On the 25th of April, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) gave Givinostat (Duvyzat) a …
European Medicines Agency Issues Positive Opinion on GivinostatRead More
MindJam – The Benefits and Possibilities of Gaming
MindJam - The Benefits and Possibilities of Gaming MindJam provide emotional and SEN support for young people through gaming, game design and …
Expressing our gratitude to Pope Francis
Following the sad news of the death of Pope Francis on 21st April 2025, we want to express our gratitude for the ongoing support he offered the …
European Commission Confirms Decision Not to Renew the Market Authorisation for Translarna (Ataluren).
The European Commission (EC) has adopted the opinion of the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency …
Roche’s update on clinical hold in Duchenne muscular dystrophy
Roche’s update on clinical hold in Duchenne muscular dystrophy On the 18th March Sarepta announced the sad news regarding a young man with …
Roche’s update on clinical hold in Duchenne muscular dystrophyRead More
Family Science Education Day
Family Science Education Day On Saturday 22nd March, we welcomed some of our amazing families to our Family Science Education Day at the Think Tank …
Wave Life Science Announce Positive Data from FORWARD-53 Clinical Trial
Wave Life Sciences announced today positive data following 48 weeks of dosing in the FORWARD-53 clinical trial evaluating the investigational molecule …
Wave Life Science Announce Positive Data from FORWARD-53 Clinical TrialRead More
Dyne Therapeutics Announce New Long Terms Clinical Data from DELIVER Trial of DYNE 251
Dyne Therapeutics recently announced new long-term clinical data from its ongoing Phase 1/2 DELIVER trial of DYNE-251 demonstrating sustained …
Dyne Therapeutics Announce New Long Terms Clinical Data from DELIVER Trial of DYNE 251Read More
Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201
Entrada Therapeutics Receives Authorization in the United Kingdom to Initiate ELEVATE-45-201, a Phase 1/2 Multiple Ascending Dose Clinical Study of …
Capricor Therapeutics Reports Positive Long-Term Results for Deramiocel in Treating Duchenne Muscular Dystrophy
This week Capricor Therapeutics announced positive long-term data from its' ongoing HOPE-2 open-label extension (OLE) clinical trial, highlighting the …
Action Duchenne’s Statement Regarding Sarepta’s Sad Announcement
We are deeply saddened to learn of the passing of a young man who received ELEVIDYS gene therapy for Duchenne muscular dystrophy. Our hearts go out to …
Action Duchenne’s Statement Regarding Sarepta’s Sad AnnouncementRead More
PepGen Announces Update to Phase 2 CONNECT2-EDO51 Study in Patients with DMD
PepGen's has released an update regarding the CONNECT clinical trials for PGN-EDO51 in Duchenne muscular dystrophy (DMD). PepGen has announced a …
PepGen Announces Update to Phase 2 CONNECT2-EDO51 Study in Patients with DMDRead More
