Scientists find new approach that shows promise for treating cystic fibrosis
NIH-funded discovery uses common antifungal drug to improve lungs ’ ability to fight infection. (Source: National Institutes of Health (NIH) News Releases)
Source: National Institutes of Health (NIH) News Releases - March 13, 2019 Category: American Health Source Type: news

Heart-wrenching memoir of woman who died of cystic fibrosis and a superbug
Mallory Smith died of a superbug that infected her cystic fibrosis-ridden lungs at just 25. Her posthumous memoir, Salt in My Soul, reveals the young woman's struggle to not just survive, but thrive. (Source: the Mail online | Health)
Source: the Mail online | Health - March 12, 2019 Category: Consumer Health News Source Type: news

Posaconazole has better therapeutic efficacy in comparison with itraconazole and voriconazole in ABPA amongst Cystic Fibrosis patients
Tue, 03/12/2019 - 11:22News blog (Source: The Aspergillus Website - updates)
Source: The Aspergillus Website - updates - March 12, 2019 Category: Respiratory Medicine Authors: helenfindon Source Type: news

Proteostasis Therapeutics Appoints David Arkowitz to Board of Directors
BOSTON, March 7, 2019 -- (Healthcare Sales & Marketing Network) -- Proteostasis Therapeutics, Inc. (NASDAQ: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (C... Biopharmaceuticals, Personnel Proteostasis Therapeutics, transmembrane conductance regulator, cystic fibrosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - March 7, 2019 Category: Pharmaceuticals Source Type: news

Vertex cystic fibrosis triple combo therapy succeeds in important trials
Vertex Pharmaceuticals Inc on Wednesday said its triple-combination treatment for cystic fibrosis led to significant lung function improvement in two late-stage studies, paving the way for a therapy that could eventually address 90 percent of people with the life-shortening genetic condition. (Source: Reuters: Health)
Source: Reuters: Health - March 6, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Vertex's cystic fibrosis therapy meets goal of late-stage studies
Vertex Pharmaceuticals Inc said on Wednesday its triple-combination therapy showed improvement in lung function for people with cystic fibrosis (CF) in two late-stage studies. (Source: Reuters: Health)
Source: Reuters: Health - March 6, 2019 Category: Consumer Health News Tags: healthNews Source Type: news

Researchers find window of opportunity for treatment of early cystic fibrosis lung infections
(Technical University of Denmark) New research from DTU Biosustain and Rigshospitalet suggests that disease-causing microbes in young cystic fibrosis (CF) patients change rapidly within two to three years after first infection. This knowledge could lead to better treatment strategies to avoid persistent infections. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - March 4, 2019 Category: Infectious Diseases Source Type: news

Plea for NHS to give grandson wonderdrug after HEARTBREAKING cystic fibrosis diagnosis
A COUPLE stricken by cystic fibrosis tragedy and the loss of two sons have pleaded with NHS chiefs to secure Orkambi - as their young grandchild now battles the cruel condition. (Source: Daily Express - Health)
Source: Daily Express - Health - February 26, 2019 Category: Consumer Health News Source Type: news

Daily Express crusade: Sign the petition - NHS must negotiate URGENT deal with Vertex
THE DAILY Express and cystic fibrosis campaigners are urging the public to sign a petition, demanding NHS England negotiate an urgent deal with Vertex. (Source: Daily Express - Health)
Source: Daily Express - Health - February 26, 2019 Category: Consumer Health News Source Type: news

Correct antibiotic dosing could preserve lung microbial diversity in cystic fibrosis
(Children's National Health System) Children and young adults with cystic fibrosis whose lung infections were treated with suboptimal doses of antibiotics had fewer changes in lung microbial diversity during the IV treatment, and their microbial diversity levels were higher 30 days later, a multi-institutional study that includes Children's researchers shows. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - February 22, 2019 Category: Infectious Diseases Source Type: news

Schoolboy, 8, ‘living with a death sentence’ because NHS won’t fund cystic fibrosis ‘wonder drug’
George Monckton, from West Midlands, could be saved from lung deterioration by Orkambi. It is unavailable on the NHS due to a deadlock on a price deal with US manufacturers Vertex. (Source: the Mail online | Health)
Source: the Mail online | Health - February 20, 2019 Category: Consumer Health News Source Type: news

