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A four-year-old boy who hasn't been able to leave the house since the pandemic hit due to his cystic fibrosis is bouncing his way through lockdown.

Theo Powell, from Tunbridge Wells, has been getting involved in a national challenge - Bounce 4 CF for the Cystic Fibrosis Trust, taking to his trampoline in a dragon costume and bounding around his garden in a yellow sack - the brand colour of the trust.

He is one of 10,500 people in the UK who have the condition which causes thick, sticky mucus to build up in the lungs, and is therefore in the vulnerable group during the coronavirus crisis.

Trampoline sessions are part of Theo's daily exercise and physiotherapy routine, as the movement helps to dislodge the mucus and improve his breathing.

So, encouraged by his parents, Caroline and Laurence, the Nivan Lodge Pre-School pupil decided to also use this as a way to raise money for a cause which the family has been involved in for some time.

Due to Theo's condition, the family have had to stay home since advice for the shielding group was issued by the Government., and do not know when they will be able to go out again.

Rev Powell, vicar at King Charles the Martyr Church priest in charge of St Mark's Church, has been holding services via Zoom and Mrs Powell has been trying to come up with inventive ways to keep Theo entertained during lockdown.

Mrs Powell, 31, said: "Every day gets quite similar but we are trying to mix it up when we can - camping in the garden, building stuff - we're keeping busy."

Although, she says Theo is used to not being allowed out due to frequent hospital trips.

"He's been in hospital quite a lot - he spent a month and a half there in total last year and when he's there, he's not allowed out of the room in case of cross contamination," said Mrs Powell.

Her husband added: "This strict shielding lockdown is a difficult situation for the whole family.

"Theo has just been accepted at the local school but at the moment we are not sure whether he will be able to start with his cohort."

The family are also pushing to get Theo a life-extending therapy called trikafta on the NHS.

Last year, hundreds shared their delight as Orkambi, said to slow decline in lung function, was made widely available after several years of campaigning. However, it does not treat all cystic fibrosis patients, with trikafta helping others whose DNA is not compatible with Orkambi, such as Theo.

In addition, Mrs Powell is working on a separate Instagram campaign, @wearyellow2020, encouraging people to post photos of them wearing yellow in support of Cystic Fibrosis Trust on Friday, June 19.

Along with her friend, Laura Fine, Mrs Powell is running a raffle on the day, with other games and fun activities planning for the week running up to 'wear yellow day'.

To support Theo in his Bounce 4 CF campaign, click here, and to get involved in the family's Instagram campaign, click here.

Read more: All the latest news from Tunbridge Wells

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Boy with cystic fibrosis bounces his way through lockdown - Kent Online

BY: HILARY PEARSON

One thing most of us have learnt during this pandemic: germs can spread as far as six feet when someone coughs or sneezes. They can land on surfaces, such as a doorknob, or in another persons eyes, nose, or mouth. Thats why social distancing guidelines state we should be standing six feet apart to limit the spread of the Corona virus.

For people with Cystic Fibrosis (CF), social distancing has always been a way of life. Being close to others with the disease puts them at increased risk of getting and spreading dangerous germs and bacteria, a term referred to as crossinfection. Not only are these dangerous germs difficult to treat, but they can lead to worsening symptoms and faster decline in lung function for those with CF.

Thats why its important for people with CF to stay at least six feet away from others with CF and anyone with a cold, flu, or infection.

Ive listened to this audiobook, Five Feet Apart, a few times now.

Stella Grant a teen living with CF describes her experience living with chronic illness:

Counting out exact doses of multiple medications. Being extra careful to not forget one. Careful to not accidentally overdose. Careful to take them at the specified time. Missing out on social events due to a flare up of symptoms. Going to the hospital because of catching a cold. Were basically doctors by the age of twelve.

I feel this. All the work that goes into my fight with my disability. Chronic pain. Chronic fatigue. Chronic migraines. Chronic asthma. Memory loss. Aphasia. Photophobia. Phonophobia. Post concussion syndrome. Brain damage.

I cant begin to understand what CF individuals have to go through. I dont know their fight. But I can begin to connect with the things we feel. Like the isolation. The complicated relationships. The chronic everything.

Even if you dont have a chronic condition, I still recommend reading this book.

We are all fighting in this pandemic. We can all begin to relate to those who need to always keep social distancing in mind.

We can all begin to see each other.

Hilary is a Toronto-based non-fiction writer and UofT masters student. Hilary is recovering from TBI, PCS and spends much of her free time on FaceTime with Isla, her baby niece.

This post originally appeared on her Instagram, @halite_brain_beads

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How Five Feet Apart a story about Cystic Fibrosis is relevant today - stopthefud

Buying quality merchandise when it's on sale is a stock market strategy used by some of the greatest investors in the world - including Warren Buffett. After theturmoil we've seen in 2020, there are signsthat stocks likeCF Industries Holdings Inc(NYQ:CF) could fit that bill. But how can you tell?

TheCF share price has moved by-37.3%over the past three months and its currently trading at24.59.

In volatile conditions,many investors are keen to buy what they think are cheap stocks- but this can be a mistake. It's importantknow the difference between a genuine bargain and a value trap - and often, the quality of the stock makes all the difference.

The good news is thatCF scores well against some important financial and technical measures.It's a large-cap share with strong exposure to twovery influential drivers of investment returns: high quality and a relatively cheap valuation.

To understand where that shows up,here's a closer look:

GET MORE DATA-DRIVEN INSIGHTS INTO NYQ:CF

Good quality stocks are loved by the market because they'remore likely to be solid, dependable businesses. Profitability is important, but so is the firm'sfinancial strength. A track record of improving finances is essential.

One of the stand out quality metrics forCFis that it passes8of the9 financial tests in the Piotroski F-Score. The F-Score is a world-class accounting-based checklist for findingstocks with an improving financial health trend. A good F-Score suggests that the company has strong signs ofquality.

While quality is important, no-one wants to overpay for a stock, so an appealingvaluation is vital too. With a weaker economy, earnings forecasts are unclear right across the market.But there are some valuation measures that can help, and one of them is the Earnings Yield.

Earnings Yieldcompares a company's profit with itsmarket valuation (worked out by dividing itsoperating profit by its enterprise value). It gives you a total value of the stock (including its cash and debt), which makes it easier to comparedifferent stocks. As a percentage, the higher the Earnings Yield, the better value the share.

A rule of thumb for a reasonable Earnings Yield might be5%, and theEarnings Yield forCFis currently8.06%.

In summary, good quality and relatively cheap valuations are pointers to those stocks that aresome of themost appealing to contrarian value investors. It's among these shares that genuine mis-pricing can be found. Once the market recognises that these quality firms are on sale, those prices often rebound.

Finding good quality stocks at attractive prices is a strategy used by some of the world's most successful investors. If you want to find more shares that meet these rules, you can see a comprehensive list on Stockopedia's StockRanks page.

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Two reasons why the market is missing a trick with CF Industries Holdings Inc - Stockopedia

KITCHENER -- Can you see your house from here?

The CF Snowbirds passed over Waterloo Region last week as part of Operation Inspiration, aiming to boost morale for Canadians across the country amid the COVID-19 pandemic.

After being delayed by a day because of visibility issues, the nine-plane formation passed over the region on Sunday, leaving their signature trail as they flew wing-to-wing.

If you saw the Snowbirds, there's a chance they could see you, too: video from the cockpit appears to show the jets as they made their pass over the region.

The Snowbirds are making their way across the country. By Thursday, they were passing over parts of Saskatchewan in their cross-country tour.

The team said their priority was to fly over hospitals and also residential areas.

"We prioritized the residential areas to make sure people could stay home and continue to social distance but also hopefully get a show from us," says Cpt. Joel Wilson.

Days later, the Waterloo Warbirds held their own flyovers to salute frontline workers, this time over Waterloo Region and Guelph.

