April 21, 2021April 21, 2021 Quick Approval for COVID Vaccines a Concern, Rare Disease Patients Say More than half of the people with rare diseases and their caregivers, asked in a survey, were undecided or less than willing to be vaccinated ... Read more
April 21, 2021April 21, 2021 KB407 In-human Trial Expected After Gene Therapy Found Safe in Animals Krystal Biotech is expecting to start a Phase 1 clinical trial by early fall to test KB407 for cystic fibrosis (CF), after the investigational inhaled ... Read more
April 20, 2021April 16, 2021 I Swapped Health Routines With Another CF Patient for a Day I am a creature of habit. So much so that it’s easy to forget that other chronic illness patients deal with their conditions in completely ... Read more
April 20, 2021April 20, 2021 Levels of Neutrophil Elastase May Help Predict Disease Severity in CF Levels of neutrophil elastase, a pro-inflammatory enzyme normally released by immune cells known as neutrophils, plus a greater diversity and amount of microbes could drive ... Read more
April 19, 2021April 15, 2021 Amid Mental Health Struggles, It’s Challenging to Work and Be Productive I can’t separate my physical malady from my mental diseases. Cystic fibrosis, depression, and anxiety are all linked, and they’re all separate. I think of ... Read more
News Assessing Treatment Burden, Complexity May Improve Therapy Adherence April 19, 2021April 19, 2021 Routine evaluations of how cystic fibrosis (CF) patients and their families are coping with the ...
Columns, Valiant Voice – a Column by Lara Govendo After Transplant, It’s OK to Struggle and Feel Grateful April 16, 2021April 14, 2021 I talk about my life with a double-lung transplant regularly. Sharing the double-edged sword of ...
News Bioengineer to Use $2M Grant to Study Gene Editing for CF April 16, 2021April 16, 2021 A bioengineer at Rice University will use a more than $2 million federal grant for ...
News, syndicated EveryLife Scholarship Fund Open Until May 7 for Adults With Rare Diseases in US April 15, 2021April 15, 2021 The EveryLife Foundation for Rare Diseases is accepting applications for a scholarship program that aims to ...
cystic fibrosis, News Claire’s Place Foundation Launches Website to Connect CF Community April 15, 2021April 15, 2021 To mark its 10th anniversary, Claire’s Place Foundation has launched a website that seeks to ...
News ‘Fun’ Sweat-measuring Sticker May Aid CF Diagnosis in Children April 14, 2021April 14, 2021 A soft skin sticker that can rapidly measure chloride levels in sweat, which are elevated ...
Being a Parent Will Kill You (and Other Truths We Never Talk About) April 13, 2021April 8, 2021 I am writing this from my bedroom — trapped. Like a character in a movie. ...
CF Therapy’s Effectiveness With Rare Mutations Can Be Determined in Lab April 13, 2021April 13, 2021 An ex vivo technique using nasal epithelial cells from a young girl carrying a rare ...
I’m Talking About My Latest Surgery, So Read This Column April 6, 2021April 2, 2021 I just had surgery again, and now more people will follow me. I don’t want ...
Olgram Raises $1.77M to Develop New Class of Antibiotics Against Resistant Bacteria April 6, 2021April 6, 2021
One Walk/Run ‘Shuttle Test’ Captures Exercise Capacity in CF Children, Study Says April 2, 2021April 2, 2021 Disease progression and the overall health of young people with cystic fibrosis ...
New LungQ Software Can Analyze CF Airway Problems in Seconds, Thirona Says April 1, 2021April 1, 2021
Columns, Life in the Grey – a Column by Bailey Anne Vincent Unpopular Opinion: I Know What I Need, but the Healthcare System Doesn’t March 30, 2021March 26, 2021 This is what I need. I’m going to tell you instead of my doctors, because ...
