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Accelerated approval request for a new drug for a rare childhood muscle disorder

A drug for Duchenne Muscular Dystrophy (DMD), originally developed by BRC Theme Lead Professor Francesco Muntoni’s Consortium in the UK, has been filed by Sarepta Therapeutics for accelerated approval by the United States Food and Drug Adminstration (FDA).

Cannabidiol trial shows reduction in seizures for children with Dravet syndrome

A trial conducted in Europe and the USA has shown that cannabidiol – a drug derived from cannabis but with the psycho-active elements removed – reduces seizures in children with a form of drug resistant epilepsy, known as Dravet syndrome.

Role of important protein improves understanding of autoimmune diseases

Researchers – led by Great Ormond Street BRC-supported Professor Tessa Crompton in collaboration with the Paediatric Department at Oxford University – have identified the role of a key protein in normal development of the thymus, an important organ of the immune system. 

Steroid induced diabetes

Steroids are hormonal chemical messengers that are produced naturally in the body by the adrenal glands (which are just above each kidney) and by the reproductive organs. Man-made versions of these hormonal substances are used to treat a wide range of illnesses and medical conditions. 

Eating out

Fast food and takeaways are fun and easy. But most fast food is high in fat and salt. This grub should not be up for grabs often. Have it as a treat now and then, or for special occasions.

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