Cystic Fibrosis scandal: UK waits for wonder drug to save children's lives
THE parents of a little girl with cystic fibrosis have revealed their daily battle to keep her healthy as they wait for life-saving drug Orkambi (Source: Daily Express - Health)
Source: Daily Express - Health - February 19, 2019 Category: Consumer Health News Source Type: news

Aspiring makeup artist, 25, suffering from cystic fibrosis is on her third and FINAL set of lungs
Tiffany Senter (pictured) of Shasta Lake City, California is only 25, but she's been at deaths door twice. Tiffany was born with cystic fibrosis and has had to have two lung transplants. (Source: the Mail online | Health)
Source: the Mail online | Health - February 15, 2019 Category: Consumer Health News Source Type: news

Woman, 30, with cystic fibrosis is disappointed after being denied NHS funding for IVF and surrogacy
Sophie Gannon, from Bexley, South London, had a double lung transplant in 2011. Her and her partner, Josh Thomas, were told they do not fit the funding criteria, and are now fundraising money. (Source: the Mail online | Health)
Source: the Mail online | Health - February 11, 2019 Category: Consumer Health News Source Type: news

Cystic fibrosis drug scandal: It 'gave us back our lives'
A CYSTIC fibrosis sufferer says she has "got her life back" after being treated with a drug at the centre of a funding dispute. (Source: Daily Express - Health)
Source: Daily Express - Health - February 10, 2019 Category: Consumer Health News Source Type: news

Drug Combo Tolerated in Younger Children With Cystic Fibrosis
THURSDAY, Feb. 7, 2019 -- Lumacaftor/ivacaftor treatment is generally safe and well tolerated for children aged 2 to 5 years with cystic fibrosis (CF) homozygous for the F508del-CFTR mutation, according to a study published online Jan. 24 in The... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - February 7, 2019 Category: Pharmaceuticals Source Type: news

Proteostasis Therapeutics Appoints Emmanuel Dulac and Kim Drapkin to Board of Directors
BOSTON, Feb. 6, 2019 -- (Healthcare Sales & Marketing Network) -- Proteostasis Therapeutics, Inc. (NASDAQ: PTI), a clinical stage biopharmaceutical company dedicated to the discovery and development of groundbreaking therapies to treat cystic fibrosis (CF... Biopharmaceuticals, Personnel Proteostasis Therapeutics, transmembrane conductance regulator, cystic fibrosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - February 6, 2019 Category: Pharmaceuticals Source Type: news

Cystic fibrosis sufferer slams the NHS after being denied funding for IVF and surrogacy
Sophie Gannon, from Bexley, South London, had a double lung transplant in 2011. Her and her partner, Josh Thomas, were told they do not fit the funding criteria, and are now fundraising money. (Source: the Mail online | Health)
Source: the Mail online | Health - February 6, 2019 Category: Consumer Health News Source Type: news

Four-year-old cystic fibrosis sufferer may have to move to Scotland to get access to a 'wonder drug'
Harriet Corr, from Jarrow in Tyne and Wear, was diagnosed with the genetic disorder at just five days old and now takes a cocktail of drugs every day to prevent sticky mucus building up in her lungs. (Source: the Mail online | Health)
Source: the Mail online | Health - February 5, 2019 Category: Consumer Health News Source Type: news

Campaigners urge the Government to end NHS price war over £105,000 cystic fibrosis 'wonder drug'
Parents are calling for action to make life-extending Orkambi available to British children with cystic fibrosis, like eight-year-old Luis Walker (pictured), who last year wrote to a drug company. (Source: the Mail online | Health)
Source: the Mail online | Health - February 4, 2019 Category: Consumer Health News Source Type: news

'I wish I could just have a normal life'
"Life-extending" drugs for those with cystic fibrosis are unavailable due to a dispute over cost. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - February 4, 2019 Category: Consumer Health News Source Type: news

My son ’s life depends on this cystic fibrosis drug. And ministers stand in the way | Christina Walker
The government could make the drug Orkambi affordable for the NHS. Until then, people will suffer undulyLuis was three weeks old when we found out he hadcystic fibrosis. Neither my husband nor I had heard of the condition before, but we knew very quickly our lives were about to change for ever from that moment. Of all of the stats thrown at you when you hear the diagnosis, one sticks out more than any other: Luis ’slife expectancy was 38, my age at the time. We would most likely outlive our baby boy.Luis is eight years old now and, though parts of our routine will be recognisable to every parent, he has a life of man...
Source: Guardian Unlimited Science - February 4, 2019 Category: Science Authors: Christina Walker Tags: Cystic fibrosis Pharmaceuticals industry Health Society NHS Business Medical research Science UK news Source Type: news

'Deadlock must end' over cystic fibrosis drug Orkambi
Vertex, which makes Orkambi, has refused a £500m offer for the drug over five years (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - February 4, 2019 Category: Consumer Health News Source Type: news

Campaigners urge the Government to end NHS price war over £105,000 cystic fibrosis 'wonder drug'
Parents are calling for action to make life-extending Orkambi available to British children with cystic fibrosis, like eight-year-old Luis Walker (pictured), who last year wrote to a drug company. (Source: the Mail online | Health)
Source: the Mail online | Health - February 4, 2019 Category: Consumer Health News Source Type: news

Drug firm urged to cut £100,000 price of cystic fibrosis drug
Campaigners say the drug could extend the lives of thousands of children Related items fromOnMedica Health innovation hampered by drive for profit, report warns Remedy quest Multidisciplinary prescribing can help reduce errors Guidance issued on prescribing responsibilities Cancer Drugs Fund ‘wasteful and harmful’ (Source: OnMedica Latest News)
Source: OnMedica Latest News - February 4, 2019 Category: UK Health Source Type: news

The Guardian view on new drugs: high hopes, higher prices | Editorial
The row over the cost of a cystic fibrosis treatment has prevented its use in the UK. These patients, many of them children, deserve betterMedical advances come at a cost, and that cost is increasingly steep. We rely on pharmaceutical companies, as well as public and philanthropic investment, to develop the medicines that can transform and even save lives. New drugs emerge not according to need, but when companies know they can make sizable profits from them. The problem with this, beyond our intuitive sense of wrongness when people reap lavish rewards thanks to products others cannot live without, is twofold. Some disease...
Source: Guardian Unlimited Science - February 3, 2019 Category: Science Authors: Editorial Tags: Pharmaceuticals industry Drugs NHS Cystic fibrosis Matt Hancock Science Business The National Institute for Health and Care Excellence (Nice) Society Politics Health policy Public services policy Source Type: news

Help NHS obtain cystic fibrosis drug, ministers urged
Exclusive: Ministers urged to consider revoking patent on life-extending Orkambi, which NHS cannot affordCampaigners are calling on the government to urgently intervene over the cost of a drug that could extend the lives of thousands of children but which manufacturers have set at £105,000 per patient per year, a price the NHS cannot afford.For more than two years an emotive battle has been raging over Orkambi, which tackles the cause ofcystic fibrosis. Previously, children with the genetic disorder did not often survive to adulthood and even today, half will be dead before the age of 32. Orkambi could extend lives, ...
Source: Guardian Unlimited Science - February 3, 2019 Category: Science Authors: Sarah Boseley Health editor Tags: Drugs Cystic fibrosis Pharmaceuticals industry UK news Health NHS Science Politics Source Type: news

Inhaled mannitol cost effective in cystic fibrosis in Australia
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - February 1, 2019 Category: Drugs & Pharmacology Source Type: news

Bespoke Babies to Prevent CF: Best Use Case or Ethical Bog?
(MedPage Today) -- Bioethicist and pediatrician debate cystic fibrosis gene-editing (Source: MedPage Today OB/GYN)
Source: MedPage Today OB/GYN - January 28, 2019 Category: OBGYN Source Type: news

Her gene therapy company granted $4.5M by Cystic Fibrosis Foundation
Talee Bio Inc., a Philadelphia biopharmaceutical company developing gene therapy treatments for cystic fibrosis, received a grant of up to $4.5 million from the Cystic Fibrosis Foundation. The funds will be used by Talee Bio to fund the preclinical development of two gene therapy product candidates. The company has set a goal of initiating clinical trials for the first one in 2020. The foundation has previously supported the company's product development efforts, which Talee Bio sa id is the result… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 24, 2019 Category: Pharmaceuticals Authors: John George Source Type: news

