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
Roche Shares Top-line Results from Year Two of the EMBARK Phase 3 Clinical Trial.
Roche have provided an update on the latest top-line results from the second year of their EMBARK Phase 3 clinical trial (NCT05096221) investigating …
Roche Shares Top-line Results from Year Two of the EMBARK Phase 3 Clinical Trial.Read More
Calling all Siblings and Caregivers of Individuals with Duchenne
Help researchers and mental health professionals at the University of Newcastle better understand how Duchenne muscular dystrophy (DMD) impacts …
Calling all Siblings and Caregivers of Individuals with DuchenneRead More
Vamorolone for Duchenne Muscular Dystrophy: A Summary of Evidence So Far
DMD Care UK will host a webinar to summarise the evidence for vamorolone as a treatment for Duchenne muscular dystrophy (DMD), and provide an …
Vamorolone for Duchenne Muscular Dystrophy: A Summary of Evidence So FarRead More
Open Letter from the Chair and CEO of Action Duchenne for Rare Disease Day 2025
Dear Friends, Supporters, and Advocates, On Rare Disease Day 2025, we stand together to raise awareness, celebrate our community, and drive …
Open Letter from the Chair and CEO of Action Duchenne for Rare Disease Day 2025Read More
