Category Archives: Cf

Will CF Industries Holdings, Inc. (CF) Stay at the Top of the Basic Materials Sector?  InvestorsObserver

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Will CF Industries Holdings, Inc. (CF) Stay at the Top of the Basic Materials Sector? - InvestorsObserver

Wanyama on Sunday played his last match for CF Montreal- Here are potential destinations  The Standard

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Wanyama on Sunday played his last match for CF Montreal- Here are potential destinations - The Standard

Scouting Report: 5 things you should know about CF Montral when they play NYCFC in the MLS Cup Playoffs  Hudson River Blue

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Scouting Report: 5 things you should know about CF Montral when they play NYCFC in the MLS Cup Playoffs - Hudson River Blue

Brax Pope is two years old, and thanks to new treatment options, his parents see more hope for his future.

ATLANTA Medical advancements in the treatment of cystic fibrosis have made a Brookhaven toddler's life much easier and given his parents more hope for their son's future.

Brax Pope is two years old and lives with cystic fibrosis, a chronic and rare genetic disorder that attacks the lungs, making it hard to breathe and, over time, can be life-threatening. It affects nearly 30,000 people in the US.

Through the pain, Brax is still non-stop. He runs from his dads arms to his playroom and back. His parents said he's truly changed how they view life.

He is 100-miles-per-hour. His dad, Carter Pope, said while laughing.

When Brax was a few months old, he spent 17 days at Childrens Health Care of Atlanta, but at two years old, he's happy and full of personality.

Everyone who meets him says hes a joy to be around. His mom, Jessica Pope, said. Youd have no idea he has CF.

Brax wears a vest that shakes him daily, helping clear mucus from his lungs. He's a pro at swelling pills, taking a handful a day. The Pope family is grateful for a fairly new medicine that's kept him relatively healthy since his initial hospital visit.

There is so much hope they are working towards a cure. His mom said. We know that is the difference in saving his life.

This push for a cure and the new medicine is the result of work in Atlanta decades before Brax was born. Carter and Jessica quickly point out its possible because of a local family, the Choates.

They are a family of builders and started Choate Construction in Atlanta in 1989. That year they committed to raising money for CF research in honor of Leann Rittenbaum Ott. Leann was a close family friend of the Choates and was diagnosed with CF as a toddler.

I see Leann in every CF patient, those like Leann who paved the way. Emily Choate, Leann's childhood best friend, said.

Yearly their company hosts a fundraiser and car show called 'Cars & 'Q for the Cause' with hopes that the additional funding would bring a cure. The charity event raised over $500,000 for CFin 2021.

Shortly after Leann died in 2018 at 33 years old, researchers discovered a life-changing treatment. And in October 2019, the U.S. Food and Drug Administration approved the use of the triple-combination modulator - which includes Elexacaftor, Tezacaftor, Vacaftor - or the Trikafta.

This triple-combination drug was the game changer doctors and those with CF hoped for. It's the drug helping Brax live his best life in 2022.

It took those like Leann to go first. Choate said. And while we lost Leanne right before Leann came on the market, her spirit lives on in these new CF patients.

People with cystic fibrosis ages 12 and older, who have at least one copy of the F508del mutation, can use the drug - which is 90% of all cases. The advancements in treatment are significant, but 10% of CFpatients still don't have the help they need. And the goal is still to help all and, ultimately, keep working for a cure.

The 2022 Cars & Q for the Cause fundraiser takes place on Saturday, October 22, from 3 p.m. to 6 p.m. at 8200 Roberts Drive in Atlanta. Parking is free, and shuttles from the overflow parking areas will be available.

Buy tickets early! Advanced tickets are $20 for event entry and dinner, or pay $40 for admission, dinner, and access to the bar (legal age permitting). Tickets rise to $30 and $50 on October 14 and could sell out. Children under 8 years old are free.

Continued here:

2-year-old living with cystic fibrosis helped by new treatments - 11Alive.com WXIA

Cystic fibrosis (CF) is an inherited disorder that causes severe damage to the lungs and other organs in the body. Nearly 40,000 children and adults in the United States live with CF, an often difficult existence exacerbated by an opportunistic bacterium called Pseudomonas aeruginosa, which is a major cause of chronic, life-threatening lung infections.

P. aeruginosa infections are not easily treated. The pathogen can be resistant to most current antibiotics. However, an early-stage clinical trial led by scientists at University of California San Diego School of Medicine, with collaborators across the country, has launched to assess the safety and efficacy of treating P. aeruginosa lung infections in CF patients with a different biological weapon: bacteriophages.

A false-color micrograph depicts phages attacking a bacterium. Photo courtesy of Thomas Deerinck, National Center for Microscopy and Imaging, UC San Diego.

Bacteriophages are viruses that have evolved to target and destroy specific bacterial species or strains. Phages are more abundant than all other life forms on Earth combined and are found wherever bacteria exist. Discovered in the early 20th century, they have long been investigated for their therapeutic potential, but increasingly so with the rise and spread of antibiotic-resistant bacteria.

In 2016, scientists and physicians at UC San Diego School of Medicine and UC San Diego Health used an experimental intravenous phage therapyto successfully treat and cure colleague Tom Patterson, PhD, who was near death from a multidrug-resistant bacterial infection. Pattersons was the first documented case in the U.S. to employ intravenous phages to eradicate a systemic bacterial infection. Subsequent successful cases helped lead to creation of the Center for Innovative Phage Applications and Therapeutics (IPATH) at UC San Diego, the first such center in North America.

In 2020, IPATH researchers published data from 10 cases of intravenous bacteriophage therapy to treat multidrug-resistant bacterial infections, all at UC San Diego. In 7 of 10 cases, there was a successful outcome.

The new Phase 1b/2 clinical trial advances this work. The trial is co-led by Robert Schooley, MD, professor of medicine at UC San Diego School of Medicine and an infectious disease expert at UC San Diego School Health, who is co-director of IPATH and helped lead the clinical team that treated and cured Patterson in 2016.

It will consist of three elements, all intended to assess the safety and microbiological activity of a single dose of intravenous phage therapy in males and non-pregnant females 18 years and older, all residing in the United States.

The dose is a cocktail of four phages that target P. aeruginosa, a bacterial species commonly found in the environment (soil and water) that can cause infections in the blood, lungs and other parts of the body after surgery.

For persons with CF, P. aeruginosa is a familiar and sometimes fatal foe. The Cystic Fibrosis Foundation estimates that roughly half of all people with CF are infected by Pseudomonas. Previous studies have indicated that chronic P. aeruginosa lung infections negatively impact life expectancy of CF patients, who currently live, on average, to approximately 44 years.

In the first stage of the trial, two sentinel subjects will receive one of three dosing strengths of the IV bacteriophage therapy. If, after 96 hours and no adverse effects, the second stage (2a) will enroll 32 participants into one of four arms: the three doses and a placebo.

After multiple follow-up visits over 30 days and an analysis of which dosing strength exhibited the most favorable safety and microbiologic activity, i.e. most effective at reducing P. aeruginosa, stage 2b will recruit up to 72 participants to either receive that IV dose or a placebo.

Robert Schooley, MD, is co-principal investigator of the bacteriophage/cystic fibrosis clinical trial and professor of medicine at UC San Diego School of Medicine and an infectious disease expert at UC San Diego Health.

Enrollment will occur at 16 cystic fibrosis clinical research sites in the United States, including UC San Diego. It is randomized, double-blind and placebo-controlled. The trial is being conducted through the Antibacterial Resistance Leadership Group and funded by the National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, with additional support for the UC San Diego trial site from the Mallory Smith Legacy Fund.

Mallory Smith was born with cystic fibrosis and died in 2017 at the age of 25 from a multidrug-resistant bacterial infection following a double lung transplant.

Mallory's death was a preventable tragedy, said her mother, Diane Shader Smith. We are supporting the IPATH trial through Mallory's Legacy Fund because Mark and I deeply believe in the promise of phage therapy to save lives by combatting multidrug-resistant bacteria.

