Role: Head of Fundraising and Communications Responsible To: CEO Hours: 22.5 hours per week (0.6 FTE) Location: Remote, with in-person Team …
Edgewise Therapeutics Reports Positive Results on Sevasemten Program for Becker and Duchenne Muscular Dystrophies
Edgewise Therapeutics has shared positive results in its Sevasemten program for Becker and Duchenne muscular dystrophies. Highlights include:● New …
We’re Hiring: Support Team Lead
Support Team Lead Role: Support Team Lead Responsible To: Head of Operations & Events Hours: 30 hours (0.8 FTE) Location: Remote, …
Yes I Can Activity Day – Scotland
Yes I Can Activity Day - Scotland Are you aged 12 and over, living with Duchenne and live in Scotland? Join Action Duchenne in Scotland …
Could you become a mentor for young people living with Duchenne?
Do you have lived experience of Duchenne, have a skill you want to share, pass on practical tips about developing hobbies, moving into further …
Could you become a mentor for young people living with Duchenne?Read More
REGENXBIO reports new positive functional data from Phase I/II Affinity Duchenne Trial of RGX-202
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 …
