Nivalis cystic fibrosis drug gets key FDA 'orphan' designation
Nivalis Therapeutics Inc. said the U.S. Food and Drug Administration has given its N91115 cystic fibrosis drug an "orphan drug" designation, and the company's shares soared in Friday trading. Boulder-based Nivalis (Nasdaq: NVLS) shares rose more than 15 percent, rising 77 cents to close at $5.75 on Friday after the FDA announcement. An "orphan drug" designation is given to drugs for diseases that affect less than 200,000 people in the U.S., and gives a drug company a seven-year marketing exclusivity… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - January 18, 2016 Category: Biotechnology Authors: Ben Miller Source Type: news

NIH genome sequencing program targets the genomic bases of common, rare disease
The National Institutes of Health will fund a set of genome sequencing and analysis centers whose research will focus on understanding the genomic bases of common and rare human diseases. The National Human Genome Research Institute (NHGRI), part of NIH, today launched the Centers for Common Disease Genomics (CCDG), which will use genome sequencing to explore the genomic contributions to common diseases such as heart disease, diabetes, stroke and autism. NHGRI also announced the next phase of a complementary program, the Centers for Mendelian Genomics (CMG), which will continue investigating the genomic underpinnings of ra...
Source: NHGRI Press Releases - January 14, 2016 Category: Genetics & Stem Cells Source Type: news

Researchers further illuminate pathway for treatment of cystic fibrosis
New research findings add further clarity to a question that has polarized the cystic fibrosis (CF) research community. It is well established that people with cystic fibrosis have two faulty copies of the CFTR gene, but debate continues on the question of whether certain symptoms of the airway disease are caused by the mutation or if the genetic defect precedes, but does not directly lead to some of the worst symptoms patients face. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - January 12, 2016 Category: Science Source Type: news

Manchester teen with cystic fibrosis writes bucket list before she dies
Chloe Hopkins, 19 of Manchester, has cystic fibrosis and her health is deteriorating. Without a double lung transplant, doctors have said she won't live more than two years (Source: the Mail online | Health)
Source: the Mail online | Health - January 12, 2016 Category: Consumer Health News Source Type: news

Advanced Inhalation Therapies scales back IPO terms to $10m
Advanced Inhalation Therapies yesterday scaled its pending initial public offering by more than ⅔ as it looks to raise funds for its drug delivery device, which is designed to treat severe respiratory infections and lung diseases using a high dose of nitric oxide. When it registered for the IPO back in August 2015, Rehovot, Israel-based AIT set the high end of the offering at $36 million. Yesterday the company said it plans to float 675,000 shares at $15 apiece, for gross proceeds of $10.1 million. Losses for AIT last year widened by 200%, to -$4.6 million, or -41¢ per share, compared with&n...
Source: Mass Device - January 12, 2016 Category: Medical Equipment Authors: Brad Perriello Tags: Funding Roundup Initial Public Offering (IPO) Respiratory Advanced Inhalation Therapies Source Type: news

What Are Risk Factors for Cholelithiasis?
Discussion Bile is produced by the liver to aid absorption of fat soluble vitamins and lipids from the gastrointestinal tract and to transport bilirubin, cholesterol and other substances to the gastrointestinal tract. Bile is the main form of cholesterol excretion. Gallstones or cholelithiasis form when the balance of substances within the hepatobiliary tract favors supersaturation with crystal formation and gallstone formation. It is a dynamic state of affairs as gallstones can form and also have a high rate of resorption of up to 50%. Gallstones 3 mm are called gallstones. Gallstones, while not as common as adult popula...
Source: PediatricEducation.org - January 4, 2016 Category: Pediatrics Authors: pediatriceducationmin Tags: Uncategorized Source Type: news