In an article published in 2020 in Nature Microbiology, Schooley and Steffanie Strathdee, PhD, associate dean of global health sciences, Harold Simon Professor in the Department of Medicine and IPATH co-director, describe phages as living antibiotics.

As such, said Schooley, researchers need to learn how to best use them to benefit patients through the same systematic clinical trials employed to evaluate traditional antibiotics.

The primary objectives of the new trial are first to determine the safety of a single IV phage dose in clinically stable patients with CF who are also infected with P. aeruginosa, said Schooley.

Second, its to describe the microbiological activity of a single IV dose and third, to assess the benefit-to-risk profile for CF patients with P. aeruginosa infections. This is one study, with a distinct patient cohort and carefully prescribed goals. Its a step, but an important one that can, if ultimately proven successful, help address the growing, global problem of antimicrobial resistance and measurably improve patients lives.

Estimated study completion date is early 2025.

For more information on the clinical trial and participant eligibility criteria, visit clinicaltrials.gov or visit IPATH and click on the Contact Us button.

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Phage Trial to Treat CF Patients with Multi-Drug Resistant Bacterial Infections - UC San Diego Health

High blood-sugar levels at night are associated with poorer lung function in adults, but not in children, with cystic fibrosis (CF), a study has found.

During a week of continuousglucose monitoring, blood-sugar abnormalitieswere observed in almost all CF adults and children evaluated, even when other tests taken over the same period returned normal results.

To our knowledge, this is the first study reporting glycemic profiles during day and night in children and adults with CF without previous diagnosis of [CF-related diabetes], the researchers wrote. Our results support continued screening for glucose intolerance in patients with CF.

The study, Glycemic indices at night measured by CGM are predictive for a lower pulmonary function in adults but not in children with cystic fibrosis, was published in the Journal of Cystic Fibrosis.

Abnormalities in glucose metabolism are common in CF, occurring in about 40% of infants and children with this disease, and about half go on to developCF-related diabetes (CFRD) in adulthood.

Notably, alterations in glucose metabolism are reported to associate with declines in lung function and nutritional status. Care guidelines recommend that CF patients be screened for glucose abnormalities starting at age 10.

Continuous glucose monitoring (CGM), which automatically tracks blood-sugar levels throughout the day or night, is one way of detecting these changes, but test parameters that might be most useful are not well established.

Researchers in Belgium used CGM to evaluate blood-sugar patterns in 47 children and adults with CF but not a CFRD diagnosis, although all were considered at risk for disease-related diabetes. None were usingCFTR modulators, considered a highly effective treatment.

These patients 21 adults and 26 minors, all being followed at Ghent University Hospitals cystic fibrosis center underwent continuous glucose monitoring there between January 2016 and June 2019. Clinical data from the their medical records in the year before and after the CGM were also evaluated.

Per the centers protocol, patients were offered CGM if they showed abnormalities on an oral glucose tolerance test done to compare fasting blood-sugar levels with those shortly after consuming sugar, often as a syrupy glucose solution or had an elevated HbA1C value, a measure of a persons average blood-sugar level in the last three months.

About half of the 25 patients given an oral glucose tolerance test at the time of CGM showed normal glucose levels.

For continuous glucose monitoring, a sensor was inserted into patients abdominal fat for seven days;data from the device were downloaded and analyzed. Patients also measured their own blood-sugar levels twice a day.

Normal CGM results were defined as blood-sugar levels greater than 140 milligrams per deciliter (mg/dL) for less than 4.5% of the time, and no reading at or above 200 mg/dL.

Both children and adults showed a deviant glycemic profile on CGM, the researchers wrote, with 96% of patients (45 of 47 people) having an abnormal result.

Specifically, more than half of the patients (27 or 57.4%)did not reach a normal glucose range during the day (70140 mg/dL) for at least 90% of the time, and 87.2% (41 people) didnt reach it at night.

Among children, most measures of glucose exposure were higher during the day than at night. Among adults, none of these measures differed significantly by time of day.

