Some existing anti-cancer drugs may act in part by targeting RNA, study shows
(Scripps Research Institute) The research offers another approach for tackling diseases that have been considered 'undruggable,' including amyotrophic lateral sclerosis (ALS), muscular dystrophy, cystic fibrosis and certain cancers. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 28, 2018 Category: International Medicine & Public Health Source Type: news

Children's Hospital Colorado joins cystic fibrosis triple combination therapy trials
(Children's Hospital Colorado) Children's Hospital Colorado has announced that its Mike McMorris Cystic Fibrosis Research and Care Center, one of the largest cystic fibrosis clinical care centers in the U.S., is participating in one of two Phase Three clinical trials related to the development of a possible breakthrough therapy for people with CF. Phase Three trials are generally the last round of trials before possible FDA approval of a new drug. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 28, 2018 Category: International Medicine & Public Health Source Type: news

Azithromycin Cuts Pulmonary Exacerbation in CF With Early Pa
WEDNESDAY, June 27, 2018 -- For children with cystic fibrosis (CF) and early Pseudomonas aeruginosa (Pa) infection, the risk of pulmonary exacerbation is significantly reduced with the addition of azithromycin to tobramycin inhalation solution... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - June 27, 2018 Category: Pharmaceuticals Source Type: news

MDI Biological Laboratory receives $70,000 to support organoid course
(Mount Desert Island Biological Laboratory) The MDI Biological Laboratory has announced that it received more than $70,000 in cash and in-kind awards for its inaugural " Applications of Organoid Technology " course, held May 27 through June 2 on the institution's Bar Harbor, Maine, campus. The awards included contributions from Baker, Corning, the Cystic Fibrosis Foundation, Nikon, Leica, Novartis Pharmaceuticals Corp., STEMCELL Technologies, Fisher Scientific and ThermoFisher Scientific. The week-long course was among the first to provide extensive hands-on training in organoid culture. (Source: EurekAlert! - Cancer)
Source: EurekAlert! - Cancer - June 26, 2018 Category: Cancer & Oncology Source Type: news

A Drug Costs $272,000 a Year. Not So Fast, Says New York State.
New York ’ s Medicaid program says Orkambi, a new drug to treat cystic fibrosis, is not worth the price. The case is being closely watched around the country. (Source: NYT Health)
Source: NYT Health - June 24, 2018 Category: Consumer Health News Authors: KATIE THOMAS Tags: Drugs (Pharmaceuticals) Health Insurance and Managed Care Cystic Fibrosis Prices (Fares, Fees and Rates) Medicaid Institute for Clinical and Economic Review orkambi Source Type: news

Improved treatment targeting root cause of cystic fibrosis may be on horizon
A clinical trial led by a pharmaceutical company with additional support from an NIDDK-supported Cystic Fibrosis Center has shown that a combination of two medications provides significant clinical benefit in a subgroup of patients with cystic fibrosis (CF). (Source: NIDDK News)
Source: NIDDK News - June 23, 2018 Category: Endocrinology Source Type: news

Protein-folding Chaperones Have Both Positive and Negative Roles To Play in Cystic Fibrosis
Proteins involved in ensuring that other proteins are in their proper shapes may have a role to play in mitigating the effects of cystic fibrosis. (Source: NIDDK News)
Source: NIDDK News - June 23, 2018 Category: Endocrinology Source Type: news

Irish cystic fibrosis patient begs UK health officials to approve his 'wonder drug'
EXCLUSIVE: Benat Broderick, from Dublin, argued his life has been transformed since he was given Orkambi. But UK watchdogs have denied patients access to the £100,000 medication. (Source: the Mail online | Health)
Source: the Mail online | Health - June 22, 2018 Category: Consumer Health News Source Type: news

Scientists Are Using a New Weapon to Fight Drug-Resistant Bacteria —Viruses
A new medical research center in San Diego is embracing an innovative way to treat antibiotic resistant infections called bacteriophage therapy—phage therapy for short—which uses viruses as weapons against hard-to-treat infections. Antibiotic-resistant infections are part of a growing global health problem. Each year in the United States, at least two million people contract drug-resistant infections, and 23,000 die from those illnesses. Bacteria naturally grow resistant to the drugs used to treat them, and for people with especially tough infections that aren’t responding to the usual medications, the op...
Source: TIME: Health - June 21, 2018 Category: Consumer Health News Authors: Alexandra Sifferlin Tags: Uncategorized healthytime medicine Source Type: news

