Glossary of organisations and what they do:

Information on important organisations and regulators.Read more

Food & Drug Administration’s (FDA) Open Public Hearing

Emily and Alex were among 42 parents who travelled to the US in April 2016, to witness the Adcomm for Eteplirsen, an exon skipping drug being developed by Sarepta Therapeutics. Read more about this in our news section.Read more

Empower: Data4Health

Duchenne UK attended the launch of the Empower: Data4Health campaign. The campaign is calling for a central database of health records that empowers patients, doctors and researchers, and helps to deliver better treatment across the country.Read more

European Medicines Agency (EMA)

It is important to engage with the regulatory process at European level, and Alex was involved in an exon skipping workshop to engage all stakeholders at the EMA. A paper will shortly be published on the outcomes of the meeting.Read more

The Campaign For Early Access To Medicines

We’ve lobbied with the Empower campaign, which has close links to the Minister for Life Sciences, George Freeman MP, for earlier access to medicines.Read more