Mapping a way to medication adherence
Eighteen-year-old Maggie Mansfield, a communications major at Boston College, is a two-time double-lung transplant recipient — once, at age 4, due to a condition called pulmonary hypertension in which blood pressure in the arteries of the lungs is abnormally high, and again at age 7, when her body rejected the first transplant. Since then, Maggie has remained relatively healthy due in part to her strict medication regimen. Maggie Mansfield “Timing is the most challenging part of taking my medications,” Maggie says. “As I get older, I get busier. It’s not always easy to stop what I am doing and...
Source: Thrive, Children's Hospital Boston - December 29, 2015 Category: Pediatrics Authors: Emily Williams Tags: Health & Wellness Our Patients’ Stories Teen Health double lung transplant Liver transplant Pediatric Transplant Center (PTC) prescription medication pulmonary hypertension Source Type: news

Behind the Headlines' Top Five of Top Fives 2015
In this study, researchers wanted to see why this is and if there could be any human applications.Researchers collected white blood cells from African and Asian elephants. They found that elephants have at least 20 copies of a gene called TP53. TP53 is known to encourage cell "suicide" when DNA is damaged, stopping any potential cancer in its tracks. In contrast, humans are thought to have only a single copy of the TP53 gene.Of course the big question – the elephant in the room, if you will – is how we can boost TP53 activity in humans to stimulate a similar protective effect. The simple answer is: we...
Source: NHS News Feed - December 24, 2015 Category: Consumer Health News Tags: QA articles Medical practice Special reports Source Type: news

Behind the Headlines Top Five of Top Fives 2015
In this study, researchers wanted to see why this is and if there could be any human applications. Researchers collected white blood cells from African and Asian elephants. They found elephants have at least 20 copies of a gene called TP53. TP53 is known to encourage cell "suicide" when DNA is damaged, stopping any potential cancer in its tracks. In contrast, humans are thought to have only a single copy of the TP53 gene. Of course the big question – the elephant in the room, if you will – is how we can boost TP53 activity in humans to stimulate a similar protective effect. The simple answer is: we do...
Source: NHS News Feed - December 24, 2015 Category: Consumer Health News Tags: QA articles Medical practice Special reports Source Type: news

News Quiz: 2015 an Important Year for the LungsNews Quiz: 2015 an Important Year for the Lungs
New drugs for COPD, asthma, and cystic fibrosis made headlines this year. Experts debated the apnea treatment vacuum and what to do about tricky pulmonary fibrosis and contaminated bronchoscopes. Test how well you kept up with the news this year Medscape Medical News (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - December 22, 2015 Category: Consumer Health News Tags: Pulmonary Medicine News Source Type: news

Case Western Reserve School of Nursing scientist to lead new cystic fibrosis research
(Case Western Reserve University) A scientist at Case Western Reserve University Frances Payne Bolton School of Nursing will lead a pair of studies to develop more effective treatment for symptoms of cystic fibrosis (CF), a life-threatening genetic disease that causes persistent lung infections and progressively limits the ability to breathe. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - December 17, 2015 Category: Biology Source Type: news

Ethnic differences in CF genetic coding not addressed in screening tests for nonwhite patients
(Elsevier Health Sciences) Cystic fibrosis (CF) occurs less frequently in nonwhites than in whites, and nonwhites tend to be diagnosed at a later age. Delaying diagnosis can result in postponed treatment and clinical deterioration. A new study in The Journal of Molecular Diagnostics found that one reason for this ethnic disparity in CF diagnoses is that the variants examined in the most common CF newborn screening panels do not sufficiently include the variants present in nonwhite populations. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - December 16, 2015 Category: Biology Source Type: news

Orkambi pill gives fresh hope to cystic fibrosis victims
Orkambi was licensed for UK use last month, and watchdog the National Institute for Health and Care Excellence (NICE) is now considering the case for it to be made available on the NHS. (Source: the Mail online | Health)
Source: the Mail online | Health - December 13, 2015 Category: Consumer Health News Source Type: news

Pulmatrix Appoints Mark Iwicki Chairman of the Board
LEXINGTON, Mass., Dec. 8, 2015 -- (Healthcare Sales & Marketing Network) -- Pulmatrix, Inc. (PULM), a biopharmaceutical company developing inhaled therapies for pulmonary disease, today announced the appointment of Mark Iwicki as chairman of its board of ... Biopharmaceuticals, Drug Delivery, PersonnelPulmatrix, iSPERSE, cystic fibrosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - December 8, 2015 Category: Pharmaceuticals Source Type: news

Cystic Fibrosis sufferer pleads for access to ‘groundbreaking’ drug
HSE says it cannot afford Orkambi which reportedly costs €160,000 per patient per year (Source: The Irish Times - Health)
Source: The Irish Times - Health - December 8, 2015 Category: Consumer Health News Source Type: news

Cystic Fibrosis sufferer calls for access to ‘groundbreaking’ drug
HSE says it cannot afford Orkambi which reportedly costs €160,000 per patient per year (Source: The Irish Times - Health)
Source: The Irish Times - Health - December 8, 2015 Category: Consumer Health News Source Type: news

Kalydeco, For Cystic Fibrosis, Wins First Annual Forbes Breakthrough Drug Award
Kalydeco, a treatment for cystic fibrosis developed by Vertex Pharmaceuticals with support from the Cystic Fibrosis Foundation, was awarded the first Forbes Breakthrough Drug Award today for dramatically improving the lives of patients and conquering a scientific challenge that had vexed researchers for decades. (Source: Forbes.com Healthcare News)
Source: Forbes.com Healthcare News - December 3, 2015 Category: Pharmaceuticals Authors: Matthew Herper Source Type: news

Nivalis begins dosing in Phase II trial of N91115 to treat cystic fibrosis
US-based Nivalis Therapeutics has begun a Phase II clinical trial of its lead investigational drug, N91115, a stabiliser of the cystic fibrosis (CF) transmembrane conductance regulator (CFTR) protein. (Source: Drug Development Technology)
Source: Drug Development Technology - December 2, 2015 Category: Pharmaceuticals Source Type: news

Nivalis begins second phase of cystic fibrosis drug study
Nivalis Therapeutics Inc. said it's begun the second phase of a study of its N91115 drug targeting cystic fibrosis. The Boulder biotech (Nasdaq: NVLS) said results of the study, which will examine the efficiency and safety of the drug, will be available in the second half of next year. "We are optimistic about its potential ability to improve lung function in people with cystic fibrosis," said Jon Congleton, president and chief executive officer of Nivalis, in a statement. Nivalis Therapeutics,… (Source: bizjournals.com Health Care:Biotechnology headlines)
Source: bizjournals.com Health Care:Biotechnology headlines - December 1, 2015 Category: Biotechnology Authors: Ben Miller Source Type: news

Medical News Today: Protein chatter to blame for cystic fibrosis
Irrelevant chatter among proteins appears to underlie cystic fibrosis; the discovery offers hope for a solution to the life-threatening condition. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - December 1, 2015 Category: Consumer Health News Tags: Cystic Fibrosis Source Type: news

Promise and peril of gene-editing technology CRISPR
CRISPR could help rid of diseases like cystic fibrosis, muscular dystrophy and even HIV and cancer (Source: Health News: CBSNews.com)
Source: Health News: CBSNews.com - November 30, 2015 Category: Consumer Health News Source Type: news

Gene Editing - A Future Disease Cure?
Scientists are making tiny scissors called, 'TALEN's,' that can cut and fix a broken gene in a cell. The technology is not ready for use in people yet but when it is it might help people to cure a number of different genetic diseases as long as those diseases are caused by issues with a single gene. The new tool has the potential to help with diseases such as cystic fibrosis or sickle cell anemia, diseases where a single gene is broken. (Source: Disabled World)
Source: Disabled World - November 24, 2015 Category: Disability Tags: Medical Research Source Type: news

Talk About It Over Turkey: The Power of Your Family History
Discussions of genetic screening prior to or during pregnancy tend to be focused on DNA testing and less on family history. Understandably, medical providers may be more comfortable ordering a standard DNA test than interpreting three generations of family folklore. Tests such as those used to identify healthy "carrier-carrier" couples, who have a 25% chance to have a child with a recessive condition, or those used to screen for sporadic chromosomal conditions in a baby, are valuable screening tools. However, they cover a limited number of disorders and in many cases will not address significant concerns posed by...
Source: Science - The Huffington Post - November 24, 2015 Category: Science Source Type: news

