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Two approaches to treat Lysosomal Storage Diseases
Lysosomes are membrane-bound organelles found in most animal cells. They are responsible for treating cellular waste. Genetic mutations in lysosomal enzymes lead to lysosome malfunction and waste accumulation.
Generoso Andria – Enzyme and gene therapy for treating genetic disorders
Lysosomes are intracellular organelles that degrade and recycle ‘waste’ molecules. Lysosomal Storage Disorders (LSDs) comprise a large group of congenital metabolic diseases affecting altogether one in 8,000 newborns.
How many pills? Ask your genes
European researchers have looked how the analysis of your genes could help to determine the dosage for patients who need to take blood thinners.
Anke-Hilse Maitland van der Zee - Blood thinners at the right dose
Coumarins— blood thinners against thrombosis, or blood clotting, are amongst the most widely used drugs worldwide. Millions of mostly elderly people depend on them. But finding the right dosage is a painstaking process.
Better diagnostics for thrombosis are a matter of resources
Cost-effectiveness is paramount for any new medical technology. If pharmaceutical companies do not see a profit, new drugs will never see the light of day.
Malin Parmar - Reversing Pakinson’s by programming stem cells as neurons
Neurons, our brain cells, do not regenerate during our life. This makes neurodegenerative diseases, such as Parkinson's and Huntington's, difficult to treat.
Advances in Treating Huntington’s Disease
Juliet Ross is suffering from Huntington’s Disease, a rare neurological disorder that affects one in 10.000 people. Due to a genetic defect, her body produces a toxic protein that damages neurons in her brain .
Huntington's disease: A treatment in sight?
A treatment to directly fight Huntington’s disease could finally be in sight . Until now, therapies for this severely debilitating genetic disorder have only focused on alleviating physical and psychiatric symptoms.
Smart and personal: dietary advice
After personalised medicine, we are entering the era of personalised nutrition. But this approach requires testing before being applicable. This is what a new EU-funded research project, called food4me , is attempting to do.
Regenerating brains with stem cells
Curing hearts with stem cells
Gene therapy for inherited disorders
Fighting superbugs
November 18 th is the annual European Antibiotic Awareness Day . Its purpose is to highlight the increase of bacterial resistance to antibiotics on the continent .
Anti-allergy GM apples
Peanut, egg and soy are more common food known to trigger an allergic reaction, a problem affecting around 8% of children in the EU. Intuitively, you might not list apples as causing allergic reactions.
Prof. Thomas Klockgether: "Ataxia-sufferers are placing their hopes in research"
With a prevalence of about 1 in 10.000, Ataxia is considered a rare hereditary disease that attacks the central nervous system. As a result, it is not at the top of the list in terms of research topics for pharmaceutical companies.
Cystic Fibrosis: A European Cause
It strikes mainly children and young adults by causing abnormal fluid transport mainly in their lungs, pancreas and intestine. At its most advanced stage, cystic fibrosis still leaves no alternative other than lung transplant.
A New Light on Progeria
Whenever lamin mutations occur, the regulation of progenitor cell proliferation and differentiation in highly regenerative tissues, such as skin and muscles, spins out of control.
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