Lung function was monitored with the forced expiratory volume in one second (FEV1), a measure of how much air can be forcibly exhaled in a single breath.A decline in lung function was observed between the year before and after CGM in children, whereas no significant changes during the study were seen in adults.

Lung function at the time of CGM wasnt associated with any glucose measurements in children. But for adults, indicators of higher blood nighttime glucose levels linked with worse lung function.

Specifically, for every 1% increase in the amount of time spent with blood sugar levels over 140 mg/dL at night, a 0.77% decline in FEV1 values was observed.

In the adult cohort an association was foundbetween the level of[glucose expoure]at night and lower concurrent pulmonary function, the researchers wrote.

CGM parameters were not associated with nutritional status, as measured by the body mass index, in children or adults.

High blood-sugar levels, particularly at night, in CF patients could be in part due to their diet, the researchers suggested. To support a healthy nutritional status, patients are often advised to increase their intake of carbohydrates, fats, and proteins (macronutrients), with many patients eating a snack before bed.

It is justified to suggest more research on the impact of macronutrient composition of foods on glucose control in CF patients, the researchers wrote, noting that their findings challenge the nutritional intervention of adding a snack before bedtime to the meal plan of CF patients.

This study showed abnormalities of glucose homeostasis in almost all selected CF patients, despite normal readings on tests of oral glucose tolerance and HbA1C values, the researchers wrote.

Further research should focus on finding thresholds for specific CF outcomes related to age, they added.

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High Blood-sugar Levels at Night Affect Lung Function in CF Adults... - Cystic Fibrosis News Today

Kelly Marchand of London, Ont., is thankful her seven-year-old daughter Charlotte, who has cystic fibrosis, is finally taking a prescription medication that some doctors describe as life changing.

"It brought me to tears," Marchand told CBC News. "It means so much for my daughter to be able to have this medication, to hopefully slow down her disease progression and cause fewerissues."

CF is a hereditary disease that affects the cells that produce mucus, and can lead to a buildup inthe lungs, digestive tractand other organs.It can make the body prone to lung infections and often, lung transplants are required.

In July, Ontario made Trikaftaavailable to children six years old and up. The triple combination therapy works to correct the defect of the gene thatcauses CF.

According to Cystic Fibrosis Canada, anestimated one in every 3,600 children born in this countryhas CF. More than 4,300 children, adolescents, and adults with cystic fibrosis attend specialized CF clinics, according to the non-profit organization.

"This is the most promising drug for the underlying treatment of CF," said Marchand, who was able to use private insurance coverage from her job to secure Trikafta.

Charlottestarted Trikaftalast week and is still getting used to having the drug as part of her daily routine.

Butmany families in Ontariostruggle to navigate insurance coverage to affordthe drug, which costs about$300,000 a year for a single patient.

Trikaftacan be covered by the Ontario Drug Benefit(ODB),available through theExceptional Access Program (EAP).

Individuals 24 and younger areeligible to get the medication through OHIP+ as long as they don't have private insurance. People between 25 and 64canenrol in the Trillium Drug Program if they have a high drug cost relative to their incomethere's alsoa deductible and co-payment.

However,Kim Steele of Cystic Fibrosis Canada saidthis system doesn't give Ontarians the safety net to secure full coverage because if theirprivate insurance doesn't pay, they may have to be responsible for a deductible for both sectors.

"If you have private insurance, you're excluded from that program [OHIP+] altogether, even if yourinsurance won't cover the full cost of the drug," Steele said.

"It's really hitting a lot of average middle-class families and putting them in awkward positions where they or their children might not be able to get the drug unless they remove themselves from their plans, losing benefits like dental and others."

Each provincehas its own health-care model, which prevents a co-ordinated, Canada-wide approach to accessing medicine like Trikafta, Steele said.

Dr. April Price, director of the pediatric cystic fibrosis clinic at the London Health Sciences Centre, describesTrikaftaas a "game changer" in her patients' lives.

