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On Friday, 22 September, PTC Therapeutics, the company that manufactures Translarna, issued a…
News
Translarna currently has a conditional marketing approval (authorisation) from the EMA, given in…
Blog
Reducing inequalities
Tackling health inequalities is a long-standing aim of both the UK…
Publication
It is only through your help that we are able to fund vital research, and offer care and support to…
News
Today NHS England have released new data which shows nearly three in four babies born with Spinal…
News
Newborn babies in the UK are currently screened for nine – soon to be ten – rare, but serious,…
News
Zolgensma (Onasemnogene abeparvovec) is now recommended as an option for treating presymptomatic…
News
We were pleased to hear Rt Hon Jeremy Hunt MP confirm that the Energy Price Guarantee rise has been…
Blog
The Chancellor of the Exchequer, Rt Hon Jeremy Hunt MP, made an Autumn statement in the House of…
News
In the Chancellor’s Autumn Statement on 17 November 2022, we were pleased to hear commitments to…