Blog

Rare Disease Day 2022: Idiopathic Pulmonary Fibrosis

18/02/2022

Idiopathic Pulmonary Fibrosis (IPF) has an estimated prevalence of 13 to 20 per 100,000 people worldwide* and is a condition that typically affects people between the age of 50 and 70.  It is rarely seen in those under 50 years of age.  Symptoms of the condition develop gradually and can include a persistent dry cough, […]

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Rare Disease Day 2022: Cystic Fibrosis

18/02/2022

More than 70,000 children and adults are affected by Cystic Fibrosis (CF) worldwide.*  Cystic Fibrosis is a progressive, inherited disease which causes the Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) Protein to become dysfunctional.  This results in the protein being unable to move chloride to the cell surface with the consequence that the cell is then […]

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How are new drugs developed and tested?

16/02/2022

Many thousands of new drugs are developed every year. Before these can be administered by doctors and hospitals, they must undergo a thorough development and testing process. Drug development (also known as pharmaceutical product development) is a lengthy process, involving several important steps to ensure the drug is effective, safe and beneficial. It takes several […]

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Rare Disease Day 2022: Huntington’s Disease

11/02/2022

Huntington’s Disease is a genetically inherited disorder which affects the nervous system, causing progressive changes in movement, learning, thinking and emotions. It affects both men and women and although it is not prevalent in any specific population, Huntington’s Disease affects between 3 and 7 in 100,000 people of European ancestry globally*, making it a rare […]

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Rare Disease Day 2022: Niemann-Pick Disease

10/02/2022

Niemann-Pick Disease is a genetic disorder caused by autosomal recessive mutations in genes relating to lipid metabolism. The condition presents in childhood as a progressive inability to metabolise fats within cells, resulting in a build-up of fat within the cells.  Over time this causes progressive developmental and neurological problems. Many organs can be affected including […]

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Women in Science: Interview

10/02/2022

Karen McCusker, Associate Director in Marketing at Simbec-Orion, speaks to Dr Debra Davies and Romillie Cruz at Simbec-Orion on their experience of being women in science. I’m not sure where my interest in medicine came from or when it started. I often wonder if it was something to do with being diagnosed with asthma as […]

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What Are the Three Stages Of Clinical Trials?

03/02/2022

Clinical trials involve a series of studies that test the safety and effectiveness of drugs and other medical treatments. To ensure research is carried out thoroughly and safely, there are several different stages of clinical trials. In order to make sure the treatment is safe and effective for use in patients, it must successfully pass […]

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