Warren Alpert Foundation honors pioneering discoveries in cystic fibrosis
(Harvard Medical School) The 2018 Warren Alpert Foundation Prize has been awarded to five scientists for transformative discoveries in the fields of genetics, physiology, pulmonology and pharmacology that have led to the development of life-altering precision-targeted treatments for the devastating multiorgan disease cystic fibrosis (CF). (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 21, 2018 Category: International Medicine & Public Health Source Type: news

Doctors Think a Woman ’s Tattoo May Have Been Causing Her Severe Leg Pain
Doctors in Scotland believe a woman’s large tattoo on her leg may have been the cause of her unexplained inflammation and severe leg pain. The authors of a report on the woman’s case, published in BMJ Case Reports, say it is a reminder to physicians that they should be mindful of their patients’ tattoos if they encounter unusual and unexplained symptoms. The 31-year-old woman was suffering a condition called inflammatory myopathy, which affected her left leg and caused pain severe enough to disrupt her sleep. The woman had a double lung transplant in 2009, and was taking drugs to suppress her immune syst...
Source: TIME: Health - June 19, 2018 Category: Consumer Health News Authors: Alice Park Tags: Uncategorized healthytime onetime tattoos Source Type: news

New approach to advance drug pipeline for cystic fibrosis
For patients with rare genetic mutations of the debilitating disease cystic fibrosis, there are few options for effective treatment. EU-funded researchers are working to meet their needs, using new developments in personalised medicine to advance the testing of novel and promising drugs. (Source: EUROPA - Research Information Centre)
Source: EUROPA - Research Information Centre - June 19, 2018 Category: Research Source Type: news

Cystic fibrosis patient left in chronic pain after getting a TATTOO
The unnamed woman, from Scotland, had a large coloured ink tattoo on her left thigh in 2015 - but developed chronic pain within months. Doctors published the case in the BMJ Case Reports. (Source: the Mail online | Health)
Source: the Mail online | Health - June 18, 2018 Category: Consumer Health News Source Type: news

Tattoo health warning for people with weakened immune systems
A woman with cystic fibrosis and lung transplants suffered chronic pain for three years after she had a tattoo on her thighGetting a tattoo if you have a weakened immune system could put you at risk of complications, doctors have warned. The caution comes after a woman with cystic fibrosis and lung transplants developed thigh and knee pain after having body art inked on her leg.Doctors say those taking immunosuppressant drugs should take precautions if considering body art. These medicines are often given after an organ transplant or to treat autoimmune conditions such as Crohn ’s disease, lupus or rheumatoid arthrit...
Source: Guardian Unlimited Science - June 18, 2018 Category: Science Authors: Nicola Davis Tags: Tattoos Medical research Immunology Biochemistry and molecular biology Health Cystic fibrosis Diabetes Source Type: news

Sharing Mayo Clinic: New lungs give dad a chance to enjoy fatherhood
Over time, cystic fibrosis robbed Nicholas Hemmerle of his ability to enjoy active time with his daughter. But after a double lung transplant, he's breathing easier and ready to have fun again.? The last thing Nicholas Hemmerle remembers before going into surgery at?Mayo Clinic?for a double?lung transplant?is a nurse putting a blanket on him and [...] (Source: News from Mayo Clinic)
Source: News from Mayo Clinic - June 17, 2018 Category: Databases & Libraries Source Type: news

I’ll do anything to save my little girl
THOUSANDS of patients are facing an agonising wait for a miracle drug that can stop the killer disease cystic fibrosis in its tracks. One mum tells LAURA MILNE her family ’s heartbreaking story. (Source: Daily Express - Health)
Source: Daily Express - Health - June 12, 2018 Category: Consumer Health News Source Type: news

Inhaled anti-pseudomonal antibiotics for long-term therapy in cystic fibrosis
This review of 18 RCTs (n=3,042) found that inhaled anti-pseudomonal antibiotic treatment probably improves lung function and reduces exacerbation rate, but pooled estimates of the level of benefit were very limited. The best evidence is for inhaled tobramycin (Source: Current Awareness Service for Health (CASH))
Source: Current Awareness Service for Health (CASH) - June 8, 2018 Category: Consumer Health News Source Type: news

ICER issues final report on CFTR modulators for cystic fibrosis
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - June 1, 2018 Category: Drugs & Pharmacology Source Type: news