News, syndicated Cost of Rare Diseases in US? Nearly $1 Trillion in 2019, EveryLife Foundation Finds March 30, 2021March 30, 2021 As anyone affected by a rare disease knows, treating the illness while trying to go ...
cystic fibrosis Australia Approves Trikafta for Patients, 12 and Older, With Common Mutation March 30, 2021March 30, 2021 Health regulators in Australia have approved Trikafta (elexacaftor/tezacaftor/ivacaftor) to treat people with cystic fibrosis (CF), ...
News CHMP Favors Kaftrio to Treat CF Patients in EU Starting at Age 12 March 29, 2021March 29, 2021 A European Medicines Agency (EMA) committee favors extending the label for Kaftrio (ivacaftor/tezacaftor/elexacaftor), in combination with Kalydeco (ivacaftor), ...
Columns, Valiant Voice – a Column by Lara Govendo Every March, My Body Remembers the Trauma of Almost Dying March 26, 2021March 24, 2021 March is a heavy month. Like clockwork, physical and emotional symptoms seem to erupt out ...
cystic fibrosis Trikafta Found Safe, Effective for Children Ages 6–11 March 26, 2021March 26, 2021 Trikafta, a medication that combines three different therapies (elexacaftor/tezacaftor/ivacaftor), is safe and effective in children ...
cystic fibrosis CF Families in Ontario Send Video Plea for Access to Vertex Therapies March 25, 2021March 25, 2021 Parents of children and young adults with cystic fibrosis (CF) in Ontario sent a video ...
Columns, Victorious - a Column by Brad Dell My Life (and Death) Is More Than Statistics and Dead Dreams March 24, 2021April 13, 2021 It’s not my birthday or my lung transplant anniversary or anything. But today, I’m thinking ...
News Enrollment Complete in Phase 2 Trial Testing MS1819-PERT Combo for EPI March 24, 2021March 24, 2021 Patient enrollment has been completed for a Phase 2 clinical trial testing the investigational therapy ...
Columns, Life in the Grey – a Column by Bailey Anne Vincent Is Gender Bias in Healthcare Shortening My Life? March 23, 2021March 19, 2021 “I am sick of being a woman,” I say far too often. “Especially a sick ...
cystic fibrosis, News Translate Bio’s MRT5005 Shows Safety and Tolerability at Multiple Doses in Trial March 23, 2021March 23, 2021 Translate Bio’s ongoing Phase 1/2 clinical trial investigating MRT5005, its candidate inhalation therapy for cystic fibrosis ...
News Bacterial Fatty Acids May Play Role in Exacerbations in CF Children March 22, 2021March 22, 2021 Due to persistent bacterial infections, long-chain fatty acids (LCFA) are linked to continued inflammation in ...
cystic fibrosis Computer-designed Enzyme May Help to Kill P. aeruginosa in Biofilms March 19, 2021March 19, 2021 An enzyme produced using computer design, in combination with the antibiotic tobramycin, efficiently kills Pseudomonas ...
cystic fibrosis, News Bronchitol, Inhaled Powder for CF Mucus Clearance, Arrives in US March 18, 2021March 18, 2021 Bronchitol (mannitol), an inhaled mucus-clearing treatment for cystic fibrosis (CF), is now commercially available in ...
News Phase 2b Trial of MS1819, Yeast-based EPI for CF, Fully Enrolled March 17, 2021March 17, 2021 AzurRx BioPharma announced that its Phase 2b trial evaluating the safety and efficacy of MS1819 in ...
Columns, Life in the Grey – a Column by Bailey Anne Vincent Chadwick Boseman Is Still Dead, and We Need to Talk About It March 16, 2021March 11, 2021 Chadwick Boseman is dead, and I’m going to write about it. He has been dead ...
cystic fibrosis, News CF Foundation Continues Free Access to Online Exercise Platform March 16, 2021March 16, 2021 Thanks to the Cystic Fibrosis Foundation (CFF), people with cystic fibrosis (CF) in the United ...
News New Gene-editing Tool Can Reverse Specific CF-related Mutations March 15, 2021March 16, 2021 A precise gene-editing tool that reverses single DNA mutations may lead to genetic therapies for ...
