Lung Clearance Index Improved in Children Ages 6-11 on Ivacaftor/Lumacaftor
Ivacaftor/Lumacaftor Improves Lung Clearance Index Score In Children A new phase 3 study, led by The Hospital for Sick Children at the University of Toronto, Canada, has been completed on the CF treatment ivacaftor/lumacaftor in children ages 6-11 with two F508-del CFTR mutations. In a group of 206 children from …
New drug (Thymosin alpha 1) discovery: potential for Cystic Fibrosis treatment
According to an article published on www.sciencedaily.com, researchers have discovered a potential new drug to treat and stop the progression of cystic fibrosis. While many other promising drugs are in the pipeline, we here at cysticfibrosis.com will be keeping a keen eye on further developments of this medication. Read the …
Orkambi Study On Children Age 6-12 With DDF508: Cystic Fibrosis Treatment
Results are in from a 24-week study of Orkambi (lumacaftor/ivacaftor – a medication used in cystic fibrosis treatment) in 58 children with cystic fibrosis age 6-12 with two copies of the F508-delta mutation. Researchers were looking to examine the safety and efficacy of the CFTR potentiator on this age group and …
Lumacaftor/Ivacaftor longer trial on Heterozygous F508del Cystic Fibrosis Patients
At the end of November, the results of a clinical trial of Lumacaftor/Ivacaftor, also known as Orkambi, in CF patients with one F508Del mutation were published in the Annals of the American Thoracic Society. A past trial for Orkambi in this type of CF patient (heterozygous F508del) lasted 28 days …
Drug Development Pipeline for Cystic Fibrosis treatment
The CFF has a interesting and easy to use Drug Development Pipeline webpage which shows the stages of medication that is being developed for Cystic Fibrosis treatment. The stage of development of each medication is clearly indicated and the medications are divided into categories of therapeutic approach. The categories are: Restore CFTR …
CRISPR Finds Mutated DNA and Is Okayed for Clinical Trial
Since 2003, when we first started saving all our messages here at CysticFibrosis.com, Cystic Fibrosis genetics and our mutations have always been a hot topic. At first, we didn’t even know our genetics could be tested, because the sweat test was the gold standard for diagnosis. Then came Ambry Genetics. …
Matching Volunteers to Clinical Trials by App
It takes on average 17 years for clinical breakthroughs in the laboratory to reach patients. A big part of the delay is finding enough patients to take part in clinical trials. Only 5 to 10 percent of eligible adults enroll—and of those who show initial interest, only 5 percent …
In the News: Inhaled Levofloxacin & QIDP Designation
Raptor Pharmaceutical’s MP-376 (Inhaled Levofloxacin) granted QIDP Status by the FDA on March 16, 2016. What is Qualified Infectious Disease Product (QIDP) Designation? QIDP status gives a drug priority review by the FDA, eligibility for Fast Track designation, and an extension of marketing exclusivity. This incentivizes pharmaceutical companies to devote …
We Lobby
A Brief History of Our Lobbying Efforts in 2015 In April, 2015, we sent this letter to the FDA Committee: Sent: Thursday, April 02, 2015 12:17 PM To: PADAC Subject: on line patient community at cysticfibrosis.com Dear Dr. Hong, We run an on line patient community at cysticfibrosis.com. Several …