DDT COA #000007: The Cystic Fibrosis Respiratory Symptom Diary – Chronic Respiratory Infection Symptom Score (CFRSD-CRISS)
Clinical Outcome Assessments (COA) Qualification Submissions Office of Antimicrobial Products (OAP) Division of Anti-Infective Products (DAIP) DDT COA Number DDT COA #000007 (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - August 10, 2019 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Antibiotic therapy for chronic infection with Burkholderia cepacia complex in people with cystic fibrosis
Review located one suitable RCT (n=100) which found that inhaled aztreonam lysine did not improve FEV1 and time to next exacerbation vs placebo. Authors therefore conclude that there is insufficient evidence to determine an effective antibiotic strategy for this infection. (Source: Current Awareness Service for Health (CASH))
Source: Current Awareness Service for Health (CASH) - August 8, 2019 Category: Consumer Health News Source Type: news

Sickle Cell Disease Still Tends to Be Overlooked
There is a question about what role race and wealth play in how much attention and funding the disease receives. (Source: NYT)
Source: NYT - August 5, 2019 Category: American Health Authors: Aaron E. Carroll Tags: Sickle Cell Anemia Preventive Medicine Blacks Cystic Fibrosis Discrimination Source Type: news

Respiratory Drug Delivery 2019
5 - 6 December 2019, London, UK. SMi are pleased to announce the launch of the Respiratory Drug Delivery conference as part of the newly branded Asthma and COPD series. With the rising prevalence of respiratory disease such as Asthma, cystic fibrosis and COPD, this group of diseases are now amongst the leading cause of death, affecting over 500 million adults and children worldwide. (Source: World Pharma News)
Source: World Pharma News - August 5, 2019 Category: Pharmaceuticals Tags: Featured Events Source Type: news

Big drug makers trade Peninsula plant as part of larger deal
The San Carlos facility produces an important infection-fighting drug for cystic fibrosis patients. (Source: bizjournals.com Health Care:Pharmaceuticals headlines)
Source: bizjournals.com Health Care:Pharmaceuticals headlines - July 30, 2019 Category: Pharmaceuticals Authors: Ron Leuty Source Type: news

Cameron Sees No Need for National Mesothelioma Registry
Thoracic surgeon Dr. Robert Cameron, a longtime leader in the fight against pleural mesothelioma cancer, believes there is no benefit to establishing a government-funded national registry for the disease. While many of his colleagues support the idea of creating the first National Mesothelioma Registry, Cameron feels the time, effort and money spent would be better used elsewhere in helping patients. The National Institute of Occupational Safety and Health is currently conducting a National Mesothelioma Registry feasibility study for this rare and aggressive cancer. As part of that feasibility study, NIOSH opened a public ...
Source: Asbestos and Mesothelioma News - July 25, 2019 Category: Environmental Health Authors: Matt Mauney Source Type: news

Proteostasis Therapeutics Announces Management Changes and Appointments
Geoffrey S. Gilmartin, MD, MMSc, to Succeed Po-Shun Lee, MD as Chief Medical Officer; Dr. Lee to Remain a Consultant for the Company Andrey E. Belous, MD, PhD, Appointed as Senior Medical Director, Joining From Galapagos NV BOSTON, July 23, 2019 -- (... Biopharmaceuticals, Personnel Proteostasis Therapeutics, transmembrane conductance regulator, cystic fibrosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 23, 2019 Category: Pharmaceuticals Source Type: news

Vertex Submits New Drug Application to the U.S. FDA for Triple Combination Regimen of VX-445 (Elexacaftor), Tezacaftor and Ivacaftor in Cystic Fibrosis
BOSTON--(BUSINESS WIRE)--Jul. 22, 2019-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for the VX-445... (Source: Drugs.com - New Drug Applications)
Source: Drugs.com - New Drug Applications - July 22, 2019 Category: Drugs & Pharmacology Source Type: news

Aridis Pharmaceuticals Receives Orphan Drug Designation From the European Medicines Agency for AR-501
SAN JOSE, Calif., July 19, 2019 -- (Healthcare Sales & Marketing Network) -- Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS) announced today that the European Medicines Agency (EMA) has granted Orphan Drug Designation to AR-501, the Company's inhaled formulat... Biopharmaceuticals, Regulatory Aridis Pharmaceuticals, gallium citrate, cystic fibrosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - July 19, 2019 Category: Pharmaceuticals Source Type: news

