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Mashup Score: 0Cystic Fibrosis 2023 - 8 month(s) ago
Cystic fibrosis is an autosomal recessive disorder caused by pathogenic variants of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The disease is thought to affect every organ system, with prominent pathology in the respiratory, gastrointestinal, endocrine, and reproductive systems. The development of modulators to restore CFTR function has led to substantial improvements in the health of many people with cystic fibrosis and transformed the landscape of research and clinical care.
Source: www.thelancet.comCategories: Latest Headlines, PayerTweet
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Mashup Score: 0The future of cystic fibrosis treatment: from disease mechanisms to novel therapeutic approaches - 8 month(s) ago
With the 2019 breakthrough in the development of highly effective modulator therapy providing unprecedented clinical benefits for over 90% of patients with cystic fibrosis who are genetically eligible for treatment, this rare disease has become a front runner of transformative molecular therapy. This success is based on fundamental research, which led to the identification of the disease-causing CFTR gene and our subsequent understanding of the disease mechanisms underlying the pathogenesis of cystic fibrosis, working together with a continuously evolving clinical research and drug development pipeline.
Source: www.thelancet.comCategories: Latest Headlines, PulmonologyTweet-
@TheLancet Simon Graeber and Marcus Mall focus on the mechanisms of #CFTR dysfunction in #cysticfibrosis, describing novel strategies for symptomatic therapy and promising approaches to the restoration of CFTR function https://t.co/UKZwt7nONm https://t.co/Zczz3RpAAu
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Mashup Score: 1Advancing the pipeline of cystic fibrosis clinical trials: a new roadmap with a global trial network perspective - 8 month(s) ago
The growing use of modulator therapies aimed at restoring cystic fibrosis transmembrane conductance regulator (CFTR) protein function in people with cystic fibrosis has fundamentally altered clinical trial strategies needed to advance new therapeutics across an orphan disease population that is now divided by CFTR modulator eligibility. The development of a robust pipeline of nucleic acid-based therapies (NABTs)—initially directed towards the estimated 10% of the cystic fibrosis population who are genetically ineligible for, or intolerant of, CFTR modulators—is dependent on the optimisation of restricted trial participant resources across multiple development programmes, a challenge that will preclude the use of gold standard placebo-controlled trials.
Source: www.thelancet.comCategories: Latest Headlines, PulmonologyTweet-
@TheLancet Nicole Mayer-Hamblett and colleagues present a new roadmap, with a global trial network perspective, for the design and conduct of #cysticfibrosis clinical trials that addresses the challenges of clinical research in the era of #CFTR #modulators https://t.co/X9JBbaXbHH https://t.co/SC3dUrnpTH
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Mashup Score: 1Are we there yet? The ongoing journey of cystic fibrosis care - 8 month(s) ago
The landscape of cystic fibrosis care has transformed since the discovery of the cystic fibrosis transmembrane regulator (CFTR) gene defect more than 30 years ago. Due to the collaborative efforts of the global cystic fibrosis community in both research and clinical care, the focus is now on disease modification with highly effective medications rather than on solely managing the downstream effects of CFTR dysfunction (ie, mucus obstruction, infection, and inflammation). A Series of four articles1–4 now provides a comprehensive review of recent advances in cystic fibrosis care and addresses the outstanding knowledge gaps and remaining therapeutic challenges.
Source: www.thelancet.comCategories: Latest Headlines, PulmonologyTweet-
@TheLancet “The remarkable transformation of #cysticfibrosis care has made this rare disease a pioneer of transformative molecular therapy.” See the linked Comment from Lucy Perrem and Felix Ratjen https://t.co/bZcwhY2yId
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Mashup Score: 5CFTR modulator therapy: transforming the landscape of clinical care in cystic fibrosis - 8 month(s) ago
Following discovery of the cystic fibrosis transmembrane conductance regulator (CFTR) gene in 1989 and subsequent elucidation of the varied CFTR protein abnormalities that result, a new era of cystic fibrosis management has emerged—one in which scientific principles translated from the bench to the bedside have enabled us to potentially treat the basic defect in the majority of children and adults with cystic fibrosis, with a resultant burgeoning adult cystic fibrosis population. However, the long-term effects of these therapies on the multiple manifestations of cystic fibrosis are still under investigation.