Serum (1-3)-beta-D-Glucan and Galactomannan Levels in CF Serum (1-3)-beta-D-Glucan and Galactomannan Levels in CF
Find out about the role of beta-D-glucan and galactomannan levels in detecting fungal disease and assessing lung function in cystic fibrosis patients.BMC Pulmonary Medicine (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - May 30, 2018 Category: Consumer Health News Tags: Pulmonary Medicine Journal Article Source Type: news

Study finds antioxidant-enriched vitamin reduces respiratory illnesses in patients with CF
Researchers at Children's Hospital Colorado (Children's Colorado) and the University of Colorado School of Medicine have found that taking a specially formulated antioxidant-enriched multivitamin may decrease respiratory illnesses in people with cystic fibrosis (CF). The study, which was recently published online in the American Journal of Respiratory and Critical Care Medicine, looked at the effects of a 'cocktail' of multiple antioxidants on inflammation and health outcomes in patients with CF. (Source: World Pharma News)
Source: World Pharma News - May 24, 2018 Category: Pharmaceuticals Tags: Featured Research Research and Development Source Type: news

Her search for new lungs begins with changed heart
Claire Wineland vowed she wouldn't have a lung transplant, but her decline from cystic fibrosis made her reconsider. Now she's fighting for a chance at survival. (Source: CNN.com - Health)
Source: CNN.com - Health - May 23, 2018 Category: Consumer Health News Source Type: news

Study finds antioxidant-enriched vitamin reduces respiratory illnesses in patients with CF
(Children's Hospital Colorado) Researchers at Children's Hospital Colorado and the University of Colorado School of Medicine have found that taking a specially formulated antioxidant-enriched multivitamin may decrease respiratory illnesses in people with cystic fibrosis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 23, 2018 Category: International Medicine & Public Health Source Type: news

AACR Ventures into New Cancer Research Funding Model
(MedPage Today) -- Adapting model pioneered by Cystic Fibrosis Foundation (Source: MedPage Today Public Health)
Source: MedPage Today Public Health - May 22, 2018 Category: American Health Source Type: news

Antibacterial in your toothpaste may combat severe lung disease
(Michigan State University) Michigan State University researchers have found that when triclosan, a substance that reduces or prevents bacteria from growing, is combined with an antibiotic called tobramycin, it kills the cells that protect cystic fibrosis bacteria, known as Pseudomonas aeruginosa, by up to 99.9 percent. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - May 17, 2018 Category: Biology Source Type: news

Cystic fibrosis sufferer takes her first steps after lung transplant
EXCLUSIVE: Footage filmed at Stanford Medical Center in Palo Alto, California, shows Violet Gomez, 25, taking her first steps fueled by healthy lungs full of oxygen, just two days after the op. (Source: the Mail online | Health)
Source: the Mail online | Health - May 9, 2018 Category: Consumer Health News Source Type: news

Incredible moment cystic fibrosis sufferer, 25, takes her first steps days after lung transplant
EXCLUSIVE: Footage filmed at Stanford Medical Center in Palo Alto, California, shows Violet Gomez, 25, taking her first steps fueled by healthy lungs full of oxygen, just two days after the op. (Source: the Mail online | Health)
Source: the Mail online | Health - May 8, 2018 Category: Consumer Health News Source Type: news

Risk of gastrointestinal cancers in patients with cystic fibrosis: a systematic review and meta-analysis
This review suggests patients with cystic fibrosis have an increased risk of gastrointestinal cancer, risk of site-specific GI cancers was also elevated. (Source: Current Awareness Service for Health (CASH))
Source: Current Awareness Service for Health (CASH) - May 2, 2018 Category: Consumer Health News Source Type: news

An Eradication Protocol for B. Cepacia Complex in CF An Eradication Protocol for B. Cepacia Complex in CF
The authors describe a protocolized approach that effectively eradicated Burkholderia cepacia complex in cystic fibrosis patients.BMC Pulmonary Medicine (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - May 2, 2018 Category: Consumer Health News Tags: Pulmonary Medicine Journal Article Source Type: news

Improved treatment targeting root cause of cystic fibrosis may be on horizon
A clinical trial led by a pharmaceutical company has shown that a combination of two medications provides significant clinical benefit in a subgroup of patients with cystic fibrosis (CF). (Source: NIDDK News)
Source: NIDDK News - April 27, 2018 Category: Endocrinology Source Type: news

