In a press release yesterday, Solid Biosciences gave a short update on their gene therapy trial called Ignite DMD. Following the decision earlier this …
Sarepta issues clinical trials update
In recent press releases, Sarepta Therapeutics has given updates on their ongoing clinical trial programmes. These include exon skipping and gene …
3 months until the Action Duchenne International Conference 2019
We are super excited here at AD HQ that there are only 3 short months to go before #ADConf19! We will begin to announce our speakers tomorrow who will …
3 months until the Action Duchenne International Conference 2019Read More
Online learning tool for kids
We have teamed up with Purple Mash to offer you access to a fantastic online learning tool for primary-aged kids (under 12). Your children log in …
Gene silencing and Duchenne
You might have seen news stories about gene silencing, and how the NHS has agreed to fund it as a treatment for a condition called amyloidosis. …
Simon Dadd new trustee
We are delighted to announce that Simon Dadd has joined our Board of Trustees. Simon has considerable knowledge of Duchenne, being the father of two …
Capricor publish interim results of HOPE-2 trial
Capricor therapeutics has announced the results of their interim analysis of the phase 2 trial of CAP-1002 in Duchenne. The trial has recruited 17 …
Rare disease genomes event
Last week Neil attended a “demystifying genomes for patient registries” event hosted by Sano genetics. The event was meant to give charities in the …
PM launches new drive to tackle barriers faced by disabled people
In June, the outgoing Prime Minister Theresa May announced a series of measures aimed at securing higher accessibility standards for new housing and …
PM launches new drive to tackle barriers faced by disabled peopleRead More
Conference registration open
Sign up today for the most amazing weekend of knowledge, power, support, education, community and hope. We've worked tirelessly to bring you the …
MARVEL headline sponsor for Superhero Series
The Superhero Series - the UK’s only mass-participation sports series dedicated to people with disabilities – will have a powerful boost this year, as …
Conference agenda released
We are excited to release our long awaited Agenda for #ADconf19! We have carefully aligned our Agenda this year with our three core objectives - …
