We recently wrote to the Medicines and Healthcare Products Regulatory Agency (MHRA) - the UK drug regulator - to highlight the importance of …
Vamorolone trial results published
Earlier this week the results of an early trial of vamorolone in boys with Duchenne have been published in the Neurology scientific journal. …
Ignite DMD update
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
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