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How cystic fibrosis can manifest in the gastrointestinal system

In the February 2020 issue of Contemporary Pediatrics, Drs. Anna Reed and Darla Shores provide a briefing on the care of cystic fibrosis (CF) with a focus on the gastrointestinal complications. Although CF primarily affects the lungs, it is a complex disorder that involves multiple organ systems and many aspects of patients’ lives. What the primary care provider should know Although the majority of patients with CF will be managed by specialty care, the primary care provider (PCP) is often the first line of contact for many routine concerns. When encountering a patient with CF in the primary setting, Drs. Reed and Shores indicate that there are extrapulmonary symptoms that must be considered

New X-Ray Technology For Better Management Of Lung Diseases

Australia: Researchers at Monash University have developed radical non-invasive X-ray technology to help diagnose, treat and manage respiratory lung diseases. The technology, now being commercialized by med-tech company 4Dx Limited, allows researchers to see the movement of air through the lungs in real-time, improving their capacity to assess functional airflow in both healthy and diseased lungs. The early diagnosis and ongoing monitoring of chronic lung diseases, such as chronic obstructive pulmonary disease (COPD), pulmonary fibrosis, cystic fibrosis (CF), asthma and lung cancer, is currently hampered by the inability to capture the complete spatial distribution of lung function. The tech

Noninvasive XV Technology May Lead to Faster CF Diagnosis and Better Treatment

A new non-invasive tool, called XV technology, allows professionals to visualize airflow in a living lungs and could help in the diagnosis, monitoring, and treatment of cystic fibrosis (CF) and other respiratory lung diseases, scientists report. The technology was developed by researchers at Monash University, and has been patented by the Australian-based med-tech company 4Dx Limited. The company states that XV technology showed its potential in a clinical trial in people being treated for cancer. A proof-of-concept preclinical study in mice, “Real-time in vivo imaging of regional lung function in a mouse model of cystic fibrosis on a laboratory X-ray source,” was also published in the journ

Arrevus Receives FDA Orphan Drug Designation for ARV-1801 (sodium fusidate)

Arrevus, a clinical stage biopharmaceutical company committed to the development of innovative therapies to treat orphan or serious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ARV-1801 for the treatment of cystic fibrosis (CF). Orphan Drug Designation provides for close guidance by the FDA, potentially accelerating time to marketing approval and also includes the potential for orphan drug grants, tax credits, and 7-year market exclusivity upon marketing approval. "Orphan drug status is granted to clinical stage therapies targeting conditions that affect fewer than 200,000 patients in the United States. Cystic fibrosis pati

Synspira Therapeutics, CF Foundation Agree to Support Development of SNSP003 for Malabsorption Syndr

The Cystic Fibrosis Foundation has reached an agreement with Synspira Therapeutics to support the further development of SNSP003, an enzyme replacement therapy (ERT) designed to treat malabsorption syndromes, including those affecting patients with cystic fibrosis (CF). Malabsorption syndromes are a group of disorders that develop when there is a defect in the digestion and absorption of macronutrients — such as fat, protein, and carbohydrates — due to diseases of the pancreas, liver, and gastrointestinal (GI) tract. Malabsorption syndromes, such as exocrine pancreatic insufficiency (EPI), develop early in patients with CF, and represent a significant unmet medical need in these patients. C

Desaturation during exercise is not a sufficient mechanism for prediction of osteoporosis in non-cys

Recent research has proposed an association between desaturation during a six minute walking test (6MWT) and osteoporosis in an elderly group of individuals with non-cystic fibrosis bronchiectasis. A causative pathway through activation of hypoxia-inducible factor 1-alpha (HIF-1α) has been proposed. Queries regarding the statistical approaches used are identified and discussed within this correspondence. These predominate around the use of linear regression models to predict osteoporosis in a group that is already osteoporotic, presenting with extreme values for bone mineral density (BMD). Further queries are raised regarding the HIF-1α pathway, and physical activity (PA) is proposed as an

Researchers describe unique genetic identity of primordial lung progenitors Findings may lead to bet