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See what the CF Snowbirds saw: Video shows Waterloo Region from the cockpit - CTV News

CF Industries (CF) came out with quarterly earnings of $0.31 per share, beating the Zacks Consensus Estimate of $0.23 per share. This compares to earnings of $0.27 per share a year ago. These figures are adjusted for non-recurring items.

This quarterly report represents an earnings surprise of 34.78%. A quarter ago, it was expected that this fertilizer maker would post earnings of $0.26 per share when it actually produced earnings of $0.25, delivering a surprise of -3.85%.

Over the last four quarters, the company has surpassed consensus EPS estimates two times.

CF, which belongs to the Zacks Fertilizers industry, posted revenues of $971 million for the quarter ended March 2020, surpassing the Zacks Consensus Estimate by 8.95%. This compares to year-ago revenues of $1 billion. The company has topped consensus revenue estimates two times over the last four quarters.

The sustainability of the stock's immediate price movement based on the recently-released numbers and future earnings expectations will mostly depend on management's commentary on the earnings call.

CF shares have lost about 42.8% since the beginning of the year versus the S&P 500's decline of -11.2%.

What's Next for CF?

While CF has underperformed the market so far this year, the question that comes to investors' minds is: what's next for the stock?

There are no easy answers to this key question, but one reliable measure that can help investors address this is the company's earnings outlook. Not only does this include current consensus earnings expectations for the coming quarter(s), but also how these expectations have changed lately.

Empirical research shows a strong correlation between near-term stock movements and trends in earnings estimate revisions. Investors can track such revisions by themselves or rely on a tried-and-tested rating tool like the Zacks Rank, which has an impressive track record of harnessing the power of earnings estimate revisions.

Ahead of this earnings release, the estimate revisions trend for CF was unfavorable. While the magnitude and direction of estimate revisions could change following the company's just-released earnings report, the current status translates into a Zacks Rank #4 (Sell) for the stock. So, the shares are expected to underperform the market in the near future. You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here.

It will be interesting to see how estimates for the coming quarters and current fiscal year change in the days ahead. The current consensus EPS estimate is $0.70 on $1.22 billion in revenues for the coming quarter and $1.62 on $4.21 billion in revenues for the current fiscal year.

Investors should be mindful of the fact that the outlook for the industry can have a material impact on the performance of the stock as well. In terms of the Zacks Industry Rank, Fertilizers is currently in the top 10% of the 250 plus Zacks industries. Our research shows that the top 50% of the Zacks-ranked industries outperform the bottom 50% by a factor of more than 2 to 1.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free reportCF Industries Holdings Inc (CF) : Free Stock Analysis ReportTo read this article on Zacks.com click here.

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CF Industries (CF) Tops Q1 Earnings and Revenue Estimates - Yahoo Finance

A Co Down mum has welcomed positive movement on the first and only approved medicine in Europe that can treat the underlying cause of Cystic Fibrosis in some patients.

Jen Banks, whose tot Lorcn Maguire suffers from the genetic condition, says the news has brought precious hope at a time when CF patients are shielding from Coronavirus.

Vertex Pharmaceuticals said on Friday, Kalydeco (ivacaftor) was given a green light by the European Medicines Agencys Committee for Medicinal Products for Human Use for babies as young as six months.

Jen told Belfast Live: I think its wonderful!

The earlier people with CF begin to take modulators the better!

As CF is progressive these drugs will help to slow down the irreparable damage that it causes throughout the body.

The world is changing so much for everyone at the moment and for those with CF these life saving drugs have never been more important.

People with CF in the UK are having to follow shielding measures as they are regarded as being at extremely high risk of serious illness from Covid-19, anything that can help them should be welcomed with open arms.

It must be so wonderful for parents of children newly diagnosed with the R117H variation to hear that they need only wait six months before their babies can have Kalydeco.

The positive opinion on the medicine, which treats CF patients with the R117H mutation, will now be considered for approval by the European Commission.

If it gets the go ahead, Vertex says around 290 eligible patients across the UK will have access to Kalydeco within weeks of marketing authorisation as it will be reimbursed through the various agreements signed between them and UK health authorities in late 2019.

