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To: NHS England

NHS England - Approve Translarna NOW!

Approve the use of a new drug developed in the USA - Translarna - to treat a particular strain of Duchenne Muscular Dystrophy.

Why is this important?

Will and Isaac Baker are brothers, aged 6 and 3, from Colchester, Essex. Their grandparents are active members of The Ark Methodist Church, and Will and Isaac's parents regularly bring Will and Isaac to Sunday School at The Ark. Both boys have a particular strain of Duchenne Muscular Dystrophy. PTC Therapeutics, based in the USA, have developed a drug - Translarna - which is the first drug ever to be developed specifically to treat the strain of Duchenne Muscular Dystrophy which both Will and Isaac have.

The drug has been approved in the European Union and in a small number of countries across the world. The drug has also been approved for a 6 month period for a boy in Scotland only last week. It is critical that NHS England approve this drug while the boys still have mobility and meet the prescribing criteria. Will, who is now 5 (his 6th birthday is next week), meets all the criteria to commence treatment immediately. Isaac is 3 yrs 7mths and hopefully will do so on his 5th birthday.


2015-10-22 21:26:27 +0100

25 signatures reached

2015-10-22 15:47:05 +0100

10 signatures reached