Source: www.thelancet.comCategories: Latest Headlines, PulmonologyTweet-
@TheLancet @JenTaylorCousar Thanks to @DrPLungResearch for his excellent presentation on the first paper in the Series at #ERSCongress this morning https://t.co/Hibwh31cSW
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@TheLancet @JenTaylorCousar and colleagues review the development of #CFTR #modulators and highlight the need to understand the long-term effects of modulator therapies and to ensure that effective therapies are available to all eligible people with #cysticfibrosis https://t.co/vit6x5sKtG https://t.co/4DHD8JlgIS
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Mashup Score: 0Understanding and addressing the needs of people with cystic fibrosis in the era of CFTR modulator therapy - 8 month(s) ago
Cystic fibrosis is a multiorgan disease caused by impaired function of the cystic fibrosis transmembrane conductance regulator (CFTR). Since the introduction of the CFTR modulator combination elexacaftor–tezacaftor–ivacaftor (ETI), which acts directly on mutant CFTR to enhance its activity, most people with cystic fibrosis (pwCF) have seen pronounced reductions in symptoms, and studies project marked increases in life expectancy for pwCF who are eligible for ETI. However, modulator therapy has not cured cystic fibrosis and the success of CFTR modulators has resulted in immediate questions about the new state of cystic fibrosis disease and clinical challenges in the care of pwCF.
Source: www.thelancet.comCategories: Latest Headlines, PulmonologyTweet-
@TheLancet Katherine Hisert and colleagues consider the needs of people with #cysticfibrosis in the era of #CFTR #modulator therapy, identify gaps in understanding, and outline priorities and opportunities for research https://t.co/f6eaDR2u69 https://t.co/hJPJTfmqJm
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Mashup Score: 6Cystic Fibrosis 2023 - 8 month(s) ago
Cystic fibrosis is an autosomal recessive disorder caused by pathogenic variants of the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The disease is thought to affect every organ system, with prominent pathology in the respiratory, gastrointestinal, endocrine, and reproductive systems. The development of modulators to restore CFTR function has led to substantial improvements in the health of many people with cystic fibrosis and transformed the landscape of research and clinical care.
Source: www.thelancet.comCategories: Latest Headlines, PulmonologyTweet-
See a new Series from @TheLancet and @LancetRespirMed on #cysticfibrosis in the era of #CFTR modulator therapy https://t.co/EzhWwBGmdd Join the #ERSCongress session based on the Series at 10:15 CEST today in person (Coral 3) or on the live platform (https://t.co/pMXQ9oXQcA) https://t.co/U7By32ARge
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Mashup Score: 14This fish delivers a nasty sting. Could it also save lives? - 8 month(s) ago
Scorpionfish protein kills drug-resistant bacteria from patients with cystic fibrosis
Source: www.science.orgCategories: Future of Medicine, Latest HeadlinesTweet-
A new study suggests the Korean rockfish possesses a protein that’s effective at fighting off bacterial infection in people with #CysticFibrosis. https://t.co/KRETro8Ewb
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Mashup Score: 0Virtual: Chronic Conditions - Book free tickets - 9 month(s) ago
Nursing in Practice 365 Chronic Conditions is a virtual event for primary care and community nurses providing free CPD and expert guidance
Source: nursinginpractice365.ukCategories: Latest Headlines, NursingTweet-
Join Paula Dyce, @LHCHFT, at the upcoming @NIP_365 #ChronicConditions virtual event on 5 September. Paula will give an overview of #cysticfibrosis focusing in particular on diabetes, the gut, pregnancy and liver disease. Find out more: https://t.co/00ypJeZAzM #nursingeducation https://t.co/TIZekG0s1H
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Mashup Score: 7How a cystic fibrosis drug given prenatally changed the lives of one Stanford Medicine family - 10 month(s) ago
Giving a new cystic fibrosis medication to a pregnant woman who carries the gene for the disease was unexpectedly beneficial for her fetus, a Stanford Medicine team found.
Source: News CenterCategories: General Medicine News, Latest HeadlinesTweet-
“We had no clue if it was actually going to do anything for us. We were just hoping.” – Michaela Zalinski, a carrier of the gene for cystic fibrosis #CysticFibrosis #Pregnancy https://t.co/TwEb6ZEWhM
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Fantastic presentations so far at #ERSCongress Great to see the new Series from @TheLancet and @LancetRespirMed on #cysticfibrosis in the era of #CFTR modulator therapy presented yesterday https://t.co/8fcXAzb6PN https://t.co/9jNQnDbtIy https://t.co/pdfuH8NznB