Carmen Bozic, MD, Executive Vice President and Chief Medical Officer at Vertex, said: This milestone also brings us one step closer to achieving our ultimate goal of bringing medicines forward to all people with CF.

Jen, whose three-year-old, takes Orkambi which she says has made a massive difference to Lorcns energy, appetite, weight gain, and digestion and he is a much happier wee man, even with all the treatment for the pseudomonas infection he smiles and laughs so much more and even sleeps better.

She is however eagerly waiting for Trikafta and added the 6-11 trials are currently running in spite of Covid-19, which is wonderful, and hopefully will be quickly followed by 2-5 trials.

There are still so many people with CF waiting for a drug that can help them, she continued.

In ROI there is a deal in place so that as soon as the EMA licenses the newest drug in the pipeline, Trikafta, everyone who is eligible (around 90% of people with CF) over the age of 12 will have access.

We have nothing like this in place for Trikafta and I would urge the NI Executive, the HSCB, and the drug company Vertex to work quickly to put a similar deal in place as soon as, if not before, Trikafta is approved by NICE.

In these uncertain times hope is the most precious thing weve got and should not be underestimated.

Its with delight that I think of the hope this news (about Kalydeco) will bring to families across NI.

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Co Down mum of tot with Cystic Fibrosis 'delighted' with first European treatment drug Kalydeco - Belfast Live

YARMOUTH, N.S.

For Tim Vallillee, music is medicine.

Something he can reach out to when hes hurting and when hes not. Something he can tap into for his physical health and his soul.

Something, most importantly,he can share with others.

Ive always felt that the fact I sing so much has been added therapy for me, says Vallillee, 52, who has cystic fibrosis.

Vallillee grew up in Yarmouth and lives in the Valley with his wife Agatha and son Isaiah. When COVID-19 came into play, he lost his gigs as venues were no longer open to the public.

When theyre taken away your musical brain says, this really sucks. But my CF brain says I cant do my thing, how am I going to tell how Im feeling?

Singing is a barometer that I use for the level of my health, he explains, likening it to an early warning device that lets him know how hes feeling inside especially when hes reaching for those high notes.

Ive got a pretty powerful voice but you cant do that without air in your lungs, he says. If that air is not in there it shows in my voice. It can be very subtle, but I notice it. I know something is brewing.

But where theres a will, theres a way.

Vallillee has been performing and sharing music Thursday evenings via Facebook live on his personal social media page. For his first session he put his iPhone in a tripod, sat on his bed and played for a couple of hours. Its now a highlight of his week.

Its great because I get to do what I like to do and its all part of my life therapy to have music in my life.

And hes happy to have that connection with others again.

In addition to Facebook you can also hear his music on YouTube. He also has a website to connect with others http://www.timothyv.ca.

Vallillee learned to play the guitar when he was 18. But it was something that happened when he was 16 that chartered his musical course.

My dad had a massive heart attack and when he was in the hospital . . . I promised my dad that if he wouldnt die I would learn to play an instrument, he says. He always wanted one of his four kids to learn an instrument. He always joked the best thing we could play was the record player.

His father recovered and a couple of years later reminded Vallillee of his promise. So the son grabbed a guitar and taught himself to play.

He ended up playing in many bands with friends. One of his first gigs was a variety show during Yarmouth high schools winter carnival in the 1980s.

Vallillee turns to music to express himself when hes feeling happy, sad, joyful, fearful sometimes bored and, more recently, heartbroken. His most recent original song is called Home To Me.

Its a song he wrote about what a special place Nova Scotia is. He wrote the song following the April mass shooting tragedy in the province. In the lyrics he picked out places and experiences in Nova Scotiathat he connects with and hopes others will as well.

I tried to encompass as many people as I could from Yarmouth to Cape Breton, he says.

Vallillee didnt want the song to just be about healing right now but a song people can listen to at any point in their life, whether next week, next month, or next year, and it will make them feel good.

Folks from this part of the country, are different from all the rest. We've got a special something deep down in our chest.

