Ever heard of #HSPB8 #Myopathy (MFM13)? Probably not. Check out our poster from the @OrphanConf and if you're feeling bold, pass it on.

Our COO, Karen Tubridy, participated in a panel at @OrphanConf on women redefining healthcare, discussing topics including overcoming barriers in the healthcare industry and creating impactful solutions in rare diseases. #WorldOrphanUSA

RT @X4Pharma: Our VP of Sales & Marketing recently hosted a fireside chat with Medical Publisher @CheckRare at #WorldOrphanUSA, a forum foc…

Last week, we wrapped up #WorldOrphanUSA 2025 – our largest and most impactful edition to date! 🌟 🌟 Our team wants to give a huge THANK YOU to our sponsors, exhibitors, speakers, and attendees for making this year’s event unforgettable. Your passion, insights, and commitment to the rare disease community have fueled every conversation, every connection, and every breakthrough moment. Together, we’re not just talking about change – we're making it happen. We can't wait to see the impact you’ll carry forward from here! ❤️‍🩹 𝘋𝘪𝘥 𝘺𝘰𝘶 𝘩𝘦𝘢𝘳? We're returning to Boston for our 2026 edition... 👇 𝐒𝐀𝐕𝐄 𝐓𝐇𝐄 𝐃𝐀𝐓𝐄 🗓️ May 19-21, 2026 📍 Boston Convention & Exhibition Center Stay tuned for more on next year's event – we can't wait to see you back in Boston for another amazing year!

Thanks to Terrapin for another successful World Orphan Drug Congress USA! We look forward to our continued partnership with this outstanding team. #WorldOrphanUSA #Terrapin #Checkrare @OrphanConf

#WorldOrphanUSA: “The delay of MLD screening equals death for these babies. They cannot wait.” - Christina Hartman, senior director of government affairs at @orchard_tx, whose #GeneTherapy treats metachromatic leukodystrophy (MLD). #RDAatWODC @OrphanConf

I am exhausted but empowered after the last few days at the @OrphanConf in Boston, especially after today's panel with fellow rare disease advocates @Jeanc9orf72, Allison Peck, and Heidi Edwards, as well as the woman who brought us all together...

Day 2 at #WODCUSA is all about the power of partnerships—patient advocacy, storytelling, and building community. It’s inspiring to see how these voices shape progress in rare disease care. What insights have stood out most to you today? @OrphanConf #WorldOrphanUSA #RareDisease

We out here repping @APSType1 and Rare Disease International at the @orphanconf #worldorphandrugcongress

We're at @OrphanConf this week to support our clients and participate in important conversations for rare diseases. We're proud to be a part of the progress.

#WorldOrphanUSA: Jennifer McNary, CEO of Canary Advisors, speaking April 23, 2025, at @OrphanConf. @LLuxner #RDAatWODC

That’s a wrap on the 2025 @OrphanConf! SMPA was honored to contribute alongside researchers, clinicians and advocates dedicated to advancing rare disease research. #WorldOrphanUSA #RareDisease

#OrphanDrugUSA @OrphanConf #RDAatWODC

#WorldOrphanUSA: "Everyone is not their diagnosis…& we clinicians can’t assume that because we see the world & their disease a certain way, that’s the way they want to see it." - Dr. Faisal Riaz, Neuroimmunology & AATD specialist at Takeda Pharmaceutical." @OrphanConf

Good morning from Boston! Day 2 of #WorldOrphanUSA 2025 is underway 🌟 After an incredible first day packed with insight, energy, and collaboration, we’re diving into another full day of advancing the future of #OrphanDrug development and #raredisease care. We’re starting strong with today’s main stage keynote sessions, featuring thought leaders who are challenging norms, shaping policy, and pushing innovation forward – including ↴ [8:30AM] 𝘊𝘩𝘢𝘪𝘳 𝘱𝘦𝘳𝘴𝘰𝘯 𝘰𝘱𝘦𝘯𝘪𝘯𝘨 𝘳𝘦𝘮𝘢𝘳𝘬𝘴 | 𝐓𝐫𝐚𝐧𝐬𝐟𝐨𝐫𝐦𝐢𝐧𝐠 𝐭𝐡𝐞 𝐝𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭 𝐚𝐧𝐝 𝐫𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐲 𝐥𝐚𝐧𝐝𝐬𝐜𝐚𝐩𝐞 𝐭𝐨 𝐦𝐞𝐞𝐭 𝐭𝐡𝐞 𝐧𝐞𝐞𝐝𝐬 𝐨𝐟 𝐮𝐧𝐭𝐫𝐞𝐚𝐭𝐞𝐝 𝐫𝐚𝐫𝐞 𝐝𝐢𝐬𝐞𝐚𝐬𝐞𝐬 𝐢𝐧 𝐭𝐡𝐞 𝟐𝟏𝐬𝐭 𝐜𝐞𝐧𝐭𝐮𝐫𝐲 ✮ CEO, @ultragenyx [8:50AM] 𝘒𝘦𝘺𝘯𝘰𝘵𝘦 𝘗𝘢𝘯𝘦𝘭 | 𝐏𝐫𝐢𝐨𝐫𝐢𝐭𝐢𝐞𝐬, 𝐩𝐨𝐥𝐢𝐜𝐢𝐞𝐬 𝐚𝐧𝐝 𝐢𝐦𝐩𝐚𝐜𝐭 𝐨𝐧 𝐩𝐚𝐭𝐢𝐞𝐧𝐭𝐬 𝐥𝐢𝐯𝐢𝐧𝐠 𝐰𝐢𝐭𝐡 𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 ✮ Moderator: Partner, Health Policy Strategies ✮ Chief Global Value and Access Officer, @JNJInnovMed ✮ President, Co-Founder, @CureFA_org ✮ Director of Policy and Regulatory Affairs, @RareDiseases (NORD) 𝘚𝘱𝘰𝘯𝘴𝘰𝘳𝘦