ATHANASIOU DIMITRIOS

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

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

.@VitalTransform 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

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 @eupatients @eupatientsforum bit.ly/37fugzh

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

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

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

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

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…

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…

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…

Thank you @YNatsis for the meaningful questions !

Great @DuchenneDay

Read my profile in Muscular Dystrophy News Today on how #Duchenne affects one #Greek family. @ParentProjectMD #endDuchenne @DuchenneUK @MitchelsJourney @mdahellas @uppmdduchenne bit.ly/2JUdhoZ

The new platform for paediatric rare diseases has arrived! Check out the new website here: share4rare.org 🚀 Big thanks to @omada_int @Melanoma_MRV @worldduchenne @la_UPC @SJDbarcelona_es @The_Synergist 💜 #CitizenScience #S4R_eu #PatientEngagement #Live #WebsiteLaunch

Vamorolone progress cofunded by foundations @ParentProjectMD @mdausa @DuchenneUK @alljoinjack @duchennemd @SOSsaveoursons @ActionDuchenne @CureDuchenne @RyansQuestOrg @alexswish @PietrosFight @Michaelscause1 @DuchenneRF twitter.com/vision_dmd/sta…

#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?

#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

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