Nova Scotia is where I roam...Nova Scotia, that's my home. Nova Scotia is where I'll be ... Nova Scotia, is home to me.

Vallillee'swife, Agatha Bourassa, says not only is music where her husband shines, its a gift he gives to others.

This great song idea came about like every one of Tims songs have over the past two decades. Nobody really knows how he writes but he sparks an idea. Sometimes he pulls off on the side of the road and all of a sudden he regurgitates a song, she says. We just lived through this horrific tragedy in Nova Scotia and maybe no one feels like celebrating but Tim truly wrote that song to try and uplift every Nova Scotian. Lets celebrate life because every breath is worth it.

Indeed. Breaths are something those with cystic fibrosis cant take for granted. Which makes life challenging during the COVID-19 pandemic. People with immune deficiencies and underlying health issues are especially at risk.

Still, Vallillee isnt doing anything drastically different now than he does in normal times. Thats because, in a sense, hes always living in a COVID reality.

I used to say Im one bug away from getting sick and ending up in the hospital and dying in a week Ive always lived that perspective, he says, adding those with CF are very used to avoiding germs.

He calls himself a germ ninja.

Its not like I normally go around in a mask, but I will in a hospital. And Im vigilantabout always trying to wash my hands and avoid germs.

My wife, God love her, if she hears anybody coughing or sneezing in my direction even if were sitting a movie theatre and she hears somebody cough behind or near us its like weve got to move, he says. Shes seen me knock on deaths door quite a number of times. Thankfully we survived that.

Fortunately for Vallillee and his family, his health has been good in recent years. He hasnt been in the hospital in abouteight years and he attributes this to Kalydeco, a new medication he received six years ago. Its been a game-changer.

It basically goes in and changes CF genes inside my body on a genetic level. Because of that my lung function jumped about 20 per cent when I first got on it, he says. It virtually saved my life. I havent been in the hospital since. Ive had a couple of rounds of being sick, but never to the point that I was hospitalized.

The health of others with cystic fibrosis is also never far from Vallillees thoughts. Hes been promoting an online House of Commons petition aimed at helping Canadians with cystic fibrosis, cancer and other life-threatening diseases to have access to medicines and clinical trials that could save, prolong and/or improve their lives.

And then there is fundraising that has always been a special cause for Vallillees family, especially during May, which is Cystic Fibrosis Month.

COVID-19 will have an impact in terms of preventing people from coming together physically in large numbers to fundraise. In some areas that held traditional walks each May, virtual walks are being held instead.

In other words, COVID wont silence efforts in more ways than one.

On Saturday, May 16, from noonto 12 a.m. he and others will take part in an event called the 12-Hour Sing-A-Thon for Cystic Fibrosis via Facebook live. Darrin Harvey of 89.3 K-Rock will the celebrity host for the kickoff that day and Vallillee and Eben Higgins (both living with CF) will perform and also present numerous East Coast musicians from the region. The goal is to entertain, raise awareness and fundraise. Information about 12-Hour Sing-A-Thon for Cystic Fibrosis can be found on Facebook.

Vallillee is the second oldest person that he knows of in Nova Scotia living with cystic fibrosis.

Every cystic has this timeline, but we dont know what the timeline is, its all dependent on how healthy we are, if we take care of ourselves, if we get exposed to some bug, he says.

But Vallillee doesnt focus on getting sick. Instead, his focus is always on staying well.

And thats where his songs help.

Its the medicine of music, he says.

And he hopes its contagious.

Cystic fibrosis (CF) is the most common fatal genetic disease affecting Canadian children and young adults. At present, there is no cure.

CF causes various effects on the body, but mainly affects the digestive system and lungs. The degree of CF severity differs from person to person, however, the persistence and ongoing infection in the lungs, with the destruction of lungs and loss of lung function, will eventually lead to death in the majority of people with CF.

Typical complications caused by cystic fibrosis are:

It is estimatedone in every 3,600 children born in Canada has CF. More than 4,300 Canadian children, adolescents, and adults with cystic fibrosis attend specialized CF clinics.

Visit the Cystic Fibrosis Canada website to learn more and to see how you can donate.

