In May 2019 Santhera Pharmaceuticals submitted an application for a European Marketing Authorisation for Puldysa® (idebenone) for the treatment of respiratory dysfunction in patients with Duchenne muscular dystrophy (DMD).
NICE (National Institute of Clinical Excellence), the organization responsible for deciding what treatments are recommended for funding by the NHS, have now initiated the process for the appraisal of idebenone in England.
One of the key questions which needs answering as part of this review is how many people may be eligible for treatment with idebenone. This questionnaire has been developed to help answer this question.
https://www.surveymonkey.co.uk/r/JSGX2ZT
The survey is only to be completed by those residing in England and we would be immensely grateful if it could be completed by the end of February.
Thank you for taking the time to complete this survey.
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