Our campaign aims to raise awareness of SMA & build a ¬£1 million Development Fund over the next 2 years. This will allow us to fund projects which speed up the clinical trials process for new drugs & enable us to continue our support of UK centres of excellence, as well as research into SMA.
It ran from 12:00 AM, 1 January 2015 to 12:00 AM, 1 December 2016
Registered Charity in England and Wales (1097765)
The translation of scientific advances into effective therapies for SMA is made difficult by issues such as the complex regulatory environment in Europe, variations in standards of care, patient enrolment, a narrow therapeutic window and a need for more sensitive biomarkers and outcome measures. Basic, translational and pre-clinical research projects also continue to need funding.
Our campaign will ensure that funds will be available quickly to address the need to find therapies for SMA and bring them through the difficult cliinical trials process and hence to the market.
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