Key Cystic Fibrosis Pathogens Reduced With Ivacaftor Use
FRIDAY, July 19, 2019 -- For patients with cystic fibrosis (CF), ivacaftor use is associated with a reduction in pathogens, including Pseudomonas aeruginosa, according to a study published online July 19 in the Annals of the American Thoracic... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - July 19, 2019 Category: Pharmaceuticals Source Type: news

Broadening targeted therapy in cystic fibrosis
(Source: ScienceNOW)
Source: ScienceNOW - July 18, 2019 Category: Science Authors: Alderton, G. Tags: twis Source Type: news

Making precision medicine personal for cystic fibrosis
(Source: ScienceNOW)
Source: ScienceNOW - July 18, 2019 Category: Science Authors: Manfredi, C., Tindall, J. M., Hong, J. S., Sorscher, E. J. Tags: Medicine, Diseases perspective Source Type: news

Cystic fibrosis advocates hope to alert premiers to lack of affordable treatments
Advocates for the rare disease cystic fibrosis are joining forces to gain the attention of the country’s premiers as they meet in Saskatoon. (Source: CBC | Health)
Source: CBC | Health - July 11, 2019 Category: Consumer Health News Tags: News/Canada/Saskatoon Source Type: news

Hinge-like protein may open new doors in cystic fibrosis treatment
(Rockefeller University) Drugs known as potentiators alleviate some symptoms of cystic fibrosis. Researchers recently figured out how these compounds work--a finding that may lead to better drugs that patients can more easily afford. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 9, 2019 Category: International Medicine & Public Health Source Type: news

Hospital celebrates 25 years of cystic fibrosis specialist nurses
A hospital in East Anglia is celebrating 25 years of cystic fibrosis specialist nurses who are praised for helping patients to manage their condition at home and making the  transition from children to adult services easier.  (Source: Nursing Times)
Source: Nursing Times - July 8, 2019 Category: Nursing Source Type: news

Adding Clinical Variables Aids in Lung Allocation for Transplants
New variables ID the sickest waitlist candidates with cystic fibrosis, chronic obstructive pulmonary disease (Source: Pulmonary Medicine News - Doctors Lounge)
Source: Pulmonary Medicine News - Doctors Lounge - July 3, 2019 Category: Respiratory Medicine Tags: Pulmonology, Surgery, Journal, Source Type: news

University of Missouri receives $8.6 million grant for new biomedical research center
(University of Missouri-Columbia) Today, the University of Missouri announced that researchers in the College of Agriculture, Food and Natural Resources have received an $8.6 million grant from the National Institutes of Health to establish a new national research center. The Swine Somatic Cell Genome Editing Center will focus on aiding the development of biomedical treatments for human diseases such as cystic fibrosis. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 27, 2019 Category: International Medicine & Public Health Source Type: news

Cystic fibrosis sufferer told she'd die by 10 gets new lungs at 28
Elizabeth Dolan, 28, was never expected to live past age 10 due to her incurable lung condition. On February 1, she got a double lung transplant in California and is breathing easier than ever. (Source: the Mail online | Health)
Source: the Mail online | Health - June 27, 2019 Category: Consumer Health News Source Type: news

FDA Expands Cystic Fibrosis Treatment Approval to Children Ages 6 to 12
Title: FDA Expands Cystic Fibrosis Treatment Approval to Children Ages 6 to 12Category: Health NewsCreated: 6/24/2019 12:00:00 AMLast Editorial Review: 6/25/2019 12:00:00 AM (Source: MedicineNet Lungs General)
Source: MedicineNet Lungs General - June 25, 2019 Category: Respiratory Medicine Source Type: news

Adding clinical variables improves accuracy of lung allocation score
(Cleveland Clinic) Implemented in 2005, the lung allocation score is used to prioritize patients awaiting lung transplants in the US. Sicker transplant candidates have a higher calculated score and are placed at the top of the list. But a recent study led by Maryam Valapour, M.D., MPP, director of Lung Transplant Outcomes in Cleveland Clinic's Respiratory Institute, found including new clinical variables helped to better identify the sickest cystic fibrosis and chronic obstructive pulmonary disease patients awaiting transplants. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - June 25, 2019 Category: International Medicine & Public Health Source Type: news

FDA Expands Cystic Fibrosis Treatment Approval to Children Ages 6 to 12
(Source: Pulmonary Medicine News - Doctors Lounge)
Source: Pulmonary Medicine News - Doctors Lounge - June 24, 2019 Category: Respiratory Medicine Tags: Pharmacy, Pulmonology, FDA Approvals, Source Type: news

FDA Expands Cystic Fibrosis Treatment Approval for Symdeko (Tezacaftor/ivacaftor) to Children Ages 6 to 12
MONDAY, June 24, 2019 -- The indication for a cystic fibrosis treatment, Symdeko (tezacaftor/ivacaftor) tablets, has been expanded to treat children ages 6 years and older with cystic fibrosis and certain genetic mutations, the U.S. Food and Drug... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - June 24, 2019 Category: General Medicine Source Type: news

Saskatoon woman with cystic fibrosis conquers heights to see Machu Picchu
Corinne McKay says she'd always wanted to see Machu Picchu, and while the 57-year-old has cystic fibrosis, she said she figured she was healthy enough to do the trek now. So she did. (Source: CBC | Health)
Source: CBC | Health - June 24, 2019 Category: Consumer Health News Tags: News/Canada/Saskatchewan Source Type: news

FDA expands approval of treatment for cystic fibrosis to include patients ages 6 and older
FDA expanded the indication for Symdeko for treatment of pediatric patients ages 6 years and older with cystic fibrosis who have certain genetic mutations. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 22, 2019 Category: Drugs & Pharmacology Authors: FDA Source Type: news

FDA Expands Use of Symdeko for Cystic Fibrosis to Younger Children FDA Expands Use of Symdeko for Cystic Fibrosis to Younger Children
The FDA has approved tezacaftor/ivacaftor tablets for use in children as young as 6 years with cystic fibrosis with certain genetic mutations in the CFTR gene.FDA Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - June 21, 2019 Category: Consumer Health News Tags: Pulmonary Medicine News Alert Source Type: news

FDA Expands Cystic Fibrosis Treatment Approval to Children Ages 6 to 12
FRIDAY, June 21, 2019 -- The indication for a cystic fibrosis treatment, Symdeko (tezacaftor/ivacaftor) tablets, has been expanded to treat children ages 6 years and older with cystic fibrosis and certain genetic mutations, the U.S. Food and Drug... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - June 21, 2019 Category: Pharmaceuticals Source Type: news

Structural identification of a hotspot on CFTR for potentiation
Cystic fibrosis is a fatal disease caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR). Two main categories of drugs are being developed: correctors that improve folding of CFTR and potentiators that recover the function of CFTR. Here, we report two cryo–electron microscopy structures of human CFTR in complex with potentiators: one with the U.S. Food and Drug Administration (FDA)–approved drug ivacaftor at 3.3-angstrom resolution and the other with an investigational drug, GLPG1837, at 3.2-angstrom resolution. These two drugs, although chemically dissimilar, bind to the same si...
Source: ScienceNOW - June 20, 2019 Category: Science Authors: Liu, F., Zhang, Z., Levit, A., Levring, J., Touhara, K. K., Shoichet, B. K., Chen, J. Tags: Biochemistry reports Source Type: news

Experts believe viruses may be the answer to beating superbugs
Catherine Farrer from Dulwich, London, is pictured with her daughter Kate, 4, who is a Cystic Fibrosis sufferer. Caring for Kate has exposed her to the worrying realities of antibiotic resistance. (Source: the Mail online | Health)
Source: the Mail online | Health - June 18, 2019 Category: Consumer Health News Source Type: news

Cystic fibrosis drug campaign to be heard in Parliament
The family of Harriet Corr join thousands pleading for access to cystic fibrosis drug Orkambi. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - June 10, 2019 Category: Consumer Health News Source Type: news

Big pharma is denying children like my son vital drugs. So I've set up a buyers club | Robert Long
The US company Vertex has put a ludicrous price on a new cystic fibrosis drug. But we ’ve found a way to bypass its greedA few weeks ago I sat in a meeting room in east London nervously waiting the arrival of two representatives from Gador, an Argentinian drug company. It ’s no exaggeration to say that my child’s life could be shaped by the following few hours.Nearly 10 years ago, shortly after my son Aidan was born, we learnt that he hadcystic fibrosis (CF) – a life-limiting genetic condition that affects the lungs and other major organs. It is difficult to process the joy of parenthood, tempered b...
Source: Guardian Unlimited Science - June 6, 2019 Category: Science Authors: Robert Long Tags: Cystic fibrosis Society Health Pharmaceuticals industry Business Family Life and style NHS US news World news UK news Drugs Science Source Type: news