Cystic fibrosis drug combo found safe and effective for 2- to 5-year-olds
(Ann& Robert H. Lurie Children's Hospital of Chicago) An open label Phase 3 study conducted at Ann& Robert H. Lurie Children's Hospital of Chicago and other centers established that a combination of cystic fibrosis drugs lumacaftor and ivacaftor is safe and effective in children aged 2-5 years, whose disease is caused by two copies of F508del-CFTR gene mutation -- the most common and severe form of cystic fibrosis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 24, 2019 Category: International Medicine & Public Health Source Type: news

Your Sweat Will See You Now
A new device — wearable, wireless and battery free — improves the ability to monitor and diagnose health problems by analyzing the sweat on your skin. (Source: NYT Health)
Source: NYT Health - January 18, 2019 Category: Consumer Health News Authors: APOORVA MANDAVILLI Tags: Sweating Sensors Cystic Fibrosis Wearable Computing Data-Mining and Database Marketing Exercise Skin Diabetes Source Type: news

Congress Funds Feasibility Study for Mesothelioma Patient Registry
The National Institute for Occupational Safety and Health will oversee a study in 2019 to determine the feasibility of establishing the first national mesothelioma patient registry. The $100,000 feasibility study was part of the $1.2 million Congress allocated in 2019 to the Centers of Disease Control and Prevention for mesothelioma research. The bulk of the funding goes toward the National Mesothelioma Virtual Bank, which is a biospecimen registry first established in 2006. It is the only federally funded program designed exclusively for the research of mesothelioma, the rare and aggressive cancer caused by asbestos expos...
Source: Asbestos and Mesothelioma News - January 15, 2019 Category: Environmental Health Authors: Matt Mauney Source Type: news

Game-changing research reveals that parental experience is epigenetically imprinted onto 14 successive generations
(Natural News) We all know that we inherit certain specific genetic characteristics from our parents and that these are indelibly written in our DNA code. These genetic traits determine everything from the color of our eyes to whether or not we have dimples. Unfortunately, certain genetic diseases like cystic fibrosis and hemophilia can be passed... (Source: NaturalNews.com)
Source: NaturalNews.com - January 14, 2019 Category: Consumer Health News Source Type: news

Researchers study bacterial immunity to understand infectious disease
(Carl R. Woese Institute for Genomic Biology, University of Illinois at Urbana-Champaign) Patients with cystic fibrosis are often infected by pseudomonas aeruginosa, a bacterium that infects the lungs and prevents breathing, often causing death. P. aeruginosa itself can also be infected by viruses, which can affect the clinical outcomes of cystic fibrosis patients. University of Illinois Professor of Microbiology Rachel Whitaker and her team had the idea to use P. aeruginosa as a kind of 'model system' for understanding how bacteria's interactions with viruses may affect human health. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - January 8, 2019 Category: Infectious Diseases Source Type: news

What happens when someone loses disability insurance?
Megan Willis lives with cystic fibrosis, a deadly disease that causes extensive lung damage (Source: Health News: CBSNews.com)
Source: Health News: CBSNews.com - January 5, 2019 Category: Consumer Health News Source Type: news

Engineers create an inhalable form of messenger RNA
(Massachusetts Institute of Technology) In an advance that could lead to new treatments for lung disease, MIT researchers have now designed an inhalable form of mRNA. This aerosol could be administered directly to the lungs to help treat diseases such as cystic fibrosis, the researchers say. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - January 4, 2019 Category: International Medicine & Public Health Source Type: news

13 local life sciences drugs and devices to watch in 2019
It's a new year, and local life sciences companies are readying a stream of new products. A dozen Massachusetts companies are currently conducting final clinical tests and assessing rounds of data for an array of new treatments that could be sent to the FDA for approval in the next year. Among them are Vertex's highly anticipated triple combination Cystic Fibrosis drug, Intarcia's bele aguered diabetes device and Biogen's Alzheimer's treatment. Some companies are staring down a risk-filled path… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - January 2, 2019 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

