ProQR Therapeutics, a Leiden, NL based biotech company focused on development of drugs to treat severe genetic disorders, recently announced that its PQ-010-002 clinical trial is open for patient enrollment. The study’s primary endpoint is the estimation of the effect of topical administration of QR-010 on the nasal mucosa…
News
Pulmatrix, Inc., a clinical stage biopharmaceutical company developing innovative inhaled therapies to address serious pulmonary disease using its patented iSPERSE™ (inhaled small particles easily respirable and emitted) technology, has just announced it will be presenting preclinical data on the pharmacokinetics and potency of PUR1900 during the upcoming 2015 North American Cystic Fibrosis Conference. PUR1900 is an…
Earlier this week, AbbVie launched the voting contest portion of its yearly AbbVie CF Scholarship program. Everyone in the CF advocacy community and beyond is encouraged to visit www.AbbVieCFScholarship.com today through September 22, 2015 to weigh in on who best deserves this year’s Thriving Undergraduate or Graduate Scholar title and…
While the majority of patients diagnosed with cystic fibrosis (CF) at birth experience the symptoms of the disease during their childhood, Larry Brian is an uncommon case. The 25-year old patient never really understood the extent of suffering from the chronic and debilitating disease until he went to college.
Proteostasis Therapeutics, Inc. (PTI), a biotechnology company working on breakthrough therapeutics to treat diseases caused by defects in protein processing, recently announced that it has secured new financing worth $37 million, which will be dedicated to further developing its primary product candidate for cystic fibrosis for testing in human clinical studies. Cystic fibrosis…
Basilea Pharmaceutica Ltd recently announced plans to expand the delivery system of the experimental antibiotic BAL30072 to include an inhaled form suitable for cystic fibrosis patients and those with bronchiectasis.  Both conditions are known for the presence of multi-drug resistant colonization of bacteria that can lead to respiratory…
Cystic Fibrosis researcher Dr. Joshua R. Stokell, PhD, 36, of Charlotte, NC, passed away on June 8, 2015, due to complications associated with his own lifelong battle with Cystic Fibrosis (CF). A graduate of the University of North Carolina at Charlotte, Dr. Stokell had studied Cystic Fibrosis after earning his…
A recent review entitled “Inhaled therapy in cystic fibrosis: agents, devices and regimens” published in the journal Breathe explored the increasing number of inhaled therapeutic options for patients with CF, and how innovations in these therapies are dramatically improving quality of life…
In a paper entitled “Targeted delivery of liquid micro volumes into the lung” published in the Proceedings of the National Academy of Sciences of the United States of America (PNAS), a multidisciplinary team of researchers from Columbia University in New…
Researchers at Institut national de la santé et de la recherche médicale (INSERM) in France have learned one way to “fix” cells that have characteristics of cystic fibrosis. These cells, which had faulty CFTR channel proteins, were able to make more functional CFTR protein following treatment with tumor necrosis…
Your CF Community
Visit the Cystic Fibrosis News Today forums to connect with others in the CF community.