World Duchenne Organization

⏰ 𝗗𝘂𝗰𝗵𝗲𝗻𝗻𝗲 𝗣𝗮𝘁𝗶𝗲𝗻𝘁 𝗔𝗰𝗮𝗱𝗲𝗺𝘆 𝟮𝟬𝟮𝟰 – 𝗟𝗮𝘀𝘁 𝗰𝗵𝗮𝗻𝗰𝗲 𝘁𝗼 𝗮𝗽𝗽𝗹𝘆 Applications are almost closing for the Duchenne Patient Academy 2024, organized by the Duchenne Data Foundation and promoted by the World Duchenne Organization. Join us online on December 6 & 7 to gain essential advocacy skills, connect with experts, and learn about key topics like drug development, patient advocacy, and standards of care. This event is supported by PTC Therapeutics, Inc., Sarepta Therapeutics, Roche, BioMarin Pharmaceutical Inc., Italfarmaco, Santhera Pharmaceuticals, Solid Biosciences and Wave Life Sciences. 💻 Apply now and help shape the future of DMD/BMD advocacy! https://lnkd.in/eKUq3Mtb

🗣️ Duchenne Patient Academy 2024: last chance to apply! Applications are closing soon for 𝗗𝘂𝗰𝗵𝗲𝗻𝗻𝗲 𝗣𝗮𝘁𝗶𝗲𝗻𝘁 𝗔𝗰𝗮𝗱𝗲𝗺𝘆 𝟮𝟬𝟮𝟰. This is a unique opportunity for the next generation patient advocates to gain training on essential advocacy skills and knowledge for the Duchenne and Becker MD community. Organized by the World Duchenne Organization and the Duchenne Data Foundation, this two-day virtual event will empower new advocates to make a lasting impact in their communities. Through expert-led sessions, attendees will participate in engaging exercises, hear from experts in the field, and connect with peers across the globe. 📅 When? Friday, December 6, 16:00 – 19:00 CET  Saturday, December 7, 14:00 – 20:00 CET 𝗧𝗼𝗽𝗶𝗰𝘀 𝗶𝗻𝗰𝗹𝘂𝗱𝗲 → Community building → Updates on drug development → Proving optimal care → Research: Challenges in drug development → Role of POs in Standards of Care → Improving advocacy → Patient involvement in regulatory processes → Crisis communication → Best practices in fundraising This academy is a must-attend for people who are new to advocacy and looking forward to develop skills to make a difference in the DMD/BMD community. 📌 For more information and to apply, visit https://lnkd.in/eKUq3Mtb

That’s a wrap on a fantastic, two-day Kick-Off meeting for ERDERA, the new rare disease research alliance! With representatives from all 25 work streams present both in-person and online, conversations have been very productive, discussing progress since the official launch in September and setting up for success over the next seven years! Day ⁠1️⃣ commenced with a public Opening Ceremony, with speakers focusing on the consortium’s vision, mission, and goals. Nacer Boubenna, Scientific Director at the Ministère de l'Enseignement supérieur et de la Recherche, opened the plenary by highlighting the vital importance of both national plans and European cooperation in the field of rare diseases, both of which ERDERA will help to foster. Anne-Sophie Lapointe (PhD) reaffirmed this message, stating, “With coordinated action at every level - local, national, European and global, we can finally prevent people from suffering in silence.” The session closed with our CEO Virginie Bros-Facer, driving home ERDERA’s core vision - to bring hope to people living with a rare disease - and a deeper exploration of how the consortium's structure will bring the 25 work streams together to achieve this, lead by ERDERA's coordinator, Daria Julkowska. ✊ 🗣️ “ERDERA will present a new chapter in our collective journey, promising to accelerate research and translate scientific discovery into tangible benefits for rare disease patients.” - Virginie Bros-Facer Day 2️⃣ saw breakout thematic and work stream sessions. EURORDIS representatives focused on bringing the patient voice to these vital discussions to ensure that we are constantly working to address the unmet needs of people living with rare disease. Want to learn more about the ways in which we are involved in ERDERA? ➡️ https://lnkd.in/eBNgDci3 Our thanks go to the entire team involved in organising the event and everyone who attended and contributed to these important conversations! #RareDiseases #Europe #Global #research #alliance #innovation #ERDERA Roseline Favresse, Gemma Rodríguez Fabià, Rachel Butcher

Following a re-examination of available data, EMA’s human medicines committee (CHMP) has confirmed its previous recommendation to not renew the conditional marketing authorisation for Translarna (ataluren). This last round of assessment concluded that the effectiveness of Translarna has not been confirmed. Read the full article by EMA here: https://lnkd.in/evn2dmP2

Misha, the little boy who discovers life with big eyes and dreams. Find out more about his story part of the WDAD documentary and watch his full interview. Many thanks to UILDM Direzione Nazionale , Fondazione Policlinico Universitario Agostino Gemelli IRCCS and Parent Project aps

The #OpenAcademy2025 is a unique opportunity to deepen your expertise in therapeutic development for rare diseases and engage with key stakeholders. Send in your application by October 26!

