Cystic Fibrosis victory: Ayden, 13, weeps with joy as he finally gets miracle drug

BRAVE cystic fibrosis sufferer Ayden Cochrane wept with joy last night, after we helped him secure the American wonder pill that could save his life. In a dramatic change of heart, drug maker Vertex contacted the Daily Express directly, telling us Ayden, 13, will receive Trikafta in the next 24 hours.
Source: Daily Express - Health - Category: Consumer Health News Source Type: news

Related Links:

Authors: Kaur P, Kumar P, Randev S, Guglani V Abstract Allergic bronchopulmonary aspergillosis (ABPA) is an immunological disease complicating asthma or cystic fibrosis. An 8-year-old girl with no previous respiratory morbidity was diagnosed with ABPA on the basis of a raised eosinophil count and total and specific serum IgE levels combined with a positive skin-prick test for aspergillus and typical CT images of finger-in-glove hilar opacities and hyperattenuating mucous. She responded to treatment with itraconazole and corticosteroids and remains well. As far as we are aware, she is only the second child to be dia...
Source: Paediatrics and international child health - Category: Pediatrics Tags: Paediatr Int Child Health Source Type: research
Publication date: Available online 2 April 2020Source: Journal of the Academy of Nutrition and DieteticsAuthor(s): Miriam Isabel Souza dos Santos Simon, Roberta Dalle Molle, Flávia Moraes Silva, Thais Wabner Rodrigues, Marceli Feldmann, Gabriele Carra Forte, Paulo José Cauduro Marostica
Source: Journal of the Academy of Nutrition and Dietetics - Category: Nutrition Source Type: research
Learn more about the available diagnostic modalities for the identification of cystic fibrosis across all age groups.Seminars in Respiratory and Critical Care Medicine
Source: Medscape Today Headlines - Category: Consumer Health News Tags: Pulmonary Medicine Journal Article Source Type: news
PMID: 32243189 [PubMed - as supplied by publisher]
Source: American Journal of Respiratory and Critical Care Medicine - Category: Respiratory Medicine Authors: Tags: Am J Respir Crit Care Med Source Type: research
This study examined early predictors of progressive structural lung abnormalities in children who completed the Australasian CF Bronchoalveolar Lavage (ACFBAL) clinical trial at age 5-years and participated in an observational follow-up study (CF-FAB). Eight Australian and New Zealand CF centres participated in CF-FAB and provided follow-up chest computed-tomography (CT) scans for children who had completed the ACFBAL study with baseline scans at age 5-years. CT scans were annotated using PRAGMA-CF scoring. Ordinal regression analysis and linear regression were used to investigate associations between PRAGMA-CF (Perth&ndas...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: CF and non-CF bronchiectasis Original Articles: Cystic fibrosis Source Type: research
The natural history of cystic fibrosis (CF) lung disease has been described as a complex process of obstruction, infection, and inflammation, all contributing to airways injury and eventual structural disease (i.e. bronchiectasis). Consequences of the CF genetic abnormality begin in utero and are the basis for newborn screening for elevated levels of circulating immunoreactive trypsinogen (IRT) [1]. Thus, it should be no surprise that the process of airway obstruction begins immediately at birth. Ample cross-sectional evidence has been published of airway obstruction, inflammation, bacterial infection, bronchial wall thick...
Source: European Respiratory Journal - Category: Respiratory Medicine Authors: Tags: Editorials Source Type: research
Crystallization of recombinant proteins has been fundamental to our understanding of protein function, dysfunction, and molecular recognition. However, this information has often been gleaned under extremely nonphysiological protein, salt, and H+ concentrations. Here, we describe the development of a robust Inka1-Box (iBox)–PAK4cat system that spontaneously crystallizes in several mammalian cell types. The semi-quantitative assay described here allows the measurement of in vivo protein-protein interactions using a novel GFP-linked reporter system that produces fluorescent readouts from protein crystals. We combined t...
Source: Journal of Biological Chemistry - Category: Chemistry Authors: Tags: Protein Structure and Folding Source Type: research
Cystic fibrosis transmembrane conductance regulator (CFTR) is a chloride channel activated by protein kinase A (PKA) phosphorylation on the regulatory (R) domain. Phosphorylation at several R domain residues stimulates ATP-dependent channel openings and closings, termed channel gating. To explore the protein segment responsible for channel potentiation and PKA-dependent activation, deletion mutations were constructed by removing one to three protein segments of the R domain including residues 708–759 (ΔR708–759), R760–783, and R784–835, each of which contains one or two PKA phosphorylation sit...
Source: Journal of Biological Chemistry - Category: Chemistry Authors: Tags: Membrane Biology Source Type: research
Publication date: April 2020Source: The Lancet Respiratory Medicine, Volume 8, Issue 4Author(s): Deniz Dogru, Erkan Çakır, Tuğba Şişmanlar Eyüboğlu, Sevgi Pekcan, Uğur Özçelik, board members and the working group of the Cystic Fibrosis Registry of Turkey
Source: The Lancet Respiratory Medicine - Category: Respiratory Medicine Source Type: research
CONCLUSIONS: Burkholderia cepacia complex infections present a significant challenge for people with cystic fibrosis and their clinicians. The incidence is likely to increase as the cystic fibrosis population ages; and managing and treating these infections will become more important. There is a lack of trial evidence to guide decision making and no conclusions can be drawn from this review about the optimal antibiotic regimens for people with cystic fibrosis who have chronic Burkholderia cepacia complex infections. Clinicians must continue to assess each person individually, taking into account in vitro antibiotic suscept...
Source: Cochrane Database of Systematic Reviews - Category: General Medicine Authors: Tags: Cochrane Database Syst Rev Source Type: research
More News: Cardiology | Cystic Fibrosis | Health | Heart