Vamorolone progress cofunded by foundations Parent Project Muscular Dystrophy (PPMD) MDA USA Duchenne UK Joining Jack DuchenneMD Save Our Sons Action Duchenne CureDuchenne Ryan's Quest @alexswish Pietro's Fight Michaelscause DuchenneResearchFund x.com/vision_dmd/sta…

#EMA carried out a total of 925 activities involving #patients and 445 activities involving #HealthcareProfessionals during 2017. Read more in our latest Stakeholder Engagement report: bit.ly/2IvNnaf

The new platform for paediatric rare diseases has arrived! Check out the new website here: share4rare.org 🚀 Big thanks to Òmada Interactiva Melanoma MRV World Duchenne Organization Universitat Politècnica de Catalunya (UPC) Hospital Sant Joan de Déu Barcelona ES The Synergist 💜 #CitizenScience #S4R_eu #PatientEngagement #Live #WebsiteLaunch

📣Make sure to register at the best rate! 2 days left to benefit from Early Bird rates for the Joint #EFGCP & Drug Information Association (DIA) Better Medicines for Children Conference: efgcp.eu/cgi?lg=en&pag=… #Paediatrics2018

#ppmdconference there was mention about the FDA being prepared to extrapolate from trial populations to wider populations. The EMA seems less so, as well as payers. How can we address this with pharma?

Read my profile in Muscular Dystrophy News Today on how #Duchenne affects one #Greek family. Parent Project Muscular Dystrophy (PPMD) #endDuchenne Duchenne UK Mitchell's Journey mdahellas @uppmdduchenne bit.ly/2JUdhoZ

Great World Duchenne Awareness Day 🎈

Thank you Yannis Natsis for the meaningful questions !

Delighted to see strong media coverage of our new study across Europe At a glance, here's how Europe's top 30 medical universities performed on #clinicaltrials transparency transparimed.org/single-post/20…

When the medicine your child needs is priced too high for the NHS what do you do? #CFBuyersClub #OrkambiNow bbc.co.uk/news/health-48…

Three questions yesterday EFPIA dinner: 1 What does #EU contribute to #health & care? 2 Are we adding the maximum value to #patients, families, & healthcare workers? 3 Can we do more to lessen the burden on health systems today & in the future? Answers 👉europa.eu/!bQ79GB

#FDA officials see 'missed opportunity' with patient outcomes in clinical trials: FDA has shown an interest in PROs,: The information is seen as a valuable tool for improving clinical trial designs, guiding drug approvals & supporting label decisions. biopharmadive.com/news/fda-offic…

AddThis | Home doi.org/10.1016/j.nmd.…

European regulators’ views on a wearable-derived performance measurement of Ambulation for Duchenne Muscular Disease regulatory trials

On September 7, I bring my light for World Duchenne Awareness Day to raise awareness on #Duchenne muscular dystrophy. Join World Duchenne Awareness Day 🎈 for #WDAD2019 🎈 youtu.be/M8N6egJ3R80

Novartis has sold 100 lots of its gene therapy for spinal muscular atrophy so far this year at $1.75 million a shot. Not much uptake for annual payments. This is a disease with no other treatment. I do not believe these data will apply to #hemophilia. tiny.cc/cyozez

Do not miss this - Register and Share! Advancing patient-driven research in Share4Rare Join us in the Virtual Coffee with patient organisations on Tuesday, June 16, 16:00 CEST. EURORDIS-Rare Diseases Europe EUPATI European Patients' Forum bit.ly/37fugzh

.VitalTransformation is proud to be supporting the 1st Int Conference on Rare Diseases: “Building a Pathway from Diagnosis to Access” 1-2 March 2021 Info & Registration -> bit.ly/2MkWEd7 #rarediseasesconference21

September 7 is World Duchenne Awareness Day: Spreading Duchenne Muscular Dystrophy Awareness bit.ly/3jNlWyr #DuchenneMuscularDystrophy #WDAD2021 #RareDisease World Duchenne Organization

We waited far too long for the results of the For-DMD study, but here they are: jamanetwork.com/journals/jama/…