Tag Archives: foetal haemoglobin

A long life with sickle cell disease

Sickle cell disease is often described as a “life limiting disorder”. Not so long ago children and teenagers were sometimes told that they would be “lucky to see their 21st birthday”. In many parts of the world it remains the … Continue reading

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Knock down of BCL11A

There is great excitement in the sickle cell world at the moment about, what may turn out to be, a turning point in the the long road to find a practicable cure for sickle cell. A recent paper from the … Continue reading

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More on asthma and sickle cell

A team of French sickle cell doctors, from the Robert-Debre Hospital in the Sorbonne, Paris, have been studying a group of 375 babies identified at birth with sickle cell disease. Following them up carefully, recording what happens to them and … Continue reading

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Pomalidomide – more action on the foetal haemoglobin front

As many of you will know hydroxycarbamide is the only drug licensed for the treatment of sickle cell disease. It’s potential benefits in sickle cell were only discovered by accident, many years ago, when patients were being treated with the … Continue reading

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The foetal haemoglobin story just got more complicated

Many of us think that foetal haemoglobin (Hb F) is the key to a “cure” for sickle cell disease. The genes that make Hb F are switched off shortly after we are born and then remain inactive throughout the rest … Continue reading

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Sickle Cell and Body Builders

What have body building and sickle cell got in common? Well it’s a complicated story that starts nearly 40 years ago with the discovery of a simple chemical, nitric oxide. Nitric oxide is a very powerful vasodilator, meaning that it … Continue reading

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Gene Therapy

There is a growing interest in the medical world in gene therapy, for patients who have an inherited disease due to a single gene defect. Sickle cell disease fits this bill perfectly. The condition is caused by a mutation, or … Continue reading

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Homing in on BCL11A

I mentioned BCL11A in a previous blog (More on Haemoglobin F – 16/04/14). It is a protein molecule which is critically important for switching off the production of haemoglobin F (Hb F) after birth and for maintaining low levels of … Continue reading

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What are “HDAC” inhibitors?

HDAC inhibitors are a group of drugs, which have a wide range of actions, including the ability to modify the activity of genes. HDAC stands for “Histone De-ACetylase” and the HDAC inhibitors are drugs which inhibit this enzyme. They may … Continue reading

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Update on clinical trials in sickle cell disease

MST-188 Otherwise known as Purified Poloxamer 188, is a designated orphan drug by the US Food and Drug Administration (FDA) for trial in sickle cell disease. It is a synthetic co-polymer of polyoxyethylene (POE) and polyoxypropylene (POP) and is thought … Continue reading

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