REGENXBIO announced new positive interim data from the Phase I/II AFFINITY DUCHENNE trial. Updates include positive functional, safety and biomarker …
PepGen Discontinues Development of PGN-EDO51 for Duchenne Muscular Dystrophy
We are sharing an important update with our community regarding PepGen Inc.'s investigational therapy, PGN-EDO51, for Duchenne muscular dystrophy …
PepGen Discontinues Development of PGN-EDO51 for Duchenne Muscular DystrophyRead More
Entrada Therapeutics Receives Authorisation for New DMD Clinical Trial in UK and Europe
Entrada Therapeutics, Inc., today announced a significant step forward in their Duchenne muscular dystrophy (DMD) clinical development programme. They …
Entrada Therapeutics Receives Authorisation for New DMD Clinical Trial in UK and EuropeRead More
European Medicines Agency Issues Positive Opinion on Givinostat
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 …
