repair the genetic mutation in the mRNA of cystic fibrosis (CF) patients due to the DF508 mutation… (Source: Drug Development Technology)
Source: Drug Development Technology - June 28, 2015 Category: Pharmaceuticals Source Type: news

'I wanted to know: is there something lurking in my children's genes?'
From discovering their risk of cystic fibrosis to finding a sibling, three people share their experiences of home DNA-testing kit 23andMe (Source: Telegraph Health)
Source: Telegraph Health - June 27, 2015 Category: Consumer Health News Tags: cystic fibrosis anne wojcicki lactose intolerance breast cancer 23andme gene mutation home dna testing kit Source Type: news

Cystic fibrosis deadlier for Hispanic than non-Hispanic patients, Stanford study finds
(Stanford University Medical Center) Cystic fibrosis is more deadly for Hispanic than non-Hispanic patients, a disparity that is not explained by differences in their access to health care, according to a new study from the Stanford University School of Medicine. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - June 24, 2015 Category: Biology Source Type: news

Nivalis IPO nets $88M
Boulder biotech Nivalis Therapeutics Inc. said last week's IPO raised more than $88 million. Nivalis shares (NASDAQ: NVLS) began trading last Wednesday. The company, which is concentrating on treating people with cystic fibrosis, said it sold 6,325,000 shares of its common stock at $14 per share. The total amount is before underwriting commission and discounts and offering expenses. In addition to cystic fibrois, the company said it will work on pharmaceuticals to "develop therapeutics for patients… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - June 23, 2015 Category: Pharmaceuticals Authors: Ben Miller Source Type: news

Cystic fibrosis sufferer and lung transplant survivor Emily Hoyle to climb world's highest volcano
A lung transplant transformed the life of cystic fibrosis sufferer, Emma Hoyle, and she is joining a group of transplant recipients who are attempting a 6000m climb Cotopaxi, the highest volcano in the world (Source: The Telegraph : Health Advice)
Source: The Telegraph : Health Advice - June 22, 2015 Category: Consumer Health News Tags: cystic fibrosis Emma Hoyle Harefield Hospital Andre Simon Climbing for my donor lung transplant Source Type: news

How Does Spinal Muscle Atrophy Present?
Discussion Muscle tone is the slight tension that is felt in a muscle when it is voluntarily relaxed. It can be assessed by asking the patient to relax and then taking the muscles through a range of motion such as moving the wrists, forearm and upper arm. Muscle strength is the muscle’s force against active resistance. Impaired strength is called weakness or paresis. There are 5 levels of muscle strength. Hypotonia can occur with or without weakness. Decreased fetal movements in utero, persistent hypotonia and difficulty feeding are more consistent with congenital rather than an acquired hypotonia. Common treatable c...
Source: PediatricEducation.org - June 22, 2015 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Clinical Updates in Cystic Fibrosis-Related DiabetesClinical Updates in Cystic Fibrosis-Related Diabetes
Learn more about systic fibrosis-related diabetes, the commonest extrapulmonary complication of cystic fibrosis. Seminars in Respiratory and Critical Care Medicine (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - June 19, 2015 Category: Consumer Health News Tags: Critical Care Journal Article Source Type: news

Boulder biotech Nivalis sees shares rise in IPO
Stock in bIotech drug developer Nivalis Therapeutics Inc. jumped 6.5 percent on its first day of public trading Wednesday, closing at $14.92 per share. The Boulder-based company generated $77 million from sales of 5.5 million shares in its IPO. Nivalis stock (Nasdaq: NVLS) debuted at $14 and rose as high as $17.89 in mid-day trading before settling to its closing price. The company, formerly N30 Pharmaceuticals, is developing a cystic fibrosis treatment as its leading project. It plans to use… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - June 17, 2015 Category: Biotechnology Authors: Greg Avery Source Type: news