You can also visit the Cystic Fibrosis Atlantic Canada website

CF is a multi-system disorder that produces a variety of symptoms including:

CF is a genetic disease that occurs when a child inherits two defective copies of the gene responsible for cystic fibrosis, one from each parent. Approximately, one in 25 Canadians carry one defective copy of the CF gene. Carriers do not have CF, nor do they exhibit any of the related symptoms.

When two CF carriers have a child, there is a 25 percent chance that the child will be born with CF. There is also a 50 per cent chance that the child will be a carrier, and a 25 per cent chance that the child will not be a carrier, nor have CF.

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The medicine of music: Tim Vallillee turns to song as therapy for cystic fibrosis and for life - TheChronicleHerald.ca

In August 1989, scientists made a blockbuster discovery: They pinpointed the faulty gene that causes cystic fibrosis, a cruel lung disease that killed many of its victims before they reached adulthood.

The human genome was uncharted territory, and the gene hunt had become an all-out international race, with laboratories in three countries searching for the root of the disease.

That fall, biologist James Wilson stood before an audience of researchers, physicians and cystic fibrosis patients and their families and described gene therapy, a way to replace the faulty gene with a good copy. Wilson had intended his talk to be technical and prophetic, but he was overwhelmed by the surging thrill in the room that science was about to save peoples lives.

It was one of the most amazing experiences that Ive ever had, Wilson said, adding, The expectations were through the roof.

The importance of the cystic fibrosis gene discovery went far beyond a single illness. It helped build the case for the $3 billion project to sequence the entire human genome, which would alter understanding of human biology and shed light on rare and common diseases.

But the story of cystic fibrosis has been illustrative in a way that no one could have anticipated back then. In the early days of human genetics, the path seemed straightforward: Find the gene, fix the gene and repeat for other diseases. The cystic fibrosis journey, from an exuberant moment of insight to a major success, would take 30 years of persistent, methodical work: a feat of science, business, fundraising and patience that has become a model for other diseases.

I specifically remember sitting with my doctor in the exam room, having the conversation that the gene was discovered, said Josh Taylor, 48, of Virginia Beach, who has cystic fibrosis. And him telling me the cure is just he literally said, In 5 to 10 years, were going to beat this.

It was not until late 2019 that another breakthrough fulfilled many of the hopes of 1989. Now, Taylor has what he has been waiting for all these decades a new drug, Trikafta, that is effective for 90 percent of patients. Doctors marvel at what they think will be possible if it is given at an early age: a full life span.

Cystic fibrosis developed when a child had the bad luck to inherit two faulty genes, one from each parent. Back then, there was no test to detect whether a parent carried a defective gene because no one even knew what the gene was.

As scientists developed new tools to probe human genetics, cystic fibrosis quickly became one of the top targets. It is the most common inherited disease among Caucasians, afflicting 30,000 Americans, and its motivated patient group spurred the work forward with funding.

All these human disease genes were floating around. We knew they were inherited, but we knew very little. We didnt know what the genes were, or where they were located, said Robert Nussbaum, a medical geneticist who was hunting genes for other diseases.

Francis Collins, now director of the National Institutes of Health and then a scientist at the University of Michigan working on cystic fibrosis, was photographed for the universitys graduates magazine sitting in a haystack holding a needle, to convey the magnitude of the technical challenge.

Almost everybody knew some family where it had happened, and it was heartbreaking to see what these kids go through, Collins said.

Robert Beall, then an executive vice president at the Cystic Fibrosis Foundation, which was funding the work, was also the most impatient human being I ever met to his credit, Collins said.

Collins partnered with biologist Lap-Chee Tsui, in Toronto holding joint lab meetings at a midway point on the long drive, in London, Ontario.

After years of work, Tsuis lab had narrowed the search to ever smaller stretches of DNA, pioneering new techniques in the search for the gene. Collins had invented a method to speed up the process called chromosome jumping, which allowed scientists to leap over sections of DNA something he compares to leaping from one street corner to the next to initiate searches. Jack Riordan, another scientist in Toronto, discovered a bit of DNA that looked like it might be a part of the gene, providing an essential lead.

