Interesting new clinical trial for 661/Kalydeco
Was checking to see if next generation details were at clinicaltrials.gov and didn't find but discovered this study, which I hadn't been aware of: https://clinicaltrials.gov/ct2/show/...lmonary&rank=1 It is a small study for 661/Kalydeco, not the Phase 3 for homozygotes going on now, but a Phase 2 looking at "extrapulmonary systems." Primary end point is mucociliary clearance, and secondary endpoints include (beyond FEV), change in gastrointestinal pH, change in sweat chloride, change in nasal potential difference. This is interesting to me because the FDA at the Orcambi discussed (if I recall correctly) t...
Source: Cystic Fibrosis Adults Forum - November 9, 2015 Category: Respiratory Medicine Authors: Aboveallislove Tags: Adults Source Type: forums

Re: Should surgical training include involvement in a clinical trial?
(Source: BMJ Comments)
Source: BMJ Comments - November 6, 2015 Category: Journals (General) Source Type: forums

New Sodium Chloride Clinical Trial
Anyone's child in the new clinical trial for a new sodium chloride neb treatment? DS13 has an appt next week to see if he is eligible. It only lasts 4 weeks: 2 weeks off sodium chloride, and then 2 weeks in the trial for the new med. (Source: Cystic Fibrosis Families Forum)
Source: Cystic Fibrosis Families Forum - November 4, 2015 Category: Respiratory Medicine Authors: scrapper1264 Tags: Families Source Type: forums

Re: Liraglutide in people treated for type 2 diabetes with multiple daily insulin injections: randomised clinical trial (MDI Liraglutide trial)
(Source: BMJ Comments)
Source: BMJ Comments - November 1, 2015 Category: Journals (General) Source Type: forums

Re: Frequency of discrepancies in retracted clinical trial reports versus unretracted reports: blinded case-control study
(Source: BMJ Comments)
Source: BMJ Comments - October 4, 2015 Category: Journals (General) Source Type: forums

Re: Liberating the data from clinical trials
(Source: BMJ Comments)
Source: BMJ Comments - October 3, 2015 Category: Journals (General) Source Type: forums

Re: Frequency of discrepancies in retracted clinical trial reports versus unretracted reports: blinded case-control study
(Source: BMJ Comments)
Source: BMJ Comments - September 22, 2015 Category: Journals (General) Source Type: forums

GlobalGenes.org in Huntingdon Beach California
I will be there representing all of us! We are being sponsored by Healthcare at Home a company from the UK. I met Natalie Douglas in April at an eyeforpharma conference in Philadelphia. She is a Board member of www.globalgenes.org. She is impressed with our community, and the work we are doing since the important survey when 478 of you participated. I will bring the reports...but mostly KNOW that YOU are the Gatekeepers of so much important work for Rare Diseases! I want to let everyone know YOU are the ones who cheer each other into clinical trials. You are the ones who are testing personalized medicine...which as ...
Source: Cystic Fibrosis Adults Forum - September 21, 2015 Category: Respiratory Medicine Authors: Imogene Tags: Adults Source Type: forums

Re: Liberating the data from clinical trials
(Source: BMJ Comments)
Source: BMJ Comments - September 18, 2015 Category: Journals (General) Source Type: forums

Orkambi Success
I'm delta F508 homozygous, I'm 20 years old with an FEV1 of 89% and it's all thanks to Orkambi. I've been on the drug for 2 years through its clinical trial phase. Trust me everyone, it's a miracle. Diet and exercise and certain health and wellness products along with Orkambi is key to your success. Take a second to run past my blog brandoncysticfibrosis.wordpress.com I started it yesterday so folks who are interested can track my journey and know that Orkambi not only works, it blew away all of my expectations. My lung function has shot up 14 points in 2 years and this is after a steep down trend. The benefits are there a...
Source: Cystic Fibrosis Adults Forum - August 22, 2015 Category: Respiratory Medicine Authors: Brandon Justice Tags: Adults Source Type: forums

Clinical trials: stories please
Forum: Student surveys and research Posted By: *Emerlina* Post Time: 02-08-2015 at 18:56 (Source: The Student Room)
Source: The Student Room - August 2, 2015 Category: Universities & Medical Training Source Type: forums

The (Misuse) of Observational and Randomized Clinical Trials for Causal Inference: Re: Peer victimisation during adolescence and its impact on depression in early adulthood: prospective cohort study in the United Kingdom
(Source: BMJ Comments)
Source: BMJ Comments - June 29, 2015 Category: Journals (General) Source Type: forums

remarks on Hypertension management algorithm for type 2 diabetic patients applied in primary care
Letter to the editor Hypertension management algorithm for type 2 diabetic patients applied in primary care Luciana V Viana, Cristiane B Leitão, Maria F Grillo, Ennio P C C Rocha, Juliana K Brenner, Rogerio Friedman and Jorge L Gross : Diabetology and Metabolic Syndrome 2013, 5:52 Paul Smits, general practitioner with special interest in cardiovascular disease, Netherlands This interesting primary care based study studied the results of the use of an algorithm in obtaining blood pressure under the threshold of the American Diabetes Association and the Joint National Committee on Prevention. Almost half of the studie...
Source: Diabetology and Metabolic Syndrome Comments - June 22, 2015 Category: Endocrinology Authors: Paul Smits Source Type: forums