A posttranslational modification code for CFTR maturation is altered in cystic fibrosis
The multistep process regulating the maturation of membrane proteins in the endoplasmic reticulum (ER) and the secretory pathway is disrupted in many protein misfolding disorders. Mutations in the ion channel CFTR that impair its folding and subsequent localization to the plasma membrane cause cystic fibrosis (CF), an inherited and eventually lethal disease that impairs the function of multiple organs, mostly the lungs. Here, we found that proper maturation of CFTR is dependent on cross-talk between phosphorylation and methylation events in the regulatory insertion (RI) element of the protein. Manipulating these posttransl...
Source: Signal Transduction Knowledge Environment - January 1, 2019 Category: Science Authors: Pankow, S., Bamberger, C., Yates, J. R. Tags: STKE Research Articles Source Type: news

How To Design Healthcare Services For Generation Z
Is the journey to designing healthcare services for Gen Z an uphill battle? Fluxx decided to take a different approach when designing an app for cystic fibrosis patients. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - December 30, 2018 Category: Pharmaceuticals Authors: Phee Waterfield, Contributor Source Type: news

Girl with cystic fibrosis is at the top of the national 'super urgent' list for a lung transplant
Ruby Walker, from Angus, Scotland, is at the top of the national 'super urgent' list. Her family worry 'the window of time for her transplant is closing' as she has waited for more than two years. (Source: the Mail online | Health)
Source: the Mail online | Health - December 24, 2018 Category: Consumer Health News Source Type: news

Chemists discover new signatures to identify cystic fibrosis in infants sooner
(McMaster University) Scientists at McMaster University have discovered several new biomarkers from a single drop of blood that could allow earlier and more definitive detection of cystic fibrosis (CF), a genetic disease which strikes both children and adults, causing chronic problems with the digestive system and the lungs. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - December 20, 2018 Category: Biology Source Type: news

Bronchiectasis (non-cystic fibrosis), acute exacerbation: antimicrobial prescribing.NICE guideline [NG117]
This guideline sets out an antimicrobial prescribing strategy for managing and preventing acute exacerbations of bronchiectasis (non-cystic fibrosis). It aims to optimise antibiotic use and reduce antibiotic resistance. It was published in December 2018. (Source: Current Awareness Service for Health (CASH))
Source: Current Awareness Service for Health (CASH) - December 19, 2018 Category: Consumer Health News Source Type: news

Waltham's Eloxx aims to reach cystic fibrosis patients that Vertex can't
The Waltham startup is taking on the market giant Vertex Pharmaceuticals by developing a pill that targets the 10 percent of patients that Vertex and others haven't touched. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - December 19, 2018 Category: Pharmaceuticals Authors: Allison DeAngelis Source Type: news

EU Panel Recommends Tobramycin PARI for Pseudomonas in CF EU Panel Recommends Tobramycin PARI for Pseudomonas in CF
The European Medicines Agency's Committee for Medicinal Products for Human Use recommended Tobramycin PARI for Pseudomonas aeruginosa in patients aged 6 years and older with cystic fibrosis.International Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - December 14, 2018 Category: Consumer Health News Tags: Pulmonary Medicine News Alert Source Type: news

Inhaling Hypertonic Saline May Aid Infants With Cystic Fibrosis
THURSDAY, Dec. 13, 2018 -- In infants with cystic fibrosis (CF), preventive inhalation with hypertonic saline (HS) during the first months of life is safe and well tolerated and results in clinical improvements, according to a study recently... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - December 13, 2018 Category: Pharmaceuticals Source Type: news

'Our Christmas miracle is happening': 17-year-old with cystic fibrosis awakes from two-week coma
Christina VanDerVelden, 17, of Jacksonville, Florida, slipped into a week-long coma after her healthy rapidly declined. The cystic fibrosis sufferer now needs a double lung transplant to survive. (Source: the Mail online | Health)
Source: the Mail online | Health - December 12, 2018 Category: Consumer Health News Source Type: news

Blood test could lead to cystic fibrosis treatment tailored to each patient
(Ann& Robert H. Lurie Children's Hospital of Chicago) Researchers at Stanley Manne Children's Research Institute at Ann& Robert H. Lurie Children's Hospital of Chicago, and colleagues, used a blood test and microarray technology to identify distinct molecular signatures in children with cystic fibrosis. These patterns of gene expression ultimately could help predict disease severity and treatment response, and lead to therapies tailored to each patient's precise biology. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - December 12, 2018 Category: International Medicine & Public Health Source Type: news