New light shed on mysterious fungus that has major health consequences
Fungi in the mucus of patients with cystic fibrosis has been examined by researchers who have discovered how one particularly cunning fungal species has evolved to defend itself against neighboring bacteria. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 21, 2015 Category: Science Source Type: news

U of T research sheds new light on mysterious fungus that has major health consequences
(University of Toronto) Researchers at the University of Toronto examined fungi in the mucus of patients with cystic fibrosis and discovered how one particularly cunning fungal species has evolved to defend itself against neighbouring bacteria. (Source: EurekAlert! - Infectious and Emerging Diseases)
Source: EurekAlert! - Infectious and Emerging Diseases - November 20, 2015 Category: Infectious Diseases Source Type: news

Gene therapy for cystic fibrosis effective in mice
Stephen FellerLEUVEN, Belgium, Nov. 16 (UPI) -- Researchers found inserting genetic material into intestinal cells to correct the disorder causing cystic fibrosis worked in human cell cultures and mice. (Source: Health News - UPI.com)
Source: Health News - UPI.com - November 16, 2015 Category: Consumer Health News Source Type: news

Gene therapy: Promising candidate for cystic fibrosis treatment
An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment, suggest new encouraging results. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - November 16, 2015 Category: Science Source Type: news

Medical News Today: Improved gene therapy shows potential as a treatment for cystic fibrosis
A mouse study of an improved gene therapy suggests it shows promise as a treatment for cystic fibrosis, but there is still a long way to go before it can be tested in humans. (Source: Health News from Medical News Today)
Source: Health News from Medical News Today - November 16, 2015 Category: Consumer Health News Tags: Cystic Fibrosis Source Type: news

California's Newborn Cystic Fibrosis Screening Shows PromiseCalifornia's Newborn Cystic Fibrosis Screening Shows Promise
Findings over the course of 5 years came from a large, diverse group, but some experts caution that results from the 3-step protocols raised some concerns. Medscape Medical News (Source: Medscape Pediatrics Headlines)
Source: Medscape Pediatrics Headlines - November 16, 2015 Category: Pediatrics Tags: Pediatrics News Source Type: news

Gene therapy: A promising candidate for cystic fibrosis treatment
(KU Leuven) An improved gene therapy treatment can cure mice with cystic fibrosis (CF). Cell cultures from CF patients, too, respond well to the treatment. Those are the encouraging results of a study presented by the Laboratory for Molecular Virology and Gene Therapy at KU Leuven, Belgium. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - November 16, 2015 Category: Global & Universal Source Type: news

Spraying new genes into your lungs could ease misery of cystic fibrosis 
For Oli Dillon, 17, from Kent, an effective therapy for cystic fibrosis can't come soon enough. (Source: the Mail online | Health)
Source: the Mail online | Health - November 10, 2015 Category: Consumer Health News Source Type: news

First steps towards new therapy for diseases caused by defective anion transport
Research by a team at the University of Bristol has taken the first steps towards new treatments for inherited diseases such as cystic fibrosis; one form of Bartter’s syndrome (severe salt loss from the kidney); and two forms of myotonia (muscle stiffness), which are linked to defective movement of anions, such as chloride, across cell membranes. (Source: University of Bristol news)
Source: University of Bristol news - November 5, 2015 Category: Universities & Medical Training Tags: Health, Research; Faculty of Biomedical Sciences, School of Physiology and Pharmacology, Faculty of Science, School of Chemistry, Faculty of Science, Faculty of Biomedical Sciences, Institutes, Elizabeth Blackwell; Press Release Source Type: news

Charlotte, 21, reveals her gratitude for a organ donor gift beyond price
Charlotte Davies, now 21, from Kent, was born with cystic fibrosis, a genetic condition where the lungs and digestive system become clogged with thick, sticky mucus. (Source: the Mail online | Health)
Source: the Mail online | Health - November 3, 2015 Category: Consumer Health News Source Type: news

Husband of mother dying from cystic fibrosis writes letter pleading for more organ donors
Ashley Harris Moore is waiting for a double lung transplant after suffering all her life from cystic fibrosis (Source: Telegraph Health)
Source: Telegraph Health - October 29, 2015 Category: Consumer Health News Tags: cystic fibrosis alastair transplant lung donor donation organ lungs husband ashley harris moore Source Type: news

Vertex Revenue Soars on Growth for Cystic Fibrosis Drugs
Vertex Pharmaceuticals said third-quarter revenue soared 73% as the Boston biotechnology company posted better-than-expected sales of cystic fibrosis drugs Kalydeco and recently launched Orkambi. (Source: WSJ.com: Health)
Source: WSJ.com: Health - October 28, 2015 Category: Pharmaceuticals Tags: PAID Source Type: news

Who Owns Your Genes?
By Diana Brazzell, Co-Founder & Executive Editor, Footnote This post was originally published on Footnote, a website that brings academic research and ideas to a broader audience. New forms of genetic testing can predict whether a couple will have a child with cystic fibrosis, guide doctors in selecting the most effective chemotherapy for a breast cancer patient, and help researchers unlock the causes of Alzheimer's disease. While these advances have the potential to save lives and transform medical research, they also raise serious ethical questions about the balance between privacy and health - questions that scienti...
Source: Science - The Huffington Post - October 27, 2015 Category: Science Source Type: news

23andMe launches new consumer test service to check for genetic disorders
NEW YORK/CHICAGO (Reuters) - Genetics company 23andMe announced the launch of a new consumer genetic test service on Wednesday that will show whether an individual carries genes associated with 36 different disorders, such as cystic fibrosis. (Source: Reuters: Health)
Source: Reuters: Health - October 21, 2015 Category: Consumer Health News Tags: healthNews Source Type: news

Advanced Inhalation Therapies reveals finances ahead of IPO
Advanced Inhalation revealed its finances for the past 2 years as it plans for its oncoming $36 million initial public offering to raise funds for its device designed to treat severe respiratory infections and lung diseases using a high dose of nitric oxide. Advanced Inhalation reported losses of $4.6 million, or 41¢ per share for 2014. That’s up nearly 200% from 2013, when the company reported $1.6 million losses, or 14¢ per share. The last 6 months have been kinder to the Rehovot, Israel-based company, which reported losses of $2 million, or 20¢ per share this year, compared to $3.2 million and ...
Source: Mass Device - October 19, 2015 Category: Medical Equipment Authors: Fink Densford Tags: Business/Financial News Drug Pumps Respiratory Advanced Inhalation Therapies Source Type: news

Corbus begins dosing in Phase II Resunab trial to treat cystic fibrosis
US-based drug development firm Corbus Pharmaceuticals (CRBP) has started dosing in the Phase II clinical trial of its investigational new oral drug Resunab to treat cystic fibrosis (CF). (Source: Drug Development Technology)
Source: Drug Development Technology - October 16, 2015 Category: Pharmaceuticals Source Type: news

25-year-old Georgina Compton with cystic fibrosis gets much needed lung transplant
Georgina Compton, 25, from Surrey, has battled the genetic condition cystic fibrosis since birth. But being just 4'11" tall, her body was too small for most adult organs. (Source: the Mail online | Health)
Source: the Mail online | Health - October 13, 2015 Category: Consumer Health News Source Type: news

Women with cystic fibrosis bravely bare their scars for Salty Girls book
Ian Pettigrew, a 46-year-old photographer from Ontario, Canada, who is also battling cystic fibrosis, photographed 77 adult women who suffer from the disease for his book Salty Girls. (Source: the Mail online | Health)
Source: the Mail online | Health - October 13, 2015 Category: Consumer Health News Source Type: news

The Man Who Grew Eyes
The train line from mainland Kobe is a marvel of urban transportation. Opened in 1981, Japan’s first driverless, fully automated train pulls out of Sannomiya station, guided smoothly along elevated tracks that stand precariously over the bustling city streets below, across the bay to the Port Island. The island, and much of the city, was razed to the ground in the Great Hanshin Earthquake of 1995 – which killed more than 5,000 people and destroyed more than 100,000 of Kobe’s buildings – and built anew in subsequent years. As the train proceeds, the landscape fills with skyscrapers. The Rokkō mounta...
Source: Healthy Living - The Huffington Post - October 11, 2015 Category: Consumer Health News Source Type: news

The Man Who Grew Eyes
The train line from mainland Kobe is a marvel of urban transportation. Opened in 1981, Japan’s first driverless, fully automated train pulls out of Sannomiya station, guided smoothly along elevated tracks that stand precariously over the bustling city streets below, across the bay to the Port Island. The island, and much of the city, was razed to the ground in the Great Hanshin Earthquake of 1995 – which killed more than 5,000 people and destroyed more than 100,000 of Kobe’s buildings – and built anew in subsequent years. As the train proceeds, the landscape fills with skyscrapers. The Rokkō mounta...
Source: Science - The Huffington Post - October 11, 2015 Category: Science Source Type: news

Anthera begins Sollpura Phase III trial to treat EPI in patients with cystic fibrosis
Anthera Pharmaceuticals has started a Phase III trial (Solution) of Sollpura (liprotamase) to treat patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis (CF). (Source: Drug Development Technology)
Source: Drug Development Technology - October 5, 2015 Category: Pharmaceuticals Source Type: news

Bailey Anne Vincent diagnosed with cystic fibrosis reveals devastation
Bailey Anne Vincent, a beauty blogger, was diagnosed with the life-threatening disease after she had her first baby, but was just told by doctors that they now don't know exactly what she has. (Source: the Mail online | Health)
Source: the Mail online | Health - September 30, 2015 Category: Consumer Health News Source Type: news

Five genetic regions implicated in cystic fibrosis severity
If you have two faulty copies of the CFTR gene, you will have cystic fibrosis. But the severity of your disease will depend partly on many other genes. Now, researchers report that five regions of the human genome are home to the genetic variations that play major roles in disease severity. (Source: ScienceDaily Headlines)
Source: ScienceDaily Headlines - September 29, 2015 Category: Science Source Type: news

Let charities take a leading role in discovering antibiotics | the big issue
There is a thriving charity sector developing new drugs – let them work on antibiotic resistanceSeventy years ago Sir Alexander Fleming, the discoverer of penicillin, in his Nobel prize acceptance speech warned that inappropriate use of antibiotics would give rise to resistance (“Time for world to act on antibiotic resistance”, leader, Comment). Fleming’s prediction has come to pass and antibiotic resistance is now threatening modern medicine.Routine procedures including heart bypass surgery, hip and knee replacements, cancer treatments, childbirth, trauma surgery and intensive care treatments all d...
Source: Guardian Unlimited Science - September 27, 2015 Category: Science Authors: Guardian Staff Tags: Antibiotics Medical research Society Science Source Type: news

CHMP Backs Combo Drug Orkambi for Cystic FibrosisCHMP Backs Combo Drug Orkambi for Cystic Fibrosis
Orkambi combines lumacaftor and ivacaftor and is indicated for patients with CF aged 12 years and older who are homozygous for the F508del mutation in the CFTR gene. International Approvals (Source: Medscape Medical News Headlines)
Source: Medscape Medical News Headlines - September 25, 2015 Category: Consumer Health News Tags: Pulmonary Medicine News Alert Source Type: news

Terminally ill gymnast Sonny Lang has the 'heart and lungs of an 80-year-old'
Sonny Lang, 22, from Tadcaster, North Yorkshire, has cystic fibrosis, but has been told her body is too weak for another heart and lung transplant, so her prognosis is terminal. (Source: the Mail online | Health)
Source: the Mail online | Health - September 23, 2015 Category: Consumer Health News Source Type: news

Corbus enrols first patient in Phase II trial of Resunab to treat cystic fibrosis in adults
US-based Corbus Pharmaceuticals Holdings has started patient enrolment in the Phase II clinical trial of its investigational new drug Resunab to treat adult patients with cystic fibrosis (CF). (Source: Drug Development Technology)
Source: Drug Development Technology - September 22, 2015 Category: Pharmaceuticals Source Type: news