This week's update on our fast moving community campaign for access to givinostat
Following the wonderful news on Friday that the Medicines and Healthcare products Regulatory Agency (MHRA) approved givinostat as a treatment for Duchenne muscular dystrophy (DMD) for people who are ambulant and conditional approval for people who are non-ambulant, our campaign for access for everyone goes on. Here is an update.
Webinar
Dr Adnan Manzur, clinical leader of the expert NorthStar network, joined us for a webinar on Thursday, where he gave an update on the givinostat Early Access Programme (EAP), as well as the latest news on the treatment.
We also gave an update: on the incredible achievements you’ve made during this campaign! More than 200 letters have gone to the MHRA, over 30 politicians have been engaged with (including a Secretary of State), and 3 written questions have been submitted to the Department for Health and Social Care. And perhaps most important of all, at least 25 Trusts are engaging to some extent with the company on the EAP. We will get more! Duchenne UK won’t stop until everyone who is eligible for the EAP gets access.
If you missed it, the webinar was recorded and you can watch it back on YouTube here.
Parliamentary drop-in
Uptake of the EAP within the NHS is too slow, and it needs to be fixed. During the webinar we announced that we’re taking this issue directly to MPs in the new year with an event in Parliament. The event is planned to take place on Wed 22 January, the same day as Prime Minister’s Questions (PMQs). We’ll be sharing a template letter soon which you can send to your local MP asking for two things; that they try and ask a question at PMQs about givinostat, and that they come along to the event.
If you get a positive reply from your MP (and capacity allows it), you’ll be very welcome to come to London to meet your MP at the drop-in, so you can explain to them in person why this campaign matters, and what it’s like living with Duchenne. Keep an eye out for future messages from us with the details.
DMDhome
The DMDhome is an important study which needs volunteers! It’s an exciting project finding out whether we can use digital end points for clinical trials which can be assessed at home, and hopefully making it even easier for researchers to investigate DMD treatments.
To learn more, check out the study’s website here, or on the DMD Hub here.
Time is muscle
And as always, a little reminder of what else you can do. We have been urging everyone to write to their local NHS trust’s Chief Executive to ask them to make sure your neuromuscular clinic can take part in the EAP, and to write to your MP to ask them to make appeals to the Department of Health and Social Care on your behalf. Find templates for all three below: