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The DECODR app was developed to accelerate the development of CRISPR gene therapies, producing similar data as deep sequencing processes in a shorter timeframe and at less cost.
Bhavesh Patel, Cécile Matthews and Owen Male at CRA’s Life Sciences Practice assess the relationship between drug pricing for rare disease therapies and disease prevalence in Europe, taking a closer look at payer assessments of orphan drugs in Germany.
A clinical trial has shown that antibiotics administered orally are just as effective at killing Pseudomonas aeruginosa in cystic fibrosis patients as intravenous antibiotics.
The European Commission and European Medicines Agency have approved the triple combination Kaftrio for use in certain cystic fibrosis patients, triggering the drug to be accessible on the UK’s National Health Service.
Dapivirine Vaginal Ring (dapivirine) and Blenrep (belantamab mafodotin) have been recommended for approval by the EMA's CHMP, along with nine other medicines.
The European Medicines Agency's human medicines committee (CHMP) said the treatment would offer a therapeutic option for certain cystic fibrosis phenotypes that are currently untreatable.
AbbVie and Allergan have announced the Federal Trade Commission has accepted AbbVie's pending acquisition of Allergan for $63 billion.
New research from PhRMA found that in the US, there are almost 600 paediatric medicines in development in over 2,000 clinical trials.
Researchers at Aston University and Birmingham Children’s Hospital, UK, have discovered a highly effective three-drug approach to treat Mycobacterium abscessus (M. abscessus), a notoriously drug-resistant pathogen that affects up to 13 percent of cystic fibrosis (CF) patients in the UK.
The European Commission has approved the label extension for KALYDECO® (ivacaftor) to include the treatment of infants with cystic fibrosis between six and 12 months old.
Some pharmaceutical manufacturers can struggle to keep microorganisms away from their facilities, which sometimes goes unnoticed until it is too late. While there are numerous threatening microorganisms, this article focuses on some of the most troublesome, highlighting the hazards they pose and analysing how manufacturers can prevent microbial contamination in…
The application was supported by positive results from two global Phase III studies in people with cystic fibrosis aged 12 and older.
A new agreement will allow eligible cystic fibrosis patients in England to have access to CFTR modulators to treat the underlying cause of their disease.
Trikafta is the first approved treatment that is effective for patients 12 years and older with the most common cystic fibrosis mutation.