"What this drug has been able to dois really something from a science fiction moviebrought to life," she said. "Things start to move which had been stuckfor a long time, so cough andincreased mucus production are some of the known results of starting the drug."

But Price acknowledgedits high cost prohibits many patients from accessing it, especially when they don't fit criteria of provincial eligibility,she said.

"As soon as you stop taking this drug, all its effects wear off, so there's going to be an ongoing need for every patient in Canada, which there are over 4,000 per year," Price said.

Canada needs a rare disease strategy to allowprovinces and territories to work together to create a united framework in order to reduce barriers in accessibility, Steele said. In November,Cystic FibrosisCanada will hold a rally at Queen's Park in Toronto to advocate for easier access to the drug.

"Something has to change because it's not fairthat families have to struggle so much to get this drug for their children in Ontario," Steelesaid.

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This 7-year-old is finally on a 'life-changing' cystic fibrosis drug. For others in Ontario, access is tricky - CBC.ca

Last Friday night's game was do-or-die time for the Camden Fairview Cardinals as they went on the road to take on the Magnolia Panthers. The winner of the game would take full control of the 5A South Conference lead and be one step closer to securing themselves as one of the top teams in the state of Arkansas.

Through a tough battle at the line of scrimmage, the Cardinals prevailed, winning on the road, 35-19.

For a while, it didn't look like the Cardinals were going to be victorious as they found themselves down 12-0 in the first quarter. Faced with pressure, the Cardinals decided to make changes that would ultimately help them win this big game.

Offensively, the Cardinals' went away from their high-caliber passing game to focus on their dynamic RPO running game. Several players helped create a high-powered rushing performance, but it was senior quarterback Martavius Thomas that led the Cardinals to a huge second half performance.

It was an even bigger game for the Cardinals' defense, as they had to lock down one of the best running backs in the state in Magnolia's Garrion Curry. Magnolia's offense primarily runs the ball up the middle in a tight RPO-style, with Curry receiving the bulk of the carries.

In the beginning of the game, Curry had some big moments, but the defense of Camden Fairview played stout at the line of scrimmage and made big plays in the backfield when needed. Many defenders can take credit for the big game, but it was senior defensive end Kameron Easttman that led the Cardinals from start to finish with an incredible performance at a stout rush defense.

Martavius Thomas is the Camden News' offensive player of the week and Kameron Easttam is the Camden News' defensive player of the week.

The Cardinals' offense knew they would have problems trying to establish a solid passing game due to the tenacious blitzing of the Panthers and the secondary play. Instead of trying to force the passing offense into the rhythm, the Cardinals, led by Thomas, decided to beat the Panthers in their own game. The defense of Magnolia was overwhelmed by the power run game of Camden Fairview with Thomas as the leading runner.

With Thomas utilizing a solid RPO-scheme, Magnolia's defenders had to take an extra amount of time to read the play in the backfield. That amount of extra time it took to diagnosis the play was too much, as Thomas' explosive speed quickly overhwhelmed the front seven defenders. Thomas had huge support at the line of scrimmage from the offensive line.

Thomas rushed for 144 yards and a touchdown on 21 carries. Most of his carries came after the Cardinals turned their their attention away from the play-schemes for the rest of the game. Thomas pushed the ball down the field with his legs, with several of his yards coming after the first contact with defenders. He also set up multiple packages that had junior defensive back Jabauree Lockhart accounting for three rushing touchdowns for four total yards.

On the defensive side of the ball, the Cardinals had one of their best performances of the season -- not statistically, but performance-wise. Camden Fairview held Magnolia's high-powered and aggressive rushing offense to 301 rushing yards on 45 carries for an average of 6.7 yards per carry. One of the biggest game elements that head coach Nick Vaughn said would be key were the play-action passes called. The Cardinals only allowed 2-of-10 completed passes throughout the entire game, with one of them coming in a great play from Curry to a receiver.

The defense of Camden Fairview accounted for nine tackles for loss, 12 quarterback hurries and two sacks. The entire defense and all position groups had huge performances, but it was no question that Easttam had one of the biggest performances from start to finish. Easttam accounted for ten total tackles, four solo tackles, three tackles for loss, five quarterback pressures and one sack.

Easttam found himself playing multiple different roles in the game against Magnolia. On top of his typical duties of playing defensive end in certain schemes, he was also placed at middle linebacker in other schemes when the defensive line needed more size. Although he's smaller than others at the position, Easttam crashed the edge hard and closed off running lanes. He also made great pursuit angles to tackle the ballcarrier for little-to-no gain or even negative yards.

Easttam remained consistent for Camden Fairview, providing energy and confidence in the Cardinals' defense. He showed that this Panthers' offense was vulnerable and was capable of being overpowered. Easttam was among the defenders for Camden Fairview that was a physical and psychological boost on Friday night.

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CF's Thomas and Easttam dodge Panthers' high-powered defense, tackle running offense head-on - Camden News

Im always nervous about diving back into work after being out of commission. In this case, Im referring to the monthlong hiatusI took from writing this column. I was hesitant to pause my writing for Cystic Fibrosis News Today. What if I disappoint my editors or my audience? Even worse, what if I ignore my declining health? The latter is an ongoing battle.

Fortunately, I was productive during my month off. In addition to several trips to the emergency room related to my sinuses, I watched video of the London stage production Heathers the Musical, a dark comedy that follows Veronica Sawyer through a dangerous high school romance. Ive written about the show before in a column titled Big Fun: My Make-A-Wish, but this production featured a new cast. Further, I was surprised to learn that this version featured several new songs, including I Say No.

In the song, Veronica vocalizes how unhealthy her relationship has become. From acceptance to validation, the lyrics were a great addition to the story and my audition book.

This is it/ Hit the brake/ I am finally awakeVeronica took a moment to address and accept the things that were out of her control. In my case, I must accept that breaks are necessary.

As a cystic fibrosis (CF) patient, I found that pitting my health against my happiness was an ongoing Sophies choice, so I tended to work my body into the ground before coming to a full stop. As a post-transplant patient, however, my lungs force me to rest, and I decided I must follow their lead. I dont want to put my new lungs in jeopardy, nor do I want the quality of my work to suffer.

Either way, saying no is a necessary tool during periods of infection, but pushing myself is a tough habit to break.

Call it all my mistake/ long as you let me go

As hard as it is to rest, though, its twice as hard to dive back in, for three primary reasons.

One, my mind and body grow accustomed to the out of commission lifestyle. The longer I rest, the harder it is to bounce back. Im encouraged and required to rest more than usual. My emotions and immunity become even more vulnerable. I become overwhelmed by appointments and medications, and my family must step in. Being cared for is addictive, and that addiction skews my ability to identify my bounce-back period.

Two, the fear of my full-speed life paralyzes me. The little voice inside of my head, the one that grows louder with every nap or additional antibiotic, tells me that Im powerless. My symptoms, the mindset, and the situation trick me into thinking Im incapable of diving back in. Or worse, Im convinced the act of diving in is irresponsible or unrealistic.

Three, my family asks if Ill join them for dinner or at a local festival that I enjoy and I say no. I become intimidated by my previous self, even if that self existed a short time ago. What if I cant bounce back quickly enough or cant endure? Are these thoughts pessimistic or responsible? I cant write, or walk, or be.

Ive got to kick this habit now/ or else I never will I wont cry/ starting now, I will try

In lieu of stopping my column completely, I decided to decrease the frequency of it from four times a month to just twice. Im not sure if this change is temporary or permanent, but its whats best for this stage of my life.

Veronica reflected on each step of her emotional journey, and the lyrics encouraged me to explore my own issues with stepping away versus diving back in.

Im still nervous that I have nothing good to say, that my endurance wont last. Will I be expected to keep up with the previous standards that Ive set for myself and my writing? If so, should I warn my audience that I might not be able to do so? Should I have faith that I can, or simply dive in and see what happens?

If youre also scared to dive back in, worrying youre not good enough, concerned you cant compete with your previous self, or anxious that youll run out of things to say just take that first step. Write something. Walk somewhere. Its enough.

To the situation that tells me Im a failure for slowing down; to my CF, whose statistics and symptoms twist the truth and force me to question my identity; to the feeling that I must permanently sit on the couch: I say no.

Nicole in the CarolinaEast Medical Center emergency room on Sept. 25. (Courtesy of Nicole Kohr)

Note:Cystic Fibrosis News Todayis strictly a news and information website about the disease. It does not provide medical advice,diagnosis, ortreatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those ofCystic Fibrosis News Todayor its parent company, BioNews, and are intended to spark discussion about issues pertaining to cystic fibrosis.

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The Difficulties of Diving Back In After Taking a Hiatus | Cystic... - Cystic Fibrosis News Today

The overall cystic fibrosis market is expected to surge due to the increasing prevalence of the disease and expected launch of emerging therapies including Ensifentrine, VX-121/TEZ/ VX561, and others in the forecasted period (20222032).

LAS VEGAS, Oct. 12, 2022 /PRNewswire/ --DelveInsight's Cystic Fibrosis Market Insights report includes a comprehensive understanding of current treatment practices, cystic fibrosis emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), Japan].

Key Takeaways from the Cystic Fibrosis Market Report

Discover which therapies are expected to grab the major cystic fibrosis market share @Cystic Fibrosis Market Report

Cystic Fibrosis Overview

Cystic fibrosis is a genetic disorder caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. F508del is the most common variation reported globally, but there are over 2000 others, not all causing the disease.

The cystic fibrosis symptoms usually appear 6-8 months after birth, though this varies significantly from person to person. Cystic fibrosis symptoms vary with age and can affect different body parts. Newborn screening, a genetic test to look for a gene defect, and a blood test to look for pancreatic problems are two tests used for cystic fibrosis diagnosis. After initial testing, a sweat test can be performed to confirm the cystic fibrosis diagnosis.

Cystic Fibrosis Epidemiology Segmentation

DelveInsight estimates that there were approximately 65.5K diagnosed prevalent cases of cystic fibrosis in the 7MM in 2021.

Among the EU5 countries, the UK had the highest number of cystic fibrosis diagnosed prevalent cases in 2021.

The cystic fibrosis market reportproffers epidemiological analysis for the study period 20192032 in the 7MM segmented into:

Download the report to understand which factors are driving cystic fibrosis epidemiology trends @Cystic Fibrosis Epidemiological Insights

Cystic Fibrosis Treatment Market

Current cystic fibrosis medications focus on symptom relief, complication prevention, and, more recently, protein rectifiers to correct underlying structural and functional abnormalities. Inhaled antibiotics are used to treat chronic lung infections, mucolytics are used to reduce the viscosity of pulmonary mucus, pancreatic enzyme replacement therapy (PERT) is used to treat CF-associated exocrine pancreatic insufficiency (PPI), and CFTR modulators are used to improve CFTR function while addressing the underlying cause of the disease.

In addition to mucolytics like dornase alfa, hypertonic saline inhalation has been proposed as a therapy for increasing the hydration of airway surface liquid in cystic fibrosis patients. Pancreatic enzyme supplements, multivitamins (particularly fat-soluble vitamins), mucolytics, antibiotics (including inhaled, oral, or parenteral), bronchodilators, anti-inflammatory agents, and CFTR potentiators (e.g., ivacaftor) and correctors (e.g., elexacaftor, lumacaftor,tezacaftor) may be used to treat patients with cystic fibrosis.

The current cystic fibrosis treatment is primarily based on CFTR modulators and supportive therapies such as mucolytics, antibiotics, bronchodilators, and others. TRIKAFTA, SYMDEKO, ORKAMBI, and KALYDECO currently dominate the cystic fibrosis market.

Generally, a normal diet with additional energy and unrestricted fat intake is advised. To compensate for malabsorption and the increased energy demand of chronic inflammation, a high-energy and high-fat diet is recommended, along with supplemental vitamins (especially fat-soluble) and minerals. Sometimes cystic fibrosis causes issues that can only be resolved through surgery or another type of medical procedure.

