Gene Therapy Takes Center Stage at 2019 NORD Summit
With so much recent publicity surrounding gene therapy, it’s no surprise that the topic was a major focus of the recent 2019 NORD ... Read more
Updates
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cystic fibrosis Gene Therapy Takes Center Stage at 2019 NORD Summit
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cystic fibrosis, News Kalydeco Improves Lung Function in Patient Carrying Responsive and Non-responsive Mutations, Case Study Reports
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Columns, Mutations & Conversations - a Column by Tré LaRosa We Must Build a Diverse Coalition Within the CF Community
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cystic fibrosis Copeptin a Potential Biomarker of Symptom Severity During Pulmonary Exacerbation, Study Shows
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cystic fibrosis, News Quebec Loans Laurent $2.7M to Finance Trial Testing Oral LAU-7b in All CF Types
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News HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
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cystic fibrosis, News Israeli, US Researchers Develop New Device to Eliminate Mucus Secretion from Obstructed Airways
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cystic fibrosis, News Cystic Fibrosis Canada Opens Holiday ‘Wish’ Fundraising Campaign
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Columns, Life in the Grey: a Column by Bailey Anne Vincent Casting Call: Sickly Leading Ladies Wanted
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Columns, Victorious - a Column by Brad Dell I Am Proud to Be Youthful
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cystic fibrosis, News Elexacaftor Triple Combo Improves Lung Function and Quality of Life in CF Patients, Phase 3 Trial Shows
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cystic fibrosis, News CFF Strives to Improve Lung Transplant Journey for Cystic Fibrosis Patients
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cystic fibrosis, News IGF2R Blood Levels May Help Predict Antibiotic Response in CF Pulmonary Exacerbations, Study Says
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Columns, Life in the Grey: a Column by Bailey Anne Vincent Temper Tantrums Aren’t on My To-do List
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Columns, Victorious - a Column by Brad Dell Fear of Abandonment Has Driven My Life into the Ground
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cystic fibrosis Researchers Discover Stress Response Mechanism that Allows Pseudomonas Bacteria to Evade Antibiotics
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cystic fibrosis, News Low Birth Weight, Poor Weight Gain in First Year Predict Poor Growth in CF Infants, Study Shows
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cystic fibrosis, News French Authorities Approve Reimbursement of Orkambi for CF Patients 2 Years and Older
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cystic fibrosis, News NIH Grants $2.7M for Research on Anaerobic Bacteria’s Role in CF Flare-ups
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Columns, This Lung Life - a Column by Ella Balasa Attending This Year’s North American Cystic Fibrosis Conference Gave Me Hope
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cystic fibrosis, News CF-related Diabetes Affects Girls More Often and at Younger Age than Boys, European Study Shows
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Columns, Victorious - a Column by Brad Dell A Rare Disease Film Festival Made Me Ponder Our Future Fight for Others
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cystic fibrosis, News UI Researchers Develop Breakthrough Approach to Gene Therapies for Respiratory Diseases
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Columns, Mutations & Conversations - a Column by Tré LaRosa Starting Trikafta with Hope for the CF Community
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cystic fibrosis, News Discovered Bacteria ‘Wars’ May Lead to New Antimicrobial Treatments for CF, Study Suggests
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cystic fibrosis, News Patients Adhere Well to CFTR Therapies But High Costs Could Undermine Use, Pharmacy Study Says
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cystic fibrosis, News Vertex Reaches Agreement for CF Medications in Northern Ireland and Wales
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cystic fibrosis, News Rare Disease Film Festival Highlights Patient and Researcher Unity
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Columns, Life in the Grey: a Column by Bailey Anne Vincent Inspiration for Hire: Sick Candidates Only
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cystic fibrosis, News Proteostasis Therapeutics Completes Enrollment in Phase 2 Study Testing Triple Combination Therapy in Cystic Fibrosis
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cystic fibrosis, News Latinos on 2 Caribbean Islands Carry CFTR Mutations Seen in 1% of CF Patients Elsewhere, Study Says
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Becoming Visible by Elizabeth Rogers, Columns Addressing the Issues that Trikafta Benefits Will Bring
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News Bionic Pancreas May Be Useful in Managing Blood Sugar Levels in Adults with CFRD, Small Study Says
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cystic fibrosis, News Eloxx Presents Positive Preclinical and Clinical Data of CF Therapy Candidate ELX-02
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cystic fibrosis, News Preclinical Data Supports Effectiveness of ARO-ENaC, Arrowhead’s Investigational Therapy for CF
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cystic fibrosis, News Two New CFTR Gene Variants Identified in Young CF Patients, Study Reports
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Columns, Mutations & Conversations - a Column by Tré LaRosa What We Should Expect from the CF Foundation’s ‘Path to a Cure’
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cystic fibrosis, News Arrevus Gets Support from NC Biotech Center to Advance ARV-1801 for Pulmonary Exacerbations in CF
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cystic fibrosis, News European Regulators Take Step Toward Possible Approval of Vertex’s New Combo Therapy for 90% of CF Patients
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cystic fibrosis, News $15M Donation to Support CF Research Program at University of Queensland
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cystic fibrosis Trikafta’s Approval the Outcome of ‘20-year Journey That Started with a Dream’, Says Vertex CEO Jeffrey Leiden
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Columns, Life in the Grey: a Column by Bailey Anne Vincent What to Do When Everyone Dies
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Columns, Victorious - a Column by Brad Dell The Present Realizes the Past Through Travel
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cystic fibrosis, News Alaxia Joins iABC to Speed Development of ALX-009, Potential Antibiotic for Resistant Lung Infections
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Columns, Mutations & Conversations - a Column by Tré LaRosa Giving My Anxiety a Name
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cystic fibrosis, News Vertex, NHS England Reach Agreement on Pricing and Public Availability of CF Treatments
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News CF Patients Voice Hopes and Expectations for Trikafta in Instagram Poll
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cystic fibrosis, News CF Mutation Carriers Have Increased Risk for Gastrointestinal Problems, Large-scale Study Finds
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cystic fibrosis, News Cystic Fibrosis Foundation Applauds FDA Approval of Trikafta
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cystic fibrosis, News Europe’s CHMP Issues Positive Opinion on Kalydeco Use in Infants
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Columns, Life in the Grey: a Column by Bailey Anne Vincent Sick Food Diaries: A Day In the Life
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cystic fibrosis, News FDA Approves Trikafta, 1st Vertex Triple Combo with Potential to Treat 90% of CF Patients
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cystic fibrosis, News 1st Patient Enrolled in Phase 2 Study of Inhalation Treatment for All with CF, Boehringer Says
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cystic fibrosis, News First Patients Dosed in Trial Testing MS1819-SD Plus PERT for CF-related Severe EPI, AzurRX Says
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cystic fibrosis, News Regular Exercise Promotes Lung Health in CF by Lowering Adiponectin Levels, Study Suggests
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cystic fibrosis, News Chelsea Financial Services Supporting Boomer Esiason Foundation, CF Research at NYC Marathon
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Columns, Victorious - a Column by Brad Dell The Art of Not Helping
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cystic fibrosis, News Orkambi of Definite Benefit But Risk Evident in Patients Who Can’t Tolerate It, French Study Finds
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Columns, Path Unknown - a Column by Wendy Caroline I’m Grateful for the Journey That Led to My Service Dog
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Columns, Mutations & Conversations - a Column by Tré LaRosa Preventing the Spread of Misinformation
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cystic fibrosis, News UNITY Prenatal Blood Test Accurate at Diagnosing CF, Other Hereditary Diseases, Study Shows
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cystic fibrosis, News Viral Respiratory Tract Infections in Young CF Children Usually Mild, Study Found
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cystic fibrosis, News FDA Designates Vast’s BIOC11 a Qualified Infectious Disease Product
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cystic fibrosis, News Scientists Hope They Have Discovered New Form of CF Therapy
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Columns, Life in the Grey: a Column by Bailey Anne Vincent Sorry Not Sorry That I’m Sick
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cystic fibrosis Experts Highlight Obstacles in Delivering New CF Therapies
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Columns, Mutations & Conversations - a Column by Tré LaRosa Debunking the Myths About Vaccine Safety
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cystic fibrosis, News 5,000 Expected to Attend Cystic Fibrosis Conference, NACFC 2019, in Nashville
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cystic fibrosis Patient-derived Mini-organs Work as ‘Avatars’ to Test CF Treatments
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cystic fibrosis, News Vertex Partners with Ribometrix to Discover RNA-targeted Therapies
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cystic fibrosis, News Hexoskin Smart Shirt Can Accurately Assess Respiratory Function, May Be Tool in CF, Study Shows
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Columns, Life in the Grey: a Column by Bailey Anne Vincent ‘I’m Better at Being Sick Than You Are’: Chronic Illness Is Not a Competition
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cystic fibrosis, News IL-1 Receptor Inhibition a Potential Strategy to Block Mucus Build-up in Early CF, Study Suggests
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Columns, Mutations & Conversations - a Column by Tré LaRosa An Obstacle on My Chosen Career Path Led to an Identity Crisis
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cystic fibrosis, News Public Encouraged to Vote for 2 Students with CF to Receive $22K AbbVie Scholarships
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cystic fibrosis, News AzurRx BioPharma Announces Promising Results from Trial Testing MS1819 for EPI in CF Patients
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cystic fibrosis, News NORD 2019 Rare Disease Summit Set for Oct. 21-22 in Washington, DC
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cystic fibrosis, News Professor Launches Research Initiative to Encourage Students to Develop and Test Antibiotics for CF Infections
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cystic fibrosis, News CF Foundation Awards Eloxx $1.61M for Phase 2 Trials in People with Nonsense Mutations
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Columns, Life in the Grey: a Column by Bailey Anne Vincent I Want to Break Up with My CF Clinic
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cystic fibrosis, News Kalydeco’s Efficacy Tied to Phosphorylation Levels of CFTR Protein, Study Suggests
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Columns, Mutations & Conversations - a Column by Tré LaRosa The Benefits and Perils of Social Media
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cystic fibrosis, News Physical Activity Levels in CF Should Be Objectively Researched, Review Says
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cystic fibrosis, News LCI Is Sensitive Lung Function Parameter in Patients with Mild CF, Study Says
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News 1st Biosimilar of Dornase Alfa Inhalation Solution for CF Approved in Russia, Companies Announce
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News Translate Bio Prioritizes Development of CF Therapy MRT5005, Other Lung Disease Candidates
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News Prenatal Testing for Cystic Fibrosis Influences Pregnancy Outcomes, Review Finds
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Columns, Life in the Grey: a Column by Bailey Anne Vincent Our Colons Need Your Help
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Columns, Mutations & Conversations - a Column by Tré LaRosa Reflecting on the First Year of My Column, ‘Mutations & Conversations’
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News Armata Announces New Treatment Candidate for P. Aeruginosa Infections in CF Patients
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News New Agreement in Scotland Grants CF Patients Access to Orkambi and Symkevi
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News Safety, Early Effectiveness of POL6014 Confirmed in Phase 1 Trial
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News ICER Seeking Feedback on CF Treatments from Stakeholders
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Columns, Life in the Grey: a Column by Bailey Anne Vincent I Don’t Identify with the Word Disabled
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cystic fibrosis, News Drexel Study to Look at Tailored ‘Talk’ Therapy in Helping Ease Depression and Anxiety in CF Patients
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Columns, Mutations & Conversations - a Column by Tré LaRosa I Spent a Day On Capitol Hill Speaking with Lawmakers About CF and Healthcare
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cystic fibrosis, News Tarpon Cellars Uses Wine Sales to Help CF Foundation
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cystic fibrosis, News Amikacin Liposome Therapy Has Similar Efficacy as Tobramycin for P. aeruginosa, Study Shows
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cystic fibrosis, News Claire’s Place Foundation Glow Ride Will Include Virtual Riders This Year
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Kalydeco Improves Lung Function in Patient Carrying Responsive and Non-responsive Mutations, Case Study Reports
Treatment with Kalydeco (ivacaftor) alone improved lung function and quality of life in a cystic fibrosis patient carrying both a therapy-responsive and a ... Read more -
We Must Build a Diverse Coalition Within the CF Community
I learned long ago not to expect everyone to agree with me. I also realized that disagreement isn’t inherently wrong. From a young age, ... Read more -
Copeptin a Potential Biomarker of Symptom Severity During Pulmonary Exacerbation, Study Shows
High levels of the peptide copeptin are associated with increased severity of cystic fibrosis (CF) in children, a study shows. These results suggest that ... Read more -
Quebec Loans Laurent $2.7M to Finance Trial Testing Oral LAU-7b in All CF Types
The Government of Quebec, in Canada, has approved a loan of $2.7 million Canadian (about $2.05 million U.S.) to Laurent Pharmaceuticals to help the company ... Read more
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HHS Secretary Alex Azar Touts White House Efforts to Cure Rare Diseases
Despite skyrocketing healthcare costs, President Trump is committed to protecting the 30 million or ... -
Israeli, US Researchers Develop New Device to Eliminate Mucus Secretion from Obstructed Airways
Researchers in Israel and the U.S. have developed a new technology that can be ... -
Cystic Fibrosis Canada Opens Holiday ‘Wish’ Fundraising Campaign
To heighten cystic fibrosis (CF) awareness and raise funds for research, care and advocacy, Cystic ... -
Casting Call: Sickly Leading Ladies Wanted
I miss someone. I don’t even know her … and yet I miss her. ... -
I Am Proud to Be Youthful
Loureen and I waited for our travel buddies to pass through airport security. Soon, ... -
Elexacaftor Triple Combo Improves Lung Function and Quality of Life in CF Patients, Phase 3 Trial Shows
The therapeutic triple combo elexacaftor (VX-445), tezacaftor, and ivacaftor leads to marked improvements in lung function ...
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CFF Strives to Improve Lung Transplant Journey for Cystic Fibrosis Patients
When it comes to lung transplants, the Cystic Fibrosis Foundation (CFF) admits that for ... -
IGF2R Blood Levels May Help Predict Antibiotic Response in CF Pulmonary Exacerbations, Study Says
Measuring the levels of insulin-like growth factor 2 receptor (IGF2R) during the first days ... -
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French Authorities Approve Reimbursement of Orkambi for CF Patients 2 Years and Older
The Comité Économique des Produits de Santé (CEPS) in France has approved national reimbursement of ... -
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A Rare Disease Film Festival Made Me Ponder Our Future Fight for Others
I don’t write much about my work at BioNews Services, which publishes Cystic Fibrosis News ... -
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Discovered Bacteria ‘Wars’ May Lead to New Antimicrobial Treatments for CF, Study Suggests
Pseudomonas aeruginosa, a bacteria that commonly causes serious lung infections in people with cystic ... -
Patients Adhere Well to CFTR Therapies But High Costs Could Undermine Use, Pharmacy Study Says
People with cystic fibrosis (CF) show a “high adherence” to CFTR modulators, but these ... -
Vertex Reaches Agreement for CF Medications in Northern Ireland and Wales
The Regional Pharmaceutical Procurement Service in Northern Ireland and the National Health Service (NHS) ... -
Rare Disease Film Festival Highlights Patient and Researcher Unity
Rare disease-themed videos glowed on a large screen before an audience of people in ... -
Inspiration for Hire: Sick Candidates Only
At this time last week, I was crying. I was crying because I didn’t ... -
Proteostasis Therapeutics Completes Enrollment in Phase 2 Study Testing Triple Combination Therapy in Cystic Fibrosis
Proteostasis Therapeutics (PTI) has completed patient enrollment in a Phase 2 clinical trial evaluating ... -
Latinos on 2 Caribbean Islands Carry CFTR Mutations Seen in 1% of CF Patients Elsewhere, Study Says
Latinos in the Caribbean with cystic fibrosis (CF) carry rare CFTR gene mutations not ... -
Addressing the Issues that Trikafta Benefits Will Bring
It’s time to move forward. The recent approval by the U.S. Food and Drug ... -
Bionic Pancreas May Be Useful in Managing Blood Sugar Levels in Adults with CFRD, Small Study Says
The bionic pancreas (BP), an automated system that controls blood sugar levels, may be ... -
Eloxx Presents Positive Preclinical and Clinical Data of CF Therapy Candidate ELX-02
Eloxx Pharmaceuticals presented additional positive preclinical and clinical data for ELX-02, its lead treatment candidate for cystic ... -
Preclinical Data Supports Effectiveness of ARO-ENaC, Arrowhead’s Investigational Therapy for CF
Preclinical data shows that Arrowhead Pharmaceuticals‘ investigational inhaled treatment for cystic fibrosis (CF), called ARO-ENaC, ... -
Two New CFTR Gene Variants Identified in Young CF Patients, Study Reports
Two new alterations in the CFTR gene, leading to a deficient CFTR protein and ... -
What We Should Expect from the CF Foundation’s ‘Path to a Cure’
Last week, the Cystic Fibrosis Foundation launched a new initiative called “Path to a ... -
Arrevus Gets Support from NC Biotech Center to Advance ARV-1801 for Pulmonary Exacerbations in CF
Arrevus received a Small Business Research Loan from the North Carolina Biotechnology Center to support ... -
European Regulators Take Step Toward Possible Approval of Vertex’s New Combo Therapy for 90% of CF Patients
The European Medicines Agency (EMA) has validated Vertex Pharmaceuticals’ application seeking approval for its ... -
$15M Donation to Support CF Research Program at University of Queensland
The University of Queensland (UQ), in Australia, has launched its Queensland Cystic Fibrosis Research ... -
Trikafta’s Approval the Outcome of ‘20-year Journey That Started with a Dream’, Says Vertex CEO Jeffrey Leiden
When Vertex Pharmaceuticals’ cystic fibrosis (CF) therapy Trikafta was approved recently by the U.S. Food ... -
What to Do When Everyone Dies
Two of my friends are dead. And I’m scared. Recently, I lost two friends ...