SPM in Real Life: Spring ’ 18
NEW MEMBERS: Spring ’18 Brief bios/plugs for new members (extracted from the Introduce Yourself Connect Community) Katherine Infantes-Cordova, a bilingual patient liaison passionate about improving the patient experience, is eager to take part in eliminating health inequities and empowering patients to be proactive in their health. Leah Jones has a background in consumer public relations and social media. Her company, Natiiv Facilitation, offers independent focus group moderation, event facilitation, and patient engagement projects. She advocates about fibroids and the challenges people struggling with their weight f...
Source: Society for Participatory Medicine - April 27, 2018 Category: General Medicine Authors: Danny van Leeuwen Tags: Newsletter Source Type: news

Vertex Pharma profit tops estimates on strong launch of new CF drug
(Reuters) - Vertex Pharmaceuticals Inc topped analysts' forecasts for quarterly profit on Thursday, led by stronger-than expected sales of its latest cystic fibrosis drug as well as double-digit growth in sales of older treatments. (Source: Reuters: Health)
Source: Reuters: Health - April 26, 2018 Category: Consumer Health News Tags: healthNews Source Type: news

What Causes Hyponatremia?
Discussion Normal kidneys regulate water balance to maintain a plasma osmolality of 275-290 mOsm/kg normally. Thirst and arginine vasopressin or antidiuretic hormone (ADH) are the primary regulators of plasma osmolality. ADH is made in the hypothalamus and released by the posterior pituitary gland. ADH acts on the kidney’s distal collecting duct to increase water reabsorption. ADH is appropriately released in hypovolemic states, such as dehydration caused by gastroenteritis. ADH has an ~10 minute half-life and therefore can respond to rapid changes in volume status. Sodium balance is regulated by aldosterone (as part...
Source: PediatricEducation.org - April 23, 2018 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Cystic fibrosis: Company urged to lower cost of life-changing drug
Health ministers call on company to lower price of cystic fibrosis drug Orkambi so the NHS can afford it. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - April 21, 2018 Category: Consumer Health News Source Type: news

Symdeko ™ Now Available from Fairview Specialty Pharmacy for Cystic...
Fairview Specialty Pharmacy will now be distributing Symdeko™ (tezacaftor/ivacaftor and ivacaftor), which is approved for treatment of cystic fibrosis.(PRWeb April 13, 2018)Read the full story at http://www.prweb.com/releases/2018/04/prweb15409240.htm (Source: PRWeb: Medical Pharmaceuticals)
Source: PRWeb: Medical Pharmaceuticals - April 13, 2018 Category: Pharmaceuticals Source Type: news

Cytochrome P450 3A4 induction: Lumacaftor versus ivacaftor?
(Bentham Science Publishers) Cystic fibrosis is a disease caused by genetically defective CFTR proteins. The recent approval of lumacaftor combined with ivacaftor targets 70 percent of CF patients with F508del-CFTR. Unfortunately, our understanding of the way these drugs move (pharmacokinetics) and work (pharmacodynamics) in the human body is limited. For the first time, researchers have investigated potential cytochrome interactions of ivacaftor, its major metabolites, lumacaftor and tezacaftor which could result in reduced concentrations of the drug, causing reduced efficacy. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - April 12, 2018 Category: Biology Source Type: news

Proteostasis Therapeutics Receives FDA Fast Track Designation for Triple Combination Program in Patients with Cystic Fibrosis
CAMBRIDGE, Mass., April 4, 2018 -- (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 f... Biopharmaceuticals, FDA Proteostasis Therapeutics, transmembrane conductance regulator, cystic fibrosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - April 4, 2018 Category: Pharmaceuticals Source Type: news

UC scientist patents drug to treat dangerous infections
Daniel Hassett, a scientist with the University of Cincinnati College of Medicine, has been issued a patent for a drug that is could be used to treat lung infections in patients with cystic fibrosis or chronic obstructive pulmonary disease. The drug known as AB569 was developed in the lab of Hassett, a professor in the UC Department of Molecular Genetics, Biochemistry and Microbiology.  It targets many antibiotic-resistant organisms that cause pulmonary infections in patients with cystic fibrosis… (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - April 3, 2018 Category: American Health Authors: Barrett J. Brunsman Source Type: news

UK: calls to end the deadlock on cystic fibrosis drug
(Source: PharmacoEconomics and Outcomes News)
Source: PharmacoEconomics and Outcomes News - April 1, 2018 Category: Drugs & Pharmacology Source Type: news

Test for antibiotic associated kidney damage in children with cystic fibrosis identified
(University of Liverpool) New research, published in Scientific Reports, conducted by the University and partners highlights effective methods for identifying a common side effect in children receiving drug treatments for cystic fibrosis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 28, 2018 Category: International Medicine & Public Health Source Type: news