To know more about cystic fibrosis medications, visit @Cystic Fibrosis Treatment Market

Cystic Fibrosis Pipeline Therapies and Key Companies

Learn more about the FDA-approved drugs for cystic fibrosis treatment @Cystic Fibrosis Drugs

Cystic Fibrosis Market Dynamics

The overall prevalence of cystic fibrosis is expected to rise over the forecast period, creating new opportunities for pharma players in the cystic fibrosis market. Moreover, existing therapies have room for label expansions to improve efficacy, propelling the cystic fibrosis market forward.

Furthermore, there are lucrative opportunities to address unmet therapeutic needs in the cystic fibrosis market regarding tolerability, drug resistance, dosage, and adverse effects. More combinational drugs with higher efficacy are needed.

With advancements in diagnostic tools and palliative care, the median cystic fibrosis life expectancy in the United States has risen from a few months to 46 years. Several cystic fibrosis drugs are currently being used to treat cystic fibrosis, with evidence ranging from randomized, double-blind, placebo-controlled clinical trials to anecdotal case reports. Many more are currently in the works. Pharma companies have been proactive in their research in the cystic fibrosis market, recognizing that the treatment has a long way to go.

However, due to the lack of significant competitors, the current cystic fibrosis market players will likely maintain their competitive positions throughout the study period. Despite improved outcomes, most cystic fibrosis patients die from pulmonary complications, making this one of the most significant underlying challenges in the cystic fibrosis treatment paradigm.

Moreover, the patient's pharmacological treatment history is complicated, with a plethora of drugs resulting in resistance and a constant shift in treatment regimens. Any generic drug entry will impede the cystic fibrosis market for branded drugs. Even Vertex has plans for generic cystic fibrosis medications.

Report Metrics

Details

Study Period

20192032

Coverage

7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

Base Year

2021

Market CAGR

9%

Market Size in 2021

USD 7.1 Billion

Key Cystic Fibrosis Companies

Verona Pharmaceuticals, Eloxx Pharmaceuticals, Inc., Vertex Pharmaceuticals, Santhera Pharmaceuticals, Laurent Pharmaceuticals Inc., Krystal Biotech, Translate Bio, Novartis, Algi Pharma, Atlantic Healthcare, Calithera Biosciences, Horizon Therapeutics, Reveragen Biopharma, Spli Sense, GlaxoSmithKline, EmphyCorp, Galapagos NV, PathBio Analytics, AstraZeneca, AxentisPharma AG, and others

Key Cystic Fibrosis Therapies

Ensifentrine, ELX-02, VX-121/TEZ/ VX-561, POL6014 (Lonodelestat), LAU-7b, and others

Scope of the Cystic Fibrosis Market Report

Discover more about drugs for cystic fibrosis in development @Cystic Fibrosis Clinical Trials

Table of Contents

1.

Cystic Fibrosis Market Key Insights

2.

Cystic Fibrosis Market Report Introduction

3.

Cystic Fibrosis Market Overview at a Glance

4.

Cystic Fibrosis Market Executive Summary

5.

Disease Background and Overview

6.

Cystic Fibrosis Treatment and Management

7.

Cystic Fibrosis Epidemiology and Patient Population

8.

Patient Journey

9.

Cystic Fibrosis Marketed Drugs

10.

Cystic Fibrosis Emerging Drugs

11.

Seven Major Cystic Fibrosis Market Analysis

12.

Cystic Fibrosis Market Outlook

13.

Potential of Current and Emerging Therapies

14.

KOL Views

15.

Cystic Fibrosis Market Drivers

16.

Cystic Fibrosis Market Barriers

17.

Unmet Needs

18.

SWOT Analysis

19.

Appendix

20.

DelveInsight Capabilities

21.

Disclaimer

22.

About DelveInsight

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Cystic Fibrosis Market to Showcase Growth at a CAGR of 9% During the Forecast Period (2022-2032) | DelveInsight - PR Newswire