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Solid Biosciences announce preliminary trial results

February 12, 2019 by abzali123

Solid Biosciences have announced preliminary data from their phase 1/2 IGNITE-DMD clinical trial that is testing the safety and effectiveness of …

Solid Biosciences announce preliminary trial resultsRead More

HOPE-2 update from Capricor Therapeutics

January 23, 2019 by abzali123

Following meetings with the FDA (the medicines regulator in the US), Capricor Therapeutics yesterday issued an update yesterday on it’s HOPE-2 …

HOPE-2 update from Capricor TherapeuticsRead More

What your money buys

January 22, 2019 by abzali123

If you're supporting one of our many Duchenne research projects, here's an idea of where your money goes, and how every penny …

What your money buysRead More

Edasalonexent update from catabasis

December 24, 2018 by abzali123

Catabasis has announced that the phase 3 trial of edasalonexent is now recruiting at 9 centres across the USA and that regulatory approval for the …

Edasalonexent update from catabasisRead More

Sarepta completes accelerated approval application for golodirsen

December 20, 2018 by abzali123

Sarepta therapeutics has today announced that they have completed the submission of a USA application for  accelerated approval of golodirsen …

Sarepta completes accelerated approval application for golodirsenRead More

Wave Life Sciences announce phase 1 trial results

December 6, 2018 by abzali123

In a press release published earlier, Wave Life Sciences has announced positive results of the phase 1 trial of the WVE-210201 – a potential …

Wave Life Sciences announce phase 1 trial resultsRead More

Harry Hill’s ‘Comedians Sing Christmas Songs’ for Action Duchenne was a hit!

December 5, 2018 by abzali123

A huge thank you to our patron, Harry Hill, who hosted one of Action Duchenne's biggest ever events last night. 'Comedians Sing The Christmas Hits' …

Harry Hill’s ‘Comedians Sing Christmas Songs’ for Action Duchenne was a hit!Read More

ENMC Workshop in Amsterdam

December 3, 2018 by abzali123

ENMC has a unique concept of facilitating and organizing workshops for experts in the field of neuromuscular conditions. Every month a group of 20 - …

ENMC Workshop in AmsterdamRead More

Santhera acquires rights to vamorolone

November 21, 2018 by abzali123

Santhera has announced that they have acquired the rights to vamorolone - a potential drug that has been developed and is currently being tested in …

Santhera acquires rights to vamoroloneRead More

Comedians sing the Christmas hits!

November 14, 2018 by abzali123

It's gonna be one unholy night of fun! We are delighted to announce that on 4th December 2018, our patron Harry Hill will be hosting a comedy …

Comedians sing the Christmas hits!Read More

Catabasis starts Phase 3 trial of edasalonexent

September 28, 2018 by abzali123

Earlier this week Catabasis announced that a Phase 3 trial of edasalonexent in Duchenne has started, with trial centres to open shortly – first …

Catabasis starts Phase 3 trial of edasalonexentRead More

Sarepta receives negative opinion following CHMP review of eteplirsen

September 21, 2018 by abzali123

We've just heard the disappointing news that Sarepta has received a negative opinion of eteplirsen following a second review by the European …

Sarepta receives negative opinion following CHMP review of eteplirsenRead More

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