On the 30th November Edgewise Therapeutics Inc. (a leading muscle disease biopharmaceutical company) announced that the U.S. Food & Drug …
United Nations Officially Designates September 7th as World Duchenne Awareness Day
The global rare disease community is celebrating the UN’s first formal acknowledgement of a day dedicated to a rare disease. In a groundbreaking …
Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003
Solid Biosciences Receives FDA Clearance for Duchenne Muscular Dystrophy Gene Therapy Candidate SGT-003 Solid Biosciences Inc., a leading life …
Stakes ‘couldn’t be higher’ – PTC CEO on propect of Translarna losing EU conditional authorisation
Stakes 'couldn't be higher' – PTC CEO on prospect of Translarna losing EU conditional authorisation PTC Therapeutics' Translarna, a critical …
Thank you for attending the Action Duchenne Annual International Conference 2023!
Welcome to the Annual Action Duchenne International Conference 2023
Welcome to the Annual Action Duchenne International Conference 2023 On behalf of the whole team at Action Duchenne, I would like to give you the …
Welcome to the Annual Action Duchenne International Conference 2023Read More
Update on EMBARK from Roche
On October 31, 2023, Roche provided a community letter to share timely updates about Roche Duchenne muscular dystrophy (DMD) research efforts. The …
Sarepta Therapeutics Announces Topline Results from EMBARK, a Global Pivotal Study of ELEVIDYS Gene Therapy for Duchenne Muscular Dystrophy
Sarepta Therapeutics, Inc. (NASDAQ: SRPT), the leader in precision genetic medicine for rare diseases, announced topline results from EMBARK (Study …
Edgewise Therapeutics Announces Expansion of their EDG-5506 Clinical Program in Duchenne Muscular Dystrophy (Duchenne)
Edgewise Therapeutics Announces Expansion of their EDG-5506 Clinical Program in Duchenne Muscular Dystrophy (Duchenne) (Please bear in mind that …
Santhera Receives Positive CHMP Opinion Recommending Approval of AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy
Santhera Receives Positive CHMP Opinion Recommending Approval of AGAMREE® (vamorolone) for the Treatment of Duchenne Muscular Dystrophy Ad hoc …
Our new Information and Support Pack for Schools is here and don’t miss our online Turning Point sessions for 8-14 year olds!
Our new Information and Support Pack for Schools is here and don't miss our online Turning Point sessions for begins for 8-14 year olds! We have …
Thank you to everyone who was part of Science on Tour 2023
Thank you to everyone who was part of Science on Tour 2023 We have loved every minute of meeting so many of you. We have shared knowledge, …
Thank you to everyone who was part of Science on Tour 2023Read More
