PaLaDIn

(English below) A fianco della recente 22ma Conferenza Internazionale sulla distrofia muscolare di Duchenne e Becker, Parent Project ha programmato anche?un workshop dedicato al tema della #patientadvocacy?rivolto ad organizzazioni di pazienti internazionali. ?? L’evento ha visto la partecipazione di 27 persone in rappresentanza di associazioni di pazienti provenienti da 12 nazioni e si è focalizzato sul contributo dei pazienti nello?sviluppo dei farmaci?e nei?processi di Health Technology Assessment?(HTA). Tra i relatori: Ramona Belfiore-Oshan del Critical Path Institute (C-Path), Andrea Marcellusi dell'Università degli Studi di Milano, Fleur Chandler per Duchenne UK, Pat Furlong di Parent Project Muscular Dystrophy, Rebecca Leary e Joanne Lee della Newcastle University per il progetto PaLaDIn. ?? Il dibattito tra le varie associazioni ha evidenziato l’interesse condiviso per le tematiche trattate e l’importanza di un confronto tra realtà diverse. Si tratta di un primo importante passo, rispetto ai molteplici altri aspetti che sarà necessario affrontare in futuro. Il workshop è stato realizzato anche grazie al contributo non condizionato di?Roche, Edgewise Therapeutics, Sarepta Therapeutics e ITALFARMACO S.P.A.. Leggi la notizia qui ?? https://lnkd.in/d8ktUqSQ ------------ Alongside the recent 22nd International Conference on Duchenne and Becker Muscular Dystrophy, Parent Project also scheduled a workshop dedicated to the theme of #patientadvocacy aimed at international patient organizations. ?? The event was attended by 27 people representing patient organizations from 12 countries and focused on patient input in drug development and Health Technology Assessment (HTA) processes. Speakers included Ramona Belfiore-Oshan from the Critical Path Institute (C-Path), Andrea Marcellusi from the University of Milan, Fleur Chandler for Duchenne UK, Pat Furlong from Parent Project Muscular Dystrophy, and Rebecca Leary and Joanne Lee from Newcastle University for the PaLaDIn project. ?? The discussion among the various associations highlighted the shared interest in the issues addressed and the importance of a comparison between different realities. This is an important first step, compared to the many other issues that will need to be addressed in the future. The workshop was also made possible thanks to the unconditional contribution of Roche, Edgewise Therapeutics, Sarepta Therapeutics, and Italfarmaco. Read the news here ?? https://lnkd.in/d8ktUqSQ

TREAT-NMD held a couple of well attended and received sessions at the Muscular Dystrophy Association Conference on innovative global development within NMD. Brilliant insight into the European project, PaLaDIn by David Allison and then a fantastic overview of TACT (TREAT-NMD Advisory Committee for Therapeutics) by Lindsay Alfano. Please get in touch for more information. #mda2025 #nmd #treatnmd

David Allison of PaLaDIn scientific coordinator TREAT-NMD presented PaLaDIn at the Muscular Dystrophy Association conference in Dallas yesterday. David provided an introduction to the Interactium data platform, which will bring together different sources of data including clinical data from patient registries and data collected directly from patients. Linking together these different types of data will provide a more comprehensive view and natural history of neuromuscular diseases and could be used to support health care decision making. To hear more about the Interactium, click here: https://lnkd.in/eV-SFKzh

The first PaLaDIn General Assembly has now concluded! We would like to thank all beneficiaries for their very active participation over the past two days. This has been a fantastic opportunity to meet and discuss our next steps. We would like to say a special thank you to our External Advisory Board and Ethics Board respectively for their participation and feedback. We now look forward to continuing our collaboration with all beneficiaries to meet our vision of developing and implementing an innovative data collection platform to accelerate the development of effective treatments and patient care, enhance healthcare decision-making and improve patient outcomes. Keep up to date with our progress by subscribing to our newsletter here: https://lnkd.in/dQRxgPtx

The PaLaDIn General Assembly is taking place in Rome today and tomorrow. This is a great opportunity for all PaLaDIn beneficiaries to meet, discuss our progress and plan the next steps of the project. We are looking forward to some great discussions!

This weekend PaLaDIn scientific coordinator Parent Project aps held their annual International Conference for the Duchenne and Becker community. We were extremely grateful for the opportunity to join a workshop today on the importance of patient advocacy contribution in drug development and HTA and talk about how PaLaDIn plans to support patient centered decision making, through the development of PaLaDIn Interactium data platform.?

A wonderful Annual Curators meeting of the TREAT-NMD Global Registry Network in Milan with over 70 global attendees. Great discussions on impact, data quality, synthetic data use and PaLaDIn.

Marco Roos from PaLaDIn beneficiary Leiden University Medical Center will be presenting today at a Dutch National event for Rare Disease day, to an audience of patient representatives, care providers and researchers. Marco will be presenting about care pathways and patient journeys. Marco's presentation will focus on the importance of combining both the patient and data perspective and will highlight how we will adopt this approach in PaLaDIn. Find out more about the event here: https://lnkd.in/fSmAN2k

Today is Rare Disease Day! We hope you are inspired to share your story along with the FSHD Community and those with rare diseases all around the world. https://lnkd.in/gzAbzJUN ? Head to the link in our bio to visit our Rare Disease Day page. Download the images to share on your social media. https://lnkd.in/gB6EzbG ? Use the hashtags #RareDiseaseDay #FSHD and #CureFSHD in your stories and posts, and don't forget to tag us! ? Take this opportunity to share your FSHD experience with the world ?? #RareDiseaseDay2025 #CureFSHD #FacesOfFSHD #FSHD #RareDiseaseDay #RareWarrior #MuscularDystrophy #DisabilityAwareness #RareDisease

As part of Rare Disease Day events, PaLaDIn beneficiary Newcastle University will be hosting Genetics Matters. This event, taking place on Sunday, aims to connect with patients and the local community about the research into rare diseases that is happening at Newcastle University. Find out more here: https://lnkd.in/egJVvmvP