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MECFA Blog

Pharmac funds new cystic fibrosis medicine

New treatment for cystic fibrosis is on the horizon, one that will help young children suffering from the disorder digest their food more...

Eurordis Urges Expansion of Newborn Screening Initiatives Across Europe

European authorities must step up efforts to screen babies for a multitude of genetic disorders, a panel of experts suggested during a...

Rare Diseases Clinical Research Network Opens Online Survey on COVID-19

The Rare Diseases Clinical Research Network (RDCRN) has opened an online survey to better understand how the COVID-19 outbreak is...

Lung CT Scans May Help Predict Mortality Risk of IPF Patients

Lung evaluations via a CT scan might help predict the mortality risk in idiopathic pulmonary fibrosis (IPF) patients more accurately than...

AzurRx BioPharma Will Conduct Phase 2b Trial of MS1819 at TDN Clinical Sites

AzurRx BioPharma has been authorized to conduct its Phase 2b OPTION 2 clinical trial evaluating MS1819, its investigative therapy for...

International Trial Begins To Test Nitric Oxide As COVID-19 Treatment

A new, international clinical trial is underway that assesses inhaled nitric oxide as a treatment for patients with mild to moderate...

Coronavirus Update: New COVID-19 Antibiotic, WLBU2, Has Been Developed That Could Fight Lung Infecti

According to a report from The Dailymail, a new COVID-19 antibiotic has been developed that could potentially fight lung infections, such...

COVID-19 Outcomes in CF Patients Similar to General Public, Study Suggests

People with cystic fibrosis (CF) infected with the new coronavirus appear to have better outcomes than initially anticipated, with their...

A 1st: US study finds Gilead drug works against coronavirus

FILE - In this March 2020 photo provided by Gilead Sciences, a vial of the investigational drug remdesivir is visually inspected at a...

AzurRx BioPharma Receives Approval to Conduct its Phase 2 OPTION 2 Clinical Trial of MS1819 in Cysti

On track to initiate the OPTION 2 trial by the end of Q2 2020 OPTION 2 trial to compare efficacy of standard of care to 2.2 and 4.4 gram...

Disease Duration, Flare-ups Affect Mental Health of Non-CF Bronchiectasis Patients, Study Finds

VERTEX’S CF BLOCKBUSTER TRIKAFTA EFFECTIVE IN KIDS 6 TO 11 YEARS OF AGE

A case for improved carrier screening

Vertex to Seek Expanded FDA Approval for Trikafta After Positive Child Safety Data

Vertex’s Kaftrio approved for use in Europe and made accessible on the NHS

Age May Affect Outcomes in Lung Transplant Recipients With Cystic Fibrosis

CFF Funding Will Further ContraFect’s Research Into Alternative for Antibiotics

The search for molecular glue in targeted disease control

Vest Therapy Lowers Risk of Hospitalization in Non-CF Bronchiectasis, Study Says

Harvard is working with Boston hospitals to test existing cystic fibrosis drug to help coronavirus p