This Morning viewers praise teen with cystic fibrosis who says dance is a 'drug' that saved his life
Dubbed a real life Billy Elliot, Tom Oakley, 18, from Liverpool, who appeared on This Morning today, fell in love with ballet at the age of seven and describes it as a 'drug' that he cannot live without. (Source: the Mail online | Health)
Source: the Mail online | Health - July 28, 2021 Category: Consumer Health News Source Type: news

A small molecule induces readthrough of cystic fibrosis CFTR nonsense mutations
(University of Alabama at Birmingham) An experimental drug reported in Nature Communications suggests that a " path is clearly achievable " to treat currently untreatable cases of cystic fibrosis disease caused by nonsense mutations. This includes about 11 percent of cystic fibrosis patients, as well as patients with other genetic diseases, including Duchenne muscular dystrophy,β-thalassemia and numerous types of cancers, that are also caused by nonsense mutations. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - July 19, 2021 Category: International Medicine & Public Health Source Type: news

Should the most vulnerable be shielding again - no matter what health chiefs say?
The Cystic Fibrosis Trust, Blood Cancer UK and Kidney Care UK, among others, have called for specific guidance for the most vulnerable after tomorrow's so-called Freedom Day. (Source: the Mail online | Health)
Source: the Mail online | Health - July 17, 2021 Category: Consumer Health News Source Type: news

Ex-Genentech principal scientist, husband plead guilty in trade secrets case; two others charged
The case centers on three blockbuster cancer drugs developed by Genentech as well as a cystic fibrosis treatment and spans the globe. (Source: bizjournals.com Health Care:Physician Practices headlines)
Source: bizjournals.com Health Care:Physician Practices headlines - July 8, 2021 Category: American Health Authors: Ron Leuty Source Type: news

A Disposable OPEP Device in Children With Cystic Fibrosis A Disposable OPEP Device in Children With Cystic Fibrosis
A novel daily disposable OPEP device may be as effective as reusable devices while eliminating the need for regular cleaning.BMC Pulmonary Medicine (Source: Medscape Today Headlines)
Source: Medscape Today Headlines - July 6, 2021 Category: Consumer Health News Tags: Pulmonary Medicine Journal Article Source Type: news

Cystic Fibrosis: Overjoyed family opens up about transformed life with wonder drug
AN overjoyed family poses with their new arrival a year after their lives were changed by a cystic fibrosis drug fought for by the Daily Express. (Source: Daily Express - Health)
Source: Daily Express - Health - July 5, 2021 Category: Consumer Health News Source Type: news

Whole genome sequencing of all UK newborns ‘would have public support’
Consultation shows positivity towards screening programme to spot those at heightened risk of certain health conditionsPlans to sequence the whole genome of every newborn in the UK in order to spot those at heightened risk of certain health conditions have been given a boost, with consultations suggesting the approach could have public support.The potential for genomics to improve health was at theheart of the chief medical officer annual report of 2016, with a group of experts convened by Genomics England – a government-owned genetics service – subsequently recommending a research programme to sequence the whole genom...
Source: Guardian Unlimited Science - July 4, 2021 Category: Science Authors: Nicola Davis Science correspondent Tags: Genetics Science UK news NHS Health Childbirth Sickle cell disease Cystic fibrosis Children's health Source Type: news

Diversity is a Living Thing
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Source: Johns Hopkins University and Health Systems Archive - June 29, 2021 Category: Nursing Source Type: news

What Types of New Therapies are Available for Cystic Fibrosis?
Discussion Cystic fibrosis (CF) was identified in 1938 by Dr. Dorothy Andersen who described 49 patients with pancreatic insufficiency. Since that time significant achievements in the knowledge about the disease and treatments for patients have changed the mortality from a few months to patients living into middle adulthood or even later. Quality of life for patients and their families and friends has also markedly improved. CF is the classic Mendelian autosomal recessive genetic disorder which is a worldwide disorder but affects people of north European ancestry more often where the main mutation is more common. There are...
Source: PediatricEducation.org - June 28, 2021 Category: Pediatrics Authors: Pediatric Education Tags: Uncategorized Source Type: news

Vertex Announces U.S. FDA Approval for Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) in Children With Cystic Fibrosis Ages 6 through 11 With Certain Mutations
BOSTON--(BUSINESS WIRE)--Jun. 9, 2021-- Vertex Pharmaceuticals Incorporated (Nasdaq: VRTX) today announced the U.S. Food and Drug Administration (FDA) approved expanded use of Trikafta (elexacaftor/tezacaftor/ivacaftor and ivacaftor) to include... (Source: Drugs.com - New Drug Approvals)
Source: Drugs.com - New Drug Approvals - June 9, 2021 Category: Drugs & Pharmacology Source Type: news

How strengthening the biosimilar marketplace benefits patients
Biologics are medicines that are made from living organisms through highly complex manufacturing processes and include a wide variety of medicines such as therapeutic proteins, monoclonal antibodies and vaccines. They are used to prevent or treat a variety of diseases including cancer, chronic kidney disease, diabetes, cystic fibrosis and autoimmune disorders.  (Source: The Catalyst)
Source: The Catalyst - May 18, 2021 Category: Pharmaceuticals Tags: FDA Drug Cost Biologics and Biosimilars Source Type: news

Health outcomes differ between UK and US children with cystic fibrosis
(University of Liverpool) A new study led by University of Liverpool researchers has confirmed that children with cystic fibrosis (CF) in the US have better lung function than UK children with the disease. The study suggests that differences do not appear to be explained by early growth or nutrition, but could be linked to differences in the use of early treatments. (Source: EurekAlert! - Medicine and Health)
Source: EurekAlert! - Medicine and Health - May 14, 2021 Category: International Medicine & Public Health Source Type: news

SpliSense Secures $28.5 Million in Series B Financing
Round includes up to $8.4 million investment by the Cystic Fibrosis Foundation Funds will be used to advance the Company's mRNA-altering pipeline for the treatment of cystic fibrosis and other genetic pulmonary diseases JERUSALEM, May 13, 2021 -- (He... Biopharmaceuticals, Venture Capital SpliSense, Cystic Fibrosis (Source: HSMN NewsFeed)
Source: HSMN NewsFeed - May 13, 2021 Category: Pharmaceuticals Source Type: news

Antimicrobial prescribing: nebulised liposomal amikacin - evidence summary [ES36], NICE (published 12th May 2021)
This concludes nebulised liposomal amikacin may be an option for treating non-tuberculous mycobacterial lung infections caused by Mycobacterium avium complex in combination with other antimicrobial agents in adults with limited treatment options& who do not have cystic fibrosis. (Source: Current Awareness Service for Health (CASH))
Source: Current Awareness Service for Health (CASH) - May 13, 2021 Category: Consumer Health News Source Type: news

Novel nanotech improves cystic fibrosis antibiotic by 100,000-fold
(University of South Australia) World-first nanotechnology developed by the University of South Australia could change the lives of thousands of people living with cystic fibrosis (CF) as groundbreaking research shows it can improve the effectiveness of the CF antibiotic Tobramycin, increasing its efficacy by up to 100,000-fold. (Source: EurekAlert! - Biology)
Source: EurekAlert! - Biology - May 13, 2021 Category: Biology Source Type: news