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

Cystic fibrosis sufferer had a DOUBLE lung transplant aged 22
Emma Khanahmadi, of Sweden, now 25, was diagnosed with CF at the age of eight. She received a donor when she was 'just surviving' and is now thriving. (Source: the Mail online | Health)
Source: the Mail online | Health - May 2, 2019 Category: Consumer Health News Source Type: news

Researcher gets $3.3 million grant to develop universal treatment for cystic fibrosis
(Oregon State University) A pharmaceutical sciences researcher has received a five-year, $3.3 million grant from the National Institutes of Health to develop a way for cystic fibrosis patients to get molecular treatment via an inhaler. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 30, 2019 Category: International Medicine & Public Health Source Type: news

FDA Approves Kalydeco (ivacaftor) as First and Only CFTR Modulator to Treat Eligible Infants with CF as Early as Six Months of Age
BOSTON--(BUSINESS WIRE)--Apr. 30, 2019-- Vertex Pharmaceuticals Incorporated (Nasdaq:VRTX) today announced the U.S. Food and Drug Administration (FDA) approved Kalydeco (ivacaftor) for use in children with cystic fibrosis (CF) ages six months to... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - April 30, 2019 Category: Drugs & Pharmacology Source Type: news

Gene editing could cure cystic fibrosis before birth, study suggests
Scientists at the University of Pennsylvania successfully edited the DNA of embryonic mice just days before birth to get rid of genetic defects that cause fatal lung diseases. (Source: the Mail online | Health)
Source: the Mail online | Health - April 18, 2019 Category: Consumer Health News Source Type: news

Cystic fibrosis sufferer who had double lung transplant contracted a virus from her donor 
Rima Manomaitis, 29, from Pepperell, Massachusetts, received a double lung transplant in May 2017 to treat her cystic fibrosis, only to learn her donor had a dormant virus that had become active. (Source: the Mail online | Health)
Source: the Mail online | Health - April 17, 2019 Category: Consumer Health News Source Type: news

Bacteria-infecting viruses exacerbate chronic infections in cystic fibrosis
(American Association for the Advancement of Science) A study of samples from 92 patients with cystic fibrosis (CF) has revealed that certain bacteriophages -- viruses that infect bacteria -- worsen the severity of bacterial infections associated with the disorder. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - April 17, 2019 Category: Infectious Diseases Source Type: news

New test could lead to personalized treatments for cystic fibrosis
The test combines high-speed video microscopy with a novel video analysis algorithm to measure the coordinated movement of cilia in the lungs. (Source: Yale Science and Health News)
Source: Yale Science and Health News - April 16, 2019 Category: Universities & Medical Training Source Type: news

Medical News Today: Common virus could speed up cystic fibrosis
New research finds that a virus that lies dormant in most of us can accelerate disease progression in people with cystic fibrosis. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - April 15, 2019 Category: Consumer Health News Tags: Cystic Fibrosis Source Type: news

Cytomegalovirus Linked to Faster Progression of Cystic Fibrosis
Faster progression to end - stage lung disease observed among patients with CMV IgG positivity (Source: Pulmonary Medicine News - Doctors Lounge)
Source: Pulmonary Medicine News - Doctors Lounge - April 11, 2019 Category: Respiratory Medicine Tags: Infections, Pulmonology, Journal, Source Type: news

Cytomegalovirus Linked to Faster Progression of Cystic Fibrosis
THURSDAY, April 11, 2019 -- For patients with cystic fibrosis (CF), cytomegalovirus (CMV) is associated with faster disease progression, according to a research letter published online April 7 in the European Respiratory Journal. Michael D. Parkins,... (Source: Drugs.com - Pharma News)
Source: Drugs.com - Pharma News - April 11, 2019 Category: Pharmaceuticals Source Type: news

Health Tip: Understanding Cystic Fibrosis
(Source: Pulmonary Medicine News - Doctors Lounge)
Source: Pulmonary Medicine News - Doctors Lounge - April 9, 2019 Category: Respiratory Medicine Tags: Pulmonology, FYI, Source Type: news

Health Tip: Understanding Cystic Fibrosis
-- Cystic fibrosis (CF) is a genetic condition that causes a bodily protein to malfunction. The faulty protein affects cells, tissues and glands. People with CF make thick mucus that can block, damage and infect organs. Symptoms vary, depending on... (Source: Drugs.com - Daily MedNews)
Source: Drugs.com - Daily MedNews - April 9, 2019 Category: General Medicine Source Type: news

Health Tip: Understanding Cystic Fibrosis
Title: Health Tip: Understanding Cystic FibrosisCategory: Health NewsCreated: 4/9/2019 12:00:00 AMLast Editorial Review: 4/9/2019 12:00:00 AM (Source: MedicineNet Lungs General)
Source: MedicineNet Lungs General - April 9, 2019 Category: Respiratory Medicine Source Type: news

Common virus linked to faster disease progression in cystic fibrosis
(European Lung Foundation) A new study has found that cystic fibrosis patients who have a common virus may experience faster disease progression than patients who do not have the virus. Signs of faster cystic fibrosis disease progression included earlier times to lung transplant referral and reaching the final stages of the disease. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - April 7, 2019 Category: International Medicine & Public Health Source Type: news

Amphotericin B Makes Path for Lung Defense in Cystic Fibrosis Amphotericin B Makes Path for Lung Defense in Cystic Fibrosis
Amphotericin B treatment may be able to restore the infection-fighting properties of the lungs in cystic fibrosis by standing in for a missing or deficient protein channel, studies in pigs and in human lung tissue suggest.Reuters Health Information (Source: Medscape Pathology Headlines)
Source: Medscape Pathology Headlines - March 28, 2019 Category: Pathology Tags: Pulmonary Medicine News Source Type: news

Tackling cystic fibrosis in the womb
(Source: ScienceNOW)
Source: ScienceNOW - March 28, 2019 Category: Science Authors: Maroso, M. Tags: twis Source Type: news

600 years ’ supply of cystic fibrosis drug destroyed in price row
8,000 packs of Orkambi go out of date during standoff between maker and NHSNearly 8,000 packs of Orkambi, the breakthrough medicine for cystic fibrosis, have been destroyed by the manufacturer while it has been in a stand-off with the NHS over the high price it wants to charge for the drug.The US company Vertex has distributed 80,000 packs of Orkambi around Europe from its base in the UK since the drug was licensed in 2015. Other European countries with smaller numbers of people affected by the genetic disease have agreed to a high price, sometimes in order to give access to patients before negotiating downwards.Continue r...
Source: Guardian Unlimited Science - March 27, 2019 Category: Science Authors: Sarah Boseley Health editor Tags: Cystic fibrosis Society NHS Health Drugs Science UK news Source Type: news

Synspira Therapeutics Announces Appointment of Robert Gallotto as President and Chief Executive Officer
Former President of Alcresta Therapeutics brings development, operational and strategic leadership experience as Synspira builds product pipeline Synspira's lead product targeting cystic fibrosis and other pulmonary diseases, SNSP113, to enter Phase 2 C... Biopharmaceuticals, Personnel Synspira Therapeutics, cystic fibrosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - March 27, 2019 Category: Pharmaceuticals Source Type: news