This week, Vertex Pharmaceuticals Incorporated, a global biotechnology company that aims to discover, develop and commercialize innovative new medicines for diseases such as cystic fibrosis, announced that the U.S. Food and Drug Administration (FDA) has accepted for review a supplemental New Drug Application (sNDA) for the use of…
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Researchers at Claremont, California-based biotechnology company Synedgen are developing targeted therapies that address complications of cystic fibrosis (CF) on pulmonary and GI surfaces for which only limited treatment options are currently available — an unmet need frequently leading to early mortality. CF is a genetic disease caused by mutations in…
Mechelen, Belgium-based biotech company Galapagos NV is presenting promising research findings at this year’s North American Cystic Fibrosis Conference (NACFC), which is currently underway in Phoenix, Arizona. The clinical-stage company, which specializes in the discovery and development of small molecule medicines with novel modes of action to treat diseases like…
Quest Diagnostics recently announced findings from several research projects detailing the clinical value of genetic tests to help predict or detect diseases. Data from the studies, which are being presented at the 2015 American Society of Human Genetics (ASHG) Annual Meeting being held Baltimore, Maryland from October 8-10, 2015, show that screening…
Pulmatrix, Inc. released animal-based research data at the 2015 North American Cystic Fibrosis Conference indicating that PUR1900 (iSPERSE™-formulated itraconazole) helped fight against Aspergillus fumigatus in rats. The fungus causes lung infection in cystic fibrosis. Concentrations of PUR1900 were high in the lungs but low in the overall body of the rats, indicating that…
Boulder, Colorado based Nivalis Therapeutics, Inc. a clinical stage pharmaceutical company focused on treating people with cystic fibrosis (CF), is reporting positive topline results from a Phase 1b clinical study evaluating its Cystic Fibrosis drug candidate N91115 — a novel stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR)…
Thousands of health professionals, disease experts, advocates, investors, and even patients and their families will be flocking to the Phoenix Convention Center in Arizona this coming weekend, beginning Thursday, October 8 to Saturday, October 10, 2015 for the 29th Annual North American Cystic Fibrosis Conference (NACFC). The event’s sponsor, the Cystic…
Raptor Pharmaceutical Corp., a global biopharmaceutical company focused on the development and commercialization of transformative therapeutics for rare, debilitating and often fatal diseases, recently announced it has acquired the rights to Quinsair™ (levofloxacin inhalation solution) from Tripex Pharmaceuticals. Quinsair is the first inhaled fluoroquinolone antibiotic to be approved in the European…
Anthera Pharmaceuticals, Inc. is initiating a Phase 3 clinical trial entitled SOLUTION (Study of Oral Liprotamase Unit-Matched Therapy Of Non-Porcine Origin in People With Cystic Fibrosis) to investigate the use of Sollpura® (liprotamase) in patients with exocrine pancreatic insufficiency (EPI) due to cystic fibrosis. The study…
The European Medicines Agency (EMA) recently announced that it has validated the submission of a new indication for Translarna (ataluren) developed by PTC Therapeutics as a nonsense mutation cystic fibrosis (nmCF) treatment. PTC Therapeutics, a company that develops and assembles an integrated set of proprietary technologies focused on…
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