Patient experts, join us for #OpenAcademy2025! Apply today to enhance your understanding of rare disease research.

Elizabeth Vroom shares why the World Duchenne Organization is part of the BIND Project. “𝘖𝘶𝘳 𝘨𝘰𝘢𝘭 𝘪𝘴 𝘵𝘰 𝘣𝘦𝘵𝘵𝘦𝘳 𝘶𝘯𝘥𝘦𝘳𝘴𝘵𝘢𝘯𝘥 𝘩𝘰𝘸 𝘮𝘶𝘵𝘢𝘵𝘪𝘰𝘯𝘴 𝘪𝘯 𝘵𝘩𝘦 𝘥𝘺𝘴𝘵𝘳𝘰𝘱𝘩𝘪𝘯 𝘨𝘦𝘯𝘦 𝘤𝘢𝘯 𝘤𝘢𝘶𝘴𝘦 𝘭𝘦𝘢𝘳𝘯𝘪𝘯𝘨 𝘢𝘯𝘥 𝘣𝘦𝘩𝘢𝘷𝘪𝘰𝘶𝘳𝘢𝘭 𝘪𝘴𝘴𝘶𝘦𝘴, 𝘴𝘰 𝘸𝘦 𝘤𝘢𝘯 𝘪𝘮𝘱𝘳𝘰𝘷𝘦 𝘥𝘪𝘢𝘨𝘯𝘰𝘴𝘪𝘴, 𝘤𝘢𝘳𝘦, 𝘢𝘯𝘥 𝘧𝘶𝘵𝘶𝘳𝘦 𝘵𝘳𝘦𝘢𝘵𝘮𝘦𝘯𝘵𝘴.“ At the BIND webinar tonight, she will share her experience and moderate the questions from participants. 👉 Don't miss this opportunity! https://lnkd.in/eCg4wU3y

⚽ Powerchair football to improve the quality of life. This story filmed in Brazil is part of the Documentary Raise your voice for Duchenne. 🤝 We thank Instituto Novo Ser, Casa Hunter - Associação Brasileira de Doenças Raras, Marinha do Brasil and Hospital Universitário Gaffrée e Guinle for the kind cooperation. Directed by NICOLETTA MADIA, produced by Arim Communication.

You are invited to join the BIND Patient Community webinar '𝗨𝗻𝗱𝗲𝗿𝘀𝘁𝗮𝗻𝗱𝗶𝗻𝗴 𝘁𝗵𝗲 𝗿𝗼𝗹𝗲 𝗼𝗳 𝗱𝘆𝘀𝘁𝗿𝗼𝗽𝗵𝗶𝗻 𝘁𝗵𝗲 𝗯𝗿𝗮𝗶𝗻' 🗓 Monday, September 23 🕕 18:00 CEST 📍 Online via Zoom Prof Francesco Muntoni, who is one of the speakers, says: “𝘛𝘩𝘪𝘴 𝘸𝘦𝘣𝘪𝘯𝘢𝘳 𝘱𝘳𝘰𝘷𝘪𝘥𝘦𝘴 𝘢𝘵𝘵𝘦𝘯𝘥𝘦𝘦𝘴 𝘸𝘪𝘵𝘩 𝘢 𝘰𝘱𝘱𝘰𝘳𝘵𝘶𝘯𝘪𝘵𝘺 𝘵𝘰 𝘣𝘦 𝘶𝘱𝘥𝘢𝘵𝘦𝘥 𝘢𝘣𝘰𝘶𝘵 𝘰𝘯𝘨𝘰𝘪𝘯𝘨 𝘳𝘦𝘴𝘦𝘢𝘳𝘤𝘩 𝘰𝘯 𝘣𝘳𝘢𝘪𝘯 𝘤𝘰𝘮𝘰𝘳𝘣𝘪𝘥𝘪𝘵𝘪𝘦𝘴 𝘪𝘯 𝘋𝘶𝘤𝘩𝘦𝘯𝘯𝘦 𝘢𝘯𝘥 𝘉𝘦𝘤𝘬𝘦𝘳 𝘮𝘶𝘴𝘤𝘶𝘭𝘢𝘳 𝘥𝘺𝘴𝘵𝘳𝘰𝘱𝘩𝘺.“ 👉 Please register your interest here: https://lnkd.in/eCg4wU3y