Policy and campaigning

The MHRA invite patients and carers to contribute to discussions on whether to maintain positive scientific opinion for Raxone

call to action – We need your testimony. We particularly need to hear from you if you are receiving Santhera’s drug Raxone under the Early Access to Medicine Scheme (EAMS), as part of the SIDEROS clinical trial or would like access Raxone for yourself or child. 

You can make your testimony in person at the UK’s Medicines & Healthcare products Regulatory Agency (MHRA) on the 23rd of March or in writing or by a video by the 19th of March.

A brief overview of the Situation:

The MHRA is currently reconsidering the Early Access to Medicine Scheme (EAMS) positive scientific opinion for Santhera’s drug Raxone following the European Medicine Agencies (EMA) recommendation against the marketing approval of Raxone in September 2017 and again in January 2018, following a re-examination of data. The MHRA are inviting patients and carers to contribute to discussions to decide whether they should maintain their positive opinion.

We are working with Action Duchenne, DMD Pathfinders, The Duchenne Family Support Group and MDUK to collect testimonies.

Please get in touch with us if you need any help making a submission. Written and video submissions can be sent to [email protected]  We will work with our fellow charities to collate and submit all the submissions together. If you would like to attend the meeting in person please also get in touch directly.

Published on 3 December 2018

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