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 3, 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 3, 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 3, 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 3, 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 29, 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

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:Hospitals headlines)
Source: bizjournals.com Health Care:Hospitals headlines - June 17, 2015 Category: Hospital Management Authors: Greg Avery Source Type: news

New tool identifies novel compound targeting causes of type 2 diabetes
A new drug screening technology has identified a new potential anti-diabetes compound -- and a powerful way to quickly test whether other molecules can have a positive effect on a critical molecular pathway believed to be central to diseases ranging from diabetes to retinitis pigmentosa, cystic fibrosis, Huntington's disease, and Alzheimer's. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - June 17, 2015 Category: Science Source Type: news

Lung transplant survival rates good for Canadians with cystic fibrosis
(Source: St. Michael's Hospital News and Media)
Source: St. Michael's Hospital News and Media - June 15, 2015 Category: Hospital Management Tags: Hospital News Source Type: news

Protalix BioTherapeutics Announces AIR DNase(TM) Data Presented at the 38th European Cystic Fibrosis Conference
(Source: Medical News (via PRIMEZONE))
Source: Medical News (via PRIMEZONE) - June 12, 2015 Category: Pharmaceuticals Source Type: news

A Family's Heart-Warming Meeting With Recipient of Son's Organ Donation
A 26-year-old student was saved with a lung transplant after battling cystic fibrosis. (Source: ABC News: Health)
Source: ABC News: Health - June 3, 2015 Category: Consumer Health News Tags: Health Source Type: news

Cystic Fibrosis Medicine Study Offers Hope
(Source: eMedicineHealth.com)
Source: eMedicineHealth.com - May 27, 2015 Category: Journals (General) Source Type: news

Cystic Fibrosis Medicine Study Offers Hope
Title: Cystic Fibrosis Medicine Study Offers HopeCategory: Health NewsCreated: 5/27/2015 12:00:00 AMLast Editorial Review: 5/27/2015 12:00:00 AM (Source: MedicineNet Lungs General)
Source: MedicineNet Lungs General - May 27, 2015 Category: Respiratory Medicine Source Type: news

Cystic Fibrosis Medicine Study Offers Hope
Many people with cystic fibrosis are optimistic about a new drug on the horizon, Orkambi. (Source: WebMD Health)
Source: WebMD Health - May 26, 2015 Category: Consumer Health News Source Type: news

[Research Article] A molecular switch in the scaffold NHERF1 enables misfolded CFTR to evade the peripheral quality control checkpoint
A molecular switch in a scaffolding protein enables a misfolded, but partially functional, cystic fibrosis protein to evade a quality control checkpoint. (Source: Signal Transduction Knowledge Environment)
Source: Signal Transduction Knowledge Environment - May 20, 2015 Category: Science Authors: Cláudia A. Loureiro, Ana Margarida Matos, Ângela Dias-Alves, Joana F. Pereira, Inna Uliyakina, Patrícia Barros, Margarida D. Amaral, Paulo Matos Source Type: news

Novel Drug Combo Improves Function of Cystic Fibrosis Protein - 5/20/15
A novel two-drug combination has the potential to target and restore a defective protein underlying cystic fibrosis (CF), according to two phase III clinical trials conducted at 187 medical centers around the world, including Johns Hopkins. (Source: Johns Hopkins Medicine News)
Source: Johns Hopkins Medicine News - May 20, 2015 Category: Research Source Type: news

Lung Transplantation for Cystic FibrosisLung Transplantation for Cystic Fibrosis
Find out more about when to consider lung transplantation and more for patients with cystic fibrosis. Seminars in Respiratory and Critical Care Medicine (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - May 20, 2015 Category: Consumer Health News Tags: Critical Care Journal Article Source Type: news

Key component in protein that causes cystic fibrosis identified
(University of Missouri-Columbia) Nearly 70,000 people worldwide are living with cystic fibrosis, a life-threatening genetic disease. There currently is no cure for the condition, but researchers from the University of Missouri have identified a key component in the protein that causes the disease. It is a finding that may lay the foundation for the development of new medications and improved therapies. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - May 19, 2015 Category: Biology Source Type: news

'Groundbreaking' treatment found for cystic fibrosis
Stephen FellerCHICAGO, May 18 (UPI) -- A combination of drugs cystic fibrosis drugs was found in a "groundbreaking" study to address the disease itself, rather than just ease symptoms for patients. (Source: Health News - UPI.com)
Source: Health News - UPI.com - May 18, 2015 Category: Consumer Health News Source Type: news

Drug combination for cystic fibrosis looks promising
Conclusion This trial has demonstrated that this new treatment combination could be effective in improving lung function for people with cystic fibrosis who have two copies of the common Phe508del CFTR mutation. The trial has many strengths, including its large sample size and the fact it captured outcomes at six months for almost all participants. The improvements in lung function were seen while the participants continued to use their standard cystic fibrosis treatments. As the researchers suggest, this indicates the treatment could be a beneficial add-on to normal care to further improve symptoms. The results seem very...
Source: NHS News Feed - May 18, 2015 Category: Consumer Health News Tags: Heart/lungs Medication Source Type: news

Combo Treatment for Cystic Fibrosis Shows Promise
Researchers say therapy could help 15,000 people in United States alone Source: HealthDay Related MedlinePlus Pages: Cystic Fibrosis, Genes and Gene Therapy, Medicines (Source: MedlinePlus Health News)
Source: MedlinePlus Health News - May 18, 2015 Category: Consumer Health News Source Type: news

Drug Combo Targets Common Cystic Fibrosis Gene AbnormalityDrug Combo Targets Common Cystic Fibrosis Gene Abnormality
Results from the phase 3 TRAFFIC and TRANSPORT trials suggest the oral combination of lumacaftor and ivacaftor improves lung function and curbs exacerbations. Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - May 18, 2015 Category: Consumer Health News Tags: Pulmonary Medicine News Source Type: news

Combo Treatment for Cystic Fibrosis Shows Promise
Title: Combo Treatment for Cystic Fibrosis Shows PromiseCategory: Health NewsCreated: 5/18/2015 12:00:00 AMLast Editorial Review: 5/18/2015 12:00:00 AM (Source: MedicineNet Lungs General)
Source: MedicineNet Lungs General - May 18, 2015 Category: Respiratory Medicine Source Type: news

New treatment brings hope on cystic fibrosis
Professor Stuart Elborn, who led the European part of the trial at Queen's University, Belfast, described the drug as a 'breakthrough'. (Source: the Mail online | Health)
Source: the Mail online | Health - May 18, 2015 Category: Consumer Health News Source Type: news