Guest Blog By: Meranda Honaker
Over the last several months my health has continued to decline despite being compliant and diligent with my healthcare routine. In July I developed a fever during a trip to Boston to speak to a biotech company about my journey with cystic fibrosis. I developed a fever and by the end of my visit, I was unable to walk up steps without severe dyspnea. I was so exhausted from feeling sick I would return to my hotel room to lay in the bed for hours to rest. My chest pain and shortness of breath became so severe in the coming days that I could no longer take a deep breath. I checked my SpiroPd home spirometer which displayed a definite decline in my lung function. I immediately contacted my CF clinic to see my CF doctor. Initially, despite feeling bad, I assumed I was feeling poorly and decided not to rush to CF clinic. Sometimes I have a bad day or few bad days health wise and begin to improve on my own. Once I saw my lung function had declined I knew I needed to be seen in CF clinic rather than waiting it out.
Continue reading Home Spirometers: A Useful Tool in Tracking CF Symptoms and Progress
In this Cystic Fibrosis Wind Sprint, Jerry Cahill talks about the importance of never missing your medication, especially post-transplant. To help him keep track of his meds, Jerry keeps color coordinated pill cases in his car with extra doses of medications in case he ever forgets to take them. He also keeps extra dosages in his backpack, which he carries with him everywhere, for the same reason.
The video wind sprint was made possible through an unrestricted education grant from Genentech to the Boomer Esiason Foundation.
Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) announced that the New England Journal of Medicine (NEJM) published two articles with results from two Phase 3 studies of the tezacaftor/ivacaftor combination treatment, a medicine in development that is designed to treat the underlying cause of cystic fibrosis (CF) in people ages 12 and older who have certain mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Continue reading Positive Results for Phase 3 Studies of the Tezacaftor/Ivacaftor Combination Treatment
Eloxx Pharmaceuticals Announces Expansion of Intellectual Property Estate for ELX-02 with Newly Granted Patent in Europe
Sevion Therapeutics, Inc. (OTCQB:SVON) and Eloxx Continue reading Biopharmaceutical company developing novel small molecule medicines for genetic diseases caused by nonsense mutations
ProQR Completes Dosing of Cystic Fibrosis Patients in QR-010 Phase 1b Trial
• Last patient received their final dose in the PQ-010-001 Phase 1b clinical trial of QR-010 in CF patients with the F508del mutation.
Top-line trial data are expected to be issued in a press release Continue reading Phase 1b Trial of QR-010
Tezacaftor/Ivacaftor Gets Priority Review for Cystic Fibrosis
The Food and Drug Administration (FDA) has granted Priority Review to the New Drug Application (NDA) of tezacaftor/ivacaftor (Vertex) for the treatment of patients ≥12yrs old with cystic fibrosis (CF) who have two copies of the F508del mutation or one F508del mutation and one residual function mutation.
The NDA submission was based on positive results from 2 global Phase 3 trials, which showed statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in patients treated with tezacaftor/ivacaftor.
The combination treatment consists of ivacaftor (marketed under the brand name Kalydeco), a cystic fibrosis transmembrane conductance regulator (CFTR) potentiator, and tezacaftor, a novel CFTR corrector. Tezacaftor is designed to address the processing defect of F508del-CFTR to enable it to reach the cell surface, where ivacaftor can further enhance the protein’s function.
The FDA has set a Prescription Drug User Fee Act (PDUFA) target date of February 28, 2018 to make a decision on the NDA.
For more information visit Vrtx.com.
ProQR Announces Results for the Second Quarter of 2017
• Data from two clinical trials of QR-010 presented at the European Cystic Fibrosis Conference. Enrollment completed in the Phase 1b clinical trial in cystic Continue reading Two clinical trials presented at the European Cystic Fibrosis Conference
Anthera Pharmaceuticals Announces RESULT Phase 3 Clinical Study of Sollpura will be Included in the European Cystic Fibrosis Clinical Trial Network
Anthera Pharmaceuticals (Nasdaq:ANTH) today announced that the RESULT Continue reading Results for Phase 3 Clinical Study to Treat Exocrine Pancreatic Insufficiency
AIT Therapeutics Announces Enrollment of First Patient into Its NO-NTM abscessus Phase 2 Trial in Nontuberculous Mycobacteria (NTM)
10 patients with Mycobacterium Abscessus Complex (MABSC) are expected to enroll in the trial Continue reading Phase Two Clinical Trials to treat NTM