University of Cincinnati researcher receives US patent for potential COPD treatment
(University of Cincinnati Academic Health Center) A researcher in the University of Cincinnati (UC) College of Medicine has been granted a US patent for a potential treatment for a pulmonary infection in patients with cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD).The treatment, known as AB569, was developed in the lab of Daniel Hassett, PhD, a professor in the UC Department of Molecular Genetics, Biochemistry and Microbiology. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - March 27, 2018 Category: International Medicine & Public Health Source Type: news

James Dunmore lost his two sisters to cystic fibrosis
Made In Chelsea star James Dunmore has told BBC 5 live that he lost two sisters to cystic fibrosis. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - March 22, 2018 Category: Consumer Health News Source Type: news

Cystic Fibrosis Colorectal Cancer Screening Recommendations Cystic Fibrosis Colorectal Cancer Screening Recommendations
The risk of colorectal cancer is greatly increased in adults with cystic fibrosis. This paper presents CRC screening recommendations for these patients, which differ from those for the general public.Gastroenterology (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - March 22, 2018 Category: Consumer Health News Tags: Gastroenterology Journal Article Source Type: news

Chantelle Millward on Orkambi drug for cystic fibrosis patients
MPs are debating a petition calling for all people with cystic fibrosis to be given the life-changing, but very expensive, drug. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - March 19, 2018 Category: Consumer Health News Source Type: news

Student saved by wonder drug NHS won’t pay for
A STUDENT has told how a revolutionary drug that treats cystic fibrosis saved her from a lung transplant. But last night thousands of sufferers of the killer condition were dealt a blow when NHS England announced it would not provide extra funds for the drug Orkambi, which tackles the genetic causes of the disease rather than treating its symptoms. (Source: Daily Express - Health)
Source: Daily Express - Health - March 18, 2018 Category: Consumer Health News Source Type: news

Research Identifies Key Hurdle in Quest for Cystic Fibrosis Treatment
Research offers important insight for new drug therapy to restore proper CFTR folding/function in most with cystic fibrosis, those with the ?F508 mutation. (Source: NIDDK News)
Source: NIDDK News - March 15, 2018 Category: Endocrinology Source Type: news

A Dramatic Improvement in Care for Some People with Cystic Fibrosis
Years of research led to a new medication to overcome the fundamental molecular flaw in some people with cystic fibrosis, greatly reducing their symptoms. (Source: NIDDK News)
Source: NIDDK News - March 15, 2018 Category: Endocrinology Source Type: news

An Important Proof of Principle for the “Combination Therapy” Approach to Treating the Most Common Cystic Fibrosis Mutation
Combination of existing small molecule drug treatment of mutated proteins may lead to new therapies for patients with the most common type of cystic fibrosis. (Source: NIDDK News)
Source: NIDDK News - March 15, 2018 Category: Endocrinology Source Type: news

Improving Genetic Testing for Cystic Fibrosis
Scientists used genetic records of people with cystic fibrosis to find new mutations of the CFTR gene that cause the disease and may improve genetic testing. (Source: NIDDK News)
Source: NIDDK News - March 15, 2018 Category: Endocrinology Source Type: news

East Bay company's stock plummets 81% as drug for cystic fibrosis patients fails
Anthera Pharmaceuticals Inc., which hung its hopes on a two-time loser treatment for cystic fibrosis patients, said the drug failed in a late-stage trial. The stock of the Hayward-based company (NASDAQ: ANTH) fell 81 percent Monday, from $2.64 per share to 50 cents at its close. It had dipped as low as 40 cents. Early data from the 140-patient Phase III study of the drug, called Sollpura, showed that the majority of patients with exocrine pancreati c insufficiency who received the drug saw their… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - March 12, 2018 Category: Biotechnology Authors: Ron Leuty Source Type: news

East Bay company's stock plummets 81% as drug for cystic fibrosis patients fails
Anthera Pharmaceuticals Inc., which hung its hopes on a two-time loser treatment for cystic fibrosis patients, said the drug failed in a late-stage trial. The stock of the Hayward-based company (NASDAQ: ANTH) fell 81 percent Monday, from $2.64 per share to 50 cents at its close. It had dipped as low as 40 cents. Early data from the 140-patient Phase III study of the drug, called Sollpura, showed that the majority of patients with exocrine pancreati c insufficiency who received the drug saw their… (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - March 12, 2018 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news