Antisense Therapeutics have received approval from the Royal Children’s Hospital (RCH), Melbourne Human Research Ethics Committee, to …
Antisense receive approval for Phase II trial in AustraliaRead More
Recognising early signs of Duchenne videos featured on BBC Look North
One of our wonderful families were featured in the news this week, as 7 year old Oliver and his Mum, Alix were interviewed by BBC Look North. Watch …
Recognising early signs of Duchenne videos featured on BBC Look NorthRead More
Recognising Neuromuscular Disorders eLearning module is now live
E-learning resources support paediatricians and other child health professionals to achieve the essential competences in a number of important …
Recognising Neuromuscular Disorders eLearning module is now liveRead More
Shelley Simmonds joined Findacure on the radio to celebrate Rare Disease Day
One of our wonderful Trustees, Shelley Simmonds joined Findacure on Cambridge 105 to celebrate Rare Disease Day 2018. Listen to the whole …
Shelley Simmonds joined Findacure on the radio to celebrate Rare Disease DayRead More
Action Duchenne funded Sc-OT-DMD study recruited 91 people
Action Duchenne have co-funded the ScOT-DMD research study since December 2016. This study is only open to boys in Scotland and although sufficient …
Action Duchenne funded Sc-OT-DMD study recruited 91 peopleRead More
PTC Therapeutics marks Rare Disease Day 2018 with Duchenne and Me app
PTC Therapeutics proudly supports Rare Disease Day (Feb. 28) 2018 by commemorating 150 years since the signs and symptoms of Duchenne muscular …
PTC Therapeutics marks Rare Disease Day 2018 with Duchenne and Me appRead More
Action Duchenne CEO represents the community at ENMC International Workshop
Highlights The issue of translatability of pre-clinical results was discussed with a wide range of stakeholders. Challenges and pitfalls …
Action Duchenne CEO represents the community at ENMC International WorkshopRead More
NICE guideline publication – Adult social care: improving people’s experience
NICE guideline on people's experience in adult social care services: improving the experience of care and support for people using adult social care …
NICE guideline publication – Adult social care: improving people’s experienceRead More
Summit announces new analysis showing ezutromid significantly reduced muscle inflammation
Summit has announced further positive findings from PhaseOut DMD, a Phase 2 open-label, multi-centre clinical trial of the utrophin modulator …
Summit announces new analysis showing ezutromid significantly reduced muscle inflammationRead More
Skeletal and cardiac muscle improvements in MDX mouse
Cedars-Sinai Researchers Say Findings Lay Groundwork for Testing of CAP-1002 in Patients with Duchenne Muscular Dystrophy Capricor have today …
Skeletal and cardiac muscle improvements in MDX mouseRead More
Wales joins the 100,000 Genomes Project
Wales has joined the 100,000 Genomes Project, the Welsh Government announced. The project, now a UK-wide initiative, seeks to transform patient …
PTC receives formal dispute resolution request decision from the FDA
PTC have today announced that the Office of New Drugs of the U.S. Food and Drug Administration has reiterated the FDA's prior …
PTC receives formal dispute resolution request decision from the FDARead More