(Boston)--For the first time, researchers describe the genetic program behind primordial lung progenitors--embryonic cells that give rise to all the cells that form the lining of the respiratory system after birth. They believe this study has long-term implications for the treatment of diseases affecting the respiratory system, such as chronic obstructive pulmonary disease (COPD), alpha-1 antitrypsin deficiency and cystic fibrosis. Diseases affecting the lungs are not easily treatable and result in significant morbidity and mortality worldwide. Specialized stem cells with the potential to self-renew have been proposed as a critical component of tissue homeostasis for many organs, including t

Study Describes Genetic Mechanisms Behind Embryonic Lung Cells

The researchers hope the findings could one day lead to cell replacement therapies for respiratory diseases, including chronic obstructive pulmonary disease. In a recent study, researchers described genetic underpinnings of primordial lung progenitors, the embryonic cells that lead to the development of all the cells of the respiratory system lining, which develops after birth, which may have long-term implications for the treatment of diseases affecting the respiratory system, including chronic obstructive pulmonary disease (COPD), alpha-1 antitrypsin deficiency, and cystic fibrosis. The study was conducted by the Center for Regenerative Medicine (CReM) of Boston University and Boston Med

Chronic Pain, Depression, and Anxiety in the CF Community

We need to come to grips with a problem deeply affecting the cystic fibrosis community. It’s an issue that we don’t talk about much: chronic pain and its relationship to depression and anxiety. It’s a multifaceted subject: Not only are rates of depression and anxiety higher in people with cystic fibrosis, but caregivers also experience these conditions at higher than average rates. Compared to community samples, people with CF and their parent caregivers were two to three times more likely to report depression, according to a 2014 study. It seems logical that people with chronic diseases are more likely to be depressed or anxious. People with CF are sick more often, we’re in pain much of the

Allergan Announces Agreement to Divest Zenpep, Exocrine Pancreatic Insufficiency Treatment

Allergan has entered into a definite agreement to sell Zenpep (pancrelipase), a therapy for exocrine pancreatic insufficiency due to cystic fibrosis (CF) and other conditions. The agreement was reached alongside the currently ongoing regulatory approval process for AbbVie’s acquisition of Allergan, and is contingent upon approval of the merger. Allergan also will divest brazikumab, an IL-23 inhibitor. CF can affect the pancreas, leading to exocrine pancreatic insufficiency — a lack of pancreatic enzymes that help break down food during digestion. With that condition, CF patients can experience difficulties in properly digesting food, particularly fats and proteins. Zenpep is a type of pancre

Are intestinal organoids the key to developing personalized therapy for cystic fibrosis?

Intestinal organoids have been utilized to develop personalized therapy for cystic fibrosis patients. Cystic fibrosis (CF) is a rare genetic disorder caused by a mutation in the CFTR gene. CFTR protein modulator drugs, which are effective in slowing disease progression, have been approved but only for a limited set of genotypes. With more than 2000 different CFTR mutations identified, these mutation specific therapies result in at least 10% of CF patients having no approved modulator drug. Now, the clinical-stage biopharmaceutical company Proteostasis Therapeutics, Inc. (MA, USA) have announced the results of their ex vivo study, testing CFTR modulators in intestinal organoids of CF patients

New treatment kills off infection that can be deadly to cystic fibrosis patients

Credit: CC0 Public Domain A new treatment developed by researchers at Aston University and Birmingham Children's Hospital has been found to completely kill a bacterial infection that can be deadly to cystic fibrosis patients and other chronic lung conditions such as bronchiectasis. The findings, which are published in the journal Scientific Reports, show that scientists from Aston University, Mycobacterial Research Group, combined doses of three antibiotics—amoxicillin and imipenem-relebactam and found it was 100% effective in killing off the infection which is usually extremely difficult to treat in patients with cystic fibrosis. The infection results in severe decline in lung function and

Proteostasis On Track to Develop Organoids for Personalized Medicine Strategy for CF Patients with R

A project aimed at personalized medicine based on the use of organoids, or “mini-organs,” from cystic fibrosis (CF) patients with rare mutations is on track to collect samples from 500 people with CF by the end of April, Proteostasis Therapeutics announced. The organoids will be used as a platform for developing personalized medications, and are expected to help determine patient responses to Proteostasis’ CFTR modulators. The responses will then be confirmed in a Phase 3 trial called CHOICES, the researchers said. The trial update was shared in a poster, titled “Intestinal Organoid Models as a Path for Personalized Therapy Development in Cystic Fibrosis,” at the Keystone Symposia on Tissue

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