With the licensing of Translarna (ataluren) the first ever drug to combat Duchenne muscular dystrophy there is growing pressure for the establishment of a neonatal screening programme for Duchenne alongside that already in place for sickle cell disease and cystic fibrosis.
Translarna (ataluren) which prompts the body to manufacture a protein that protects against muscle damage which is absent in children with the disease, has been licensed under special measures.
Experts are also calling for the new drug to be made available on the NHS as soon as possible. Translarna can be used in children with specific mutations aged over five who are able to walk.
DFSG have been involved in European workshops to discuss the ethical implications of new born screening and recognises that some families may not agree with this approach. We are however here to support all families with Duchenne recognising the fact that individual families may have different views on the management of the condition.
The article features DFSG newsletter editor and committee member Penny Southall and her son Dan. Read the full article here: http://www.telegraph.co.uk/women/womens-
The full article about the licensing of Translarna can be read here: