About Duchenne Current Drugs For Duchenne Steroids Steroids are currently the only medication proven to slow down the progression of Duchenne. They have been prescribed to children with Duchenne for more than 20 years and are now part of the recognised standards of care. The type of steroids used are ‘corticosteroids’ (also known as ‘glucocorticoids’). They are not the same as the anabolic steroids used by some body builders. They’re thought to help in Duchenne due to their anti-inflammatory properties and by improving muscle regeneration. Prednisolone and deflazacort are the two types of corticosteroid prescribed for Duchenne. They each have pros and cons which can be discussed with your doctor, but since prednisolone is much cheaper, this is usually tried first. Steroids are usually started sometime between the ages of four and six, when motor skills have stopped improving, but have not yet started to decline. There is recent evidence that starting earlier is more effective but this must be weighed against the risk of side effects. Steroids are recommended in the international standards of care guidelines for Duchenne because of their ability to slow the progression of weakness, reduce the development of scoliosis (curvature of the spine) and delay breathing and heart problems. On average children taking steroids are able to walk for three years longer. However the long-term use of steroids can cause a number of possible side effects: the most common being weight gain, mood changes (irritability and hyperactivity) and a round, puffy face. Some of the other possible side effects include difficulty sleeping, headaches, stomach irritation, growth suppression, delayed puberty, raised blood pressure, cataracts, bone fractures and increased susceptibility to infection. After starting steroids your doctor will monitor the benefits and side effects. If the side effects are outweighing the benefit, the dose can be altered or tapered off completely. It is very important to not suddenly stop taking corticosteroids; their dose needs to be reduced slowly. There are medicines that can be given to offset some of these side effects. Metformin can be given to adolescents to reduce cushingoid. Testosterone is also given to induce puberty. And there are a number of treatments to help increase bone strength. All routine immunisations should be given before starting corticosteroids Make sure you ask your consultant what you should do if you forget a dose and where to get advice if needed. Always carry spare packets with you if you are going abroad, Be aware that a sudden stressful event such as illness, an accident or surgery, may require a higher dose of steroids for a short time. However, do not change the dose without discussing with your doctor first. For further information we recommend this comprehensive guide for families, published by Treat NMD, PPMD, UPPMD & MDA.