Drug trials can take an extraordinarily long time and even if a pharmaceutical is approved in one country, this does not necessarily mean that it will be available across the world. A new drug that has been designed to tackle a rare genetic condition has now been approved for use in the UK. The successful drug trial, run by doctors from Hampstead’s Royal Free Hospital and Great Ormond Street, means that those afflicted with Morquio A syndrome will be able to seek improved medical help and hopefully live better and healthier lives.
Morquio A syndrome only affects around 3,000 people in the entire developed world; there may be more sufferers in developing nations, but the true numbers are not known. The affliction means that individuals are missing a vital enzyme, resulting in serious damage to their bones. This can result in stunted growth and victims having severe problems with their movement. In many cases those affected by the syndrome do not grow taller than 3ft 3in, which is equivalent to just one metre; however, despite people’s growth stopping, their organs continue to mature to full adult size, resulting in victims suffering from huge internal pressure as their organs fight for space. The result of this is that many of those with Morquio A syndrome are confined to a wheelchair for movement. The majority of sufferers have a short life expectancy, with most individuals not outliving their 20s or 30s.
The latest drug could significantly help those afflicted. Elusulfase alfa is used to replace the missing enzyme; as children grow, this helps to reduce the degenerative conditions of the disease, allowing victims to grow up as normally as possible. Even the slightest reduction in bone deformation could allow individuals to grow larger, reduce the stress on their bodies and extend their life expectancy. Ita Vickery from Reigate, for example, hopes that her two children with the condition ‒ Olivia, 14 and Luke, 9 ‒ may be able to grow up normally and go onto to have healthy adult lives. Ms Vickery added that she hoped the drug would enable her children to outlive her.
Public use of the drug is still some way off, however. Though elusulfase alfa has been licensed for use in private practices, it is not widely available via the National Health Service (NHS). This means that, for now, sufferers of the condition will have to look to the private health sector first.
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