February 26, 2021February 24, 2021 Celebrating the Opportunity to See the World Through Rare Eyes All of us who are affected by cystic fibrosis have our own unique experiences. No two stories are the same, let alone our genetic mutations. ... Read more
February 26, 2021February 26, 2021 Rare Disease Day ‘Patient Hero’ Raises Awareness Through Fashion Diagnosed with sickle cell disease as a 6-month-old, Tristan Lee has faced a lot of challenges over his 37 years of life. But from a ... Read more
February 25, 2021February 25, 2021 Girls With CF Need Reproductive Counseling, Study Asserts Female adolescents with cystic fibrosis (CF) should be offered contraceptive counseling and informed about pregnancy risks, a long-term study shows. Adding a gynecologist to the ... Read more
February 24, 2021February 24, 2021 Pravibismane, Potential New Antimicrobial Class, Gets 2nd CFF Grant The Cystic Fibrosis Foundation has given an additional $1.8 million to support early toxicology studies of pravibismane, Microbion’s investigational inhaled therapy for lung ... Read more
February 23, 2021February 19, 2021 Why My Recent Happiness Makes Me Wonder If I’m Secretly Ableist “I am so happy right now,” I say to my partner as we set off on a drive on an ordinary Sunday. I haven’t said ... Read more
cystic fibrosis, News Potential CF Mutation, F1099L, Identified in 2 Minority Children in US February 23, 2021February 23, 2021 A rare and disease-causing mutation for cystic fibrosis (CF), called the F1099L mutation and characterized ...
News, syndicated Rare Disease Day at NIH, Set for March 1, Growing Year by Year February 22, 2021February 22, 2021 Rare Disease Day at NIH, organized by the National Institutes of Health (NIH) and taking place ...
cystic fibrosis, News $1.4M Grant Supports Combo Approach to Antibiotic-resistant Infections February 22, 2021February 22, 2021 Work targeting new therapies against antibiotic-resistant infections, led by West Virginia University (WVU) researchers, received ...
cystic fibrosis, News CFF Awards $2.17M to Support Trial of Inhaled NO for NTM Lung Infections February 19, 2021February 19, 2021 The Cystic Fibrosis Foundation (CFF) has given Beyond Air up to $2.17 million to test its portable ...
cystic fibrosis, News Research Examines CF Patients’ Susceptibility to COVID-19 February 18, 2021February 18, 2021 Researchers have developed a patient-derived model of cystic fibrosis (CF) to better understand whether these ...
News, syndicated Eurordis Survey: Healthcare Experience Worse for Rare Disease Patients February 17, 2021February 17, 2021 People with rare disorders have a worse healthcare experience than those affected by chronic diseases, ...
ELX-02 Restores CFTR Activity in Organoids With G542X Mutations, Study Finds February 17, 2021February 18, 2021 ELX-02 restored the levels and function of CFTR — the defective protein in CF — ...
I’m Thin and Sick, Which Gives Me Power to Be a Dangerous Bully February 16, 2021February 11, 2021 It’s 10:25 a.m. and all I’ve had is coffee. Last week, I wrote about my ...
UK Trial to Investigate Safety of Lowering CF Treatment Burden via Kaftrio February 16, 2021February 16, 2021
Organoids Accepted as Means to Targeted Therapy, Survey Reveals February 12, 2021February 12, 2021 A recent survey shows that people with cystic fibrosis (CF) broadly favor targeted therapy using ...
Pilot Study Uses CRP, Calprotectin to Diagnose Pulmonary Exacerbations February 9, 2021February 9, 2021 Blood C-reactive protein (CRP) and calprotectin levels can reliably identify pulmonary exacerbations in cystic fibrosis ...
cystic fibrosis, News Vitamin D in High Daily Doses Doesn’t Appear to Help – or Do Harm February 5, 2021February 5, 2021 Higher doses of vitamin D supplements do not appear to lead to better clinical outcomes, ...
News NORD’s 6th ‘State Report Card’ Notes Progress, Raises Concerns February 4, 2021February 10, 2021 While progress was made last year on newborn screening and other policy issues critical to ...
cystic fibrosis, News Phage Therapy Clears Resistant Infection in CF Lung Transplant Patient February 4, 2021February 4, 2021 For the first time, bacteriophage therapy treated a boy with chronic and antibiotic-resistant Achromobacter bacteria ...
cystic fibrosis, News Emerging Bacteria Species Shows Antibiotic Resistance, Patient Transmission February 3, 2021February 3, 2021 The Achromobacter bacteria species that is an increasing source of chronic lung infections in people ...
Columns, Life in the Grey – a Column by Bailey Anne Vincent In Dance and Other Matters, Representation Counts, but Not Enough February 2, 2021January 28, 2021 I am going to write something no one wants to hear: Honesty is best when ...
cystic fibrosis, News CF Children May Take Enzyme Therapies on Own at Atlanta Hospital February 2, 2021February 2, 2021 Hospitalized children and adolescents with cystic fibrosis (CF) who can take enzyme treatments for pancreatic ...
cystic fibrosis, News Trial of MS1819, Yeast-based EPI Treatment, Opens 2 Sites in Poland February 1, 2021February 1, 2021 Two new sites for the extension of a Phase 2b trial testing an immediate release capsule ...
Columns, Valiant Voice – a Column by Lara Govendo I’ve Found Powerful Camaraderie in the CF Community January 29, 2021January 27, 2021 Two of the most powerful words in our vocabulary are “me, too.” Many of us ...
cystic fibrosis, News CF Foundation Grant Supports Testing of CSA-131 Against Resistant Bacteria January 29, 2021January 29, 2021 The Cystic Fibrosis Foundation (CFF) has given Kinnear Pharmaceuticals up to $3 million to conduct the ...
cystic fibrosis, News FDA to Review Trikafta for Patients as Young as 6 January 28, 2021January 28, 2021 The U.S. Food and Drug Administration (FDA) has accepted for review an application to expand ...
News, syndicated COVID-19 Vaccines Pose Little Risk to Rare Disease Patients, FDA, CDC Say January 27, 2021January 27, 2021 The two COVID-19 vaccines that recently received emergency approval from the U.S. and other worldwide ...
cystic fibrosis, News Bronchoscopy Plus BAL Safe Way of Checking Lung Health of Patients January 27, 2021January 27, 2021 Using bronchoscopy to examine the airways and the surrounding tissues of people with cystic fibrosis, coupled ...
Columns, Life in the Grey – a Column by Bailey Anne Vincent Confession: Vaccine Politics Are Adding to My Sadness January 26, 2021January 22, 2021 I am in a bathroom feeling sick, writing about being sick, not wanting to be ...
Columns, Victorious - a Column by Brad Dell What Will You Do to Ensure Others Aren’t Excluded? January 26, 2021January 25, 2021 I’ve often written that “the disease itself isn’t the worst part, rather it’s the …” ...
cystic fibrosis, News Pneumonia Seen as More Likely in CF Patients, Carriers of European Ancestry January 26, 2021January 26, 2021 Cystic fibrosis (CF) patients and carriers — those with mutations in only one copy of ...
Columns, Mutations & Conversations - a Column by Tré LaRosa The Long Journey of Learning to Love My Own Body January 25, 2021January 21, 2021 I used to be ashamed about my insecurities. I believed that as a man, I ...
cystic fibrosis, News Minorities in US Ineligible for CFTR Modulators Due to Poorly Studied Mutations January 25, 2021January 25, 2021 Individuals with cystic fibrosis (CF) from racial and ethnic minority groups in the U.S. are ...
News, syndicated NORD’s Caregiver Respite Program Continues Through Pandemic January 22, 2021January 22, 2021 Caring for a loved one with a rare disease, especially during these uncertain times, demands ...