Cystic fibrosis sufferer, 26, travels the world despite her disease
Chelsea Spruance, 26, from St Thomas in the US Virgin Islands, was diagnosed with cystic fibrosis at four months old after she was unable to keep food down and was not gaining weight. (Source: the Mail online | Health)
Source: the Mail online | Health - June 5, 2019 Category: Consumer Health News Source Type: news

Families desperate for cystic fibrosis drug are importing a cheaper version from Argentina
UK families have taken inspiration from the Oscar-winning movie, Dallas Buyers Club, in a bid to help children with cystic fibrosis such as Shiloh Howells, pictured. (Source: the Mail online | Health)
Source: the Mail online | Health - June 5, 2019 Category: Consumer Health News Source Type: news

Rugby Laboratories Issues Voluntary Nationwide Recall of Diocto Liquid and Diocto Syrup Manufactured By PharmaTech, LLC Due to Possible Product Contamination
Rugby ® Laboratories of Livonia, MI is voluntarily recalling all lots within the expiry of Diocto Liquid and Diocto Syrup, (docusate sodium solutions) manufactured by PharmaTech, LLC of Davie, FL due to a risk of product contamination with Burkholderia cepacia. If a product contains B. cepacia, its use c ould result in infections in patients with compromised immune systems and in patients with chronic lung conditions such as cystic fibrosis. Some of these infections may be serious or even life-threatening in the at-risk patient population. (Source: FDA Center for Drug Evaluation and Research - What's New)
Source: FDA Center for Drug Evaluation and Research - What's New - June 5, 2019 Category: Drugs & Pharmacology Authors: FDA Source Type: news

Supporting Medication Adherence for Cystic Fibrosis Supporting Medication Adherence for Cystic Fibrosis
What strategies can clinicians use to encourage medication adherence in their cystic fibrosis patients?BMC Pulmonary Medicine (Source: Medscape Allergy Headlines)
Source: Medscape Allergy Headlines - June 5, 2019 Category: Allergy & Immunology Tags: Pulmonary Medicine Journal Article Source Type: news

Screening and Counseling for Alcohol Use in Adolescents With Chronic Medical Conditions in the Ambulatory Setting - Lunstead J, Weitzman ER, Harstad E, Dedeoglu F, Gaffin JM, Garvey KC, MacGinnitie A, Rufo PA, Fishman LN, Wisk LE, Levy S.
PURPOSE: We seek to determine how youth with chronic medical conditions experience alcohol screening and counseling. METHODS: Adolescents with type I diabetes, juvenile idiopathic arthritis, moderate persistent asthma, cystic fibrosis, attention de... (Source: SafetyLit)
Source: SafetyLit - May 30, 2019 Category: International Medicine & Public Health Tags: Age: Adolescents Source Type: news

Manuka honey to kill drug-resistant bacteria found in cystic fibrosis infections
(Swansea University) Manuka honey could provide the key to a breakthrough treatment for cystic fibrosis patients following preliminary work by experts at Swansea University. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - May 29, 2019 Category: Infectious Diseases Source Type: news

In The end, Rare Drug Pricing Will Hurt Pharma Just As Much As Patients
There ’s been an odd-sounding word circulating in the English headlines recently.The word is Orkambi; a drug created by Vertex that treats approximately 40% of people born with the genetic condition, cystic fibrosis. It is the second drug to market in a promising pipeline that targets the CFTR mutation.   True gene therapy it isn’t, but it represents a near-first for a biotech firm, by successfully altering the shape and function of a protein[1]. And priced at £105,000 per patient per year, it is the latest in a long line of medicines the NHS can’t afford.Orkambi has been licensed by the EMAsin...
Source: EyeForPharma - May 24, 2019 Category: Pharmaceuticals Authors: Elly Aylwin-Foster Source Type: news

Cystic Fibrosis drugs scandal: Hope as NHS tables new offer for life-changing drug
A NEW, improved NHS offer for life-saving drugs was welcomed by cystic fibrosis sufferers last night. The Daily Express has been pressurising NHS England to strike a deal with US drugs giant Vertex for its pipeline of CF medicines like Orkambi. (Source: Daily Express - Health)
Source: Daily Express - Health - May 24, 2019 Category: Consumer Health News Source Type: news

Cystic Fibrosis Patients Turn to Experimental Phage Therapy
Phages have not been approved by the Food and Drug Administration, but there is growing interest in the treatment for cystic fibrosis. (Source: NYT Health)
Source: NYT Health - May 17, 2019 Category: Consumer Health News Authors: ABBY ELLIN Tags: Antibiotics Cystic Fibrosis Infections Drug Resistance (Microbial) Drugs (Pharmaceuticals) Bacteria Lungs Source Type: news

'Please don't let us die': Three cystic fibrosis sufferers beg the NHS to fund 'wonder drug'
EXCLUSIVE: The drug, which slows lung deterioration, received its European licence three-and-a-half years ago, in which time over 200 people have died from the cruel condition. (Source: the Mail online | Health)
Source: the Mail online | Health - May 17, 2019 Category: Consumer Health News Source Type: news

Cystic fibrosis scandal: Little Eve makes 65 roses in plea for life-saving drugs
A TODDLER with cystic fibrosis will deliver a bunch of 65 roses to Prime Minister Theresa May today, to plead for life-saving drugs to be available on the NHS. Eve Jones, one, was born with the cruel, deteriorating genetic condition, but wonder drug Orkambi could add decades to her life. (Source: Daily Express - Health)
Source: Daily Express - Health - May 16, 2019 Category: Consumer Health News Source Type: news

What does ‘Five Feet Apart’ get right and wrong about cystic fibrosis?
(Source: St. Michael's Hospital News and Media)
Source: St. Michael's Hospital News and Media - May 15, 2019 Category: Hospital Management Tags: Hospital News Source Type: news

Proteostasis Therapeutics Appoints Dr. Badrul Chowdhury, Former FDA Director of Pulmonology, Allergy, and Rheumatology, to Board of Directors
BOSTON, May 13, 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 - May 13, 2019 Category: Pharmaceuticals Source Type: news

2 young Canadian brothers, a life-threatening disease — and the harsh reality of drug prices
This week's story of two young Canadian brothers with cystic fibrosis reveals a bleak reality — only one has access to a promising new medication in a clinical trial, while a similar drug that could help his brother is too expensive. But access to the manufacturer's drugs is not just a problem in Canada. There are bitter fights in other jurisdictions over how much the company is charging. (Source: CBC | Health)
Source: CBC | Health - May 11, 2019 Category: Consumer Health News Tags: News/Health Source Type: news

Fighting for the drug that pauses cystic fibrosis
Orkambi is a drug that targets a mutation that around 50% of people with cystic fibrosis in the UK have. (Source: BBC News | Health | UK Edition)
Source: BBC News | Health | UK Edition - May 11, 2019 Category: Consumer Health News Source Type: news

FDA Panel Recommends Mannitol Inhalation Powder for CF FDA Panel Recommends Mannitol Inhalation Powder for CF
An FDA panel voted 9 to 7 to recommend mannitol inhalation powder (Bronchitol, Chiesi) for improvement of pulmonary function in adults with cystic fibrosis, along with standard therapies.Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - May 9, 2019 Category: Consumer Health News Tags: Pulmonary Medicine News Source Type: news

Genetically modified virus saves teen's life, offers hope in fight against antibiotic resistance
Isabelle Carnell-Holdaway, 17, has faced not one but two unrelenting threats to her life. Diagnosed at 11 months of age with cystic fibrosis, the progressive genetic disease that causes lung infections and breathing impairment, Isabelle has also combated an on-again, off-again infection caused by antibiotic-resistant bacteria since age 8. (Source: CNN.com - Health)
Source: CNN.com - Health - May 9, 2019 Category: Consumer Health News Source Type: news

Virus cocktail saves the life of dying girl amid hopes of breakthrough in fight against superbugs 
Isabelle Carnell-Holdaway, of Faversham, Kent, received a double lung transplant aged 17 to treat cystic fibrosis. But afterwards a drug-resistant superbug took hold of the teenager. (Source: the Mail online | Health)
Source: the Mail online | Health - May 9, 2019 Category: Consumer Health News Source Type: news