September 7 is World Duchenne Awareness Day. On this day, we raise awareness for Duchenne and Becker muscular dystrophy (DMD and BMD) around the globe. An initiative coordinated by the World [...]
Recent studies on Duchenne muscular dystrophy (DMD) have greatly deepened our understanding of the primary and secondary pathogenetic mechanisms. Guidelines for the multidisciplinary care for DMD [...]
We wish to emphasize that the information contained in this article comes mainly from the RQMO website, the CORD website and the Rare Disease Day website. We thank them. February 28th is Rare [...]
Sarepta Therapeutics announces top-line results for part 1 of study 102 evaluating SRP-9001, its investigational gene therapy for the treatment of Duchenne muscular dystrophy (DMD) Jan. 7, [...]
Giving Tuesday – the global day of giving Join us for the 8th annual Giving Tuesday on December 1rst, 2020! GivingTuesday is a global day of giving that happens each year after Black Friday and [...]
September 7 is World Duchenne Awareness Day. On this day, we raise awareness for Duchenne and Becker muscular dystrophy (DMD and BMD) around the globe. An initiative coordinated by the World [...]
Sarepta Therapeutics signs agreement with Hansa Biopharma for imlifidase. This exclusive worldwide license enables Sarepta to develop and promote imlifidase as a pre-treatment for gene therapy in [...]
WHAT WE KNOW ABOUT COVID-19 What do we know about COVID-19 for people affected by Duchenne or Becker? Due to the increasing concerns regarding the COVID-19 virus for people with Duchenne and [...]
Key Note Speaker: Dr. Jacques P. Tremblay Presented by PTC Therapeutics and in partnership with Jesse’s Journey and La Force DMD, this unique educational event provides the opportunity for [...]
February 29, 2020, will be the thirteenth international Rare Disease Day coordinated by EURORDIS. On and around this day hundreds of patient organizations from countries and regions all over the [...]