In May, a scientist in Tsuis lab found a tantalizing clue three missing letters of DNA in a patient with cystic fibrosis. The team would need to confirm that this genetic mutation was the cause of the disease. Collins and Tsui were at a scientific conference at New Haven, Conn., a month later when they got more evidence.

One rainy night after the days program was over, the pair raced to Tsuis room, where he had installed a portable fax machine to receive updates from the lab. Among the papers that had spilled onto the floor was a table showing those three letters of DNA missing in multiple patients with cystic fibrosis, while they were present in healthy people.

Lap-Chee was a little more skeptical, Ive got to see more data, Collins recalled. I bought it, that was it. I wanted to scream and jump up and down.

The news report triggered frantic preparations to present the findings officially, and the work was published in Science magazine that September in three papers.

Collins would testify before Congress that it was necessary to fund the human genome project because the flat-out effort to find the cystic fibrosis gene simply would not be scalable in trying to understand thousands of other diseases.

Gene therapy, the thinking went, would soon cure cystic fibrosis, marking a turning point in the treatment of genetic diseases. The idea was relatively straightforward: Use a virus to ferry a good, functioning copy of the gene into patients lung cells.

But human biology turned out to have all sorts of ways of resisting an easy fix, and it quickly became clear that gene therapy would not be simple in real lungs.

Then the entire gene therapy field screeched halted in 1999 with the death of Jesse Gelsinger, a teenager with a metabolic disorder who died after being treated for the disorder in one of Wilsons gene therapy trials.

As the hope for a high-profile gene therapy success crashed, research continued on the basic, less glamorous work to untangle what went wrong with the cystic fibrosis gene. That understanding made it possible to develop ways to screen chemicals, to see if any showed promise as a drug.

Beall and Preston Campbell of the Cystic Fibrosis Foundation visited Aurora Biosciences, a San Diego biotech company that used robotics to massively speed up such testing.

Bob and I were like kids in a candy shop, Campbell recalled. After a small initial investment, the foundation stunned the nonprofit world in 2000 by awarding the company $40 million, a new kind of venture philanthropy arrangement in which if the company was successful, the nonprofit group would receive a share of the royalties.

A Massachusetts company, Vertex Pharmaceuticals, acquired Aurora in 2001, and although the cystic fibrosis work continued, it was considered a long shot, called the fantasy project internally, recalled Fred Van Goor, a scientist who joined the company around that time and became the biology lead for the cystic fibrosis program.

The scientific problem was huge: The most common gene mutation in cystic fibrosis created a protein that couldnt do its essential job in the cell. The protein didnt fold correctly, which interfered with its ability to reach the surface of the cell. And it didnt function well once there, where it was supposed to work as a gate. That meant theyd need multiple drugs to help patients one to get the protein to the right spot, the other one to open the gate.

Vertexs first drug candidate was focused on just one of the problems getting the gate to work better. Alone, it would help only about 4% of patients, whose disease was caused by a rare mutation. That drug, Kalydeco, was approved in 2012, but it remained unclear whether a drug could be made that would work for a larger group of patients.

Then, Vertexs main product a hepatitis C drug was eclipsed by a better treatment from a competitor, and the future of the company and its cystic fibrosis research was cast in doubt.

It obviously created an incredible crisis here at Vertex, said Jeff Leiden, chief executive of the company.

Vertexs board decided to bet on cystic fibrosis, and in 2015, a two-drug combination called Orkambi, was approved for a larger group of cystic fibrosis patients. Excitement about the drugs began to yield to a societal debate about their high prices; Orkambis launch price was $259,000 a year.

Meanwhile, the company would need to develop a third drug to treat more patients.

Drug trials are blinded so that neither the patients nor the scientists know which people are receiving the drug and which are receiving a placebo. When Trikafta, the triple drug combination that would ultimately be approved, was unblinded from one trial in October 2018, researchers finally saw the slide showing how the drug affected lung function.

There was a stunned silence in the room for a full minute. The drug worked.

Ten percent of cystic fibrosis patients, or about 3,000 people in the United States, are still waiting for a therapy that works for them.

Stacy Carmona, who was born just three years before the gene was discovered, is one of them.

Im so excited for the community. Im so excited for the CF friends I have who so desperately need the drug. There are so many people hanging on by a thread, waiting for this, Carmona said. The flip side of that is you cant help but wonder when is it going to be my turn?

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A cystic brosis success story -- over 30 years | Health - The Union Leader

CF Industries Holdings, Inc. (NYSE:CF) just released its quarterly report and things are looking bullish. It was overall a positive result, with revenues beating expectations by 5.0% to hit US$971m. CF Industries Holdings also reported a statutory profit of US$0.31, which was an impressive 23% above what the analysts had forecast. Following the result, the analysts have updated their earnings model, and it would be good to know whether they think there's been a strong change in the company's prospects, or if it's business as usual. With this in mind, we've gathered the latest statutory forecasts to see what the analysts are expecting for next year.

View our latest analysis for CF Industries Holdings

NYSE:CF Past and Future Earnings May 8th 2020

Following the recent earnings report, the consensus from 18 analysts covering CF Industries Holdings is for revenues of US$4.21b in 2020, implying a discernible 7.6% decline in sales compared to the last 12 months. Statutory earnings per share are forecast to plummet 21% to US$1.70 in the same period. Yet prior to the latest earnings, the analysts had been anticipated revenues of US$4.19b and earnings per share (EPS) of US$1.53 in 2020. There was no real change to the revenue estimates, but the analysts do seem more bullish on earnings, given the nice increase in earnings per share expectations following these results.

The consensus price target was unchanged at US$36.76, implying that the improved earnings outlook is not expected to have a long term impact on value creation for shareholders. There's another way to think about price targets though, and that's to look at the range of price targets put forward by analysts, because a wide range of estimates could suggest a diverse view on possible outcomes for the business. The most optimistic CF Industries Holdings analyst has a price target of US$52.00 per share, while the most pessimistic values it at US$24.00. Note the wide gap in analyst price targets? This implies to us that there is a fairly broad range of possible scenarios for the underlying business.

Looking at the bigger picture now, one of the ways we can make sense of these forecasts is to see how they measure up against both past performance and industry growth estimates. These estimates imply that sales are expected to slow, with a forecast revenue decline of 7.6%, a significant reduction from annual growth of 1.6% over the last five years. Compare this with our data, which suggests that other companies in the same industry are, in aggregate, expected to see their revenue grow 4.0% next year. It's pretty clear that CF Industries Holdings' revenues are expected to perform substantially worse than the wider industry.

The biggest takeaway for us is the consensus earnings per share upgrade, which suggests a clear improvement in sentiment around CF Industries Holdings' earnings potential next year. Fortunately, the analysts also reconfirmed their revenue estimates, suggesting sales are tracking in line with expectations - although our data does suggest that CF Industries Holdings' revenues are expected to perform worse than the wider industry. The consensus price target held steady at US$36.76, with the latest estimates not enough to have an impact on their price targets.

Following on from that line of thought, we think that the long-term prospects of the business are much more relevant than next year's earnings. At Simply Wall St, we have a full range of analyst estimates for CF Industries Holdings going out to 2024, and you can see them free on our platform here..

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That said, it's still necessary to consider the ever-present spectre of investment risk. We've identified 3 warning signs with CF Industries Holdings (at least 1 which is concerning) , and understanding them should be part of your investment process.

If you spot an error that warrants correction, please contact the editor at editorial-team@simplywallst.com. This article by Simply Wall St is general in nature. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. Simply Wall St has no position in the stocks mentioned.

We aim to bring you long-term focused research analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Thank you for reading.

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Earnings Beat: CF Industries Holdings, Inc. Just Beat Analyst Forecasts, And Analysts Have Been Updating Their Models - Yahoo Finance

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Former Cards CF Jim Edmonds says he tested positive for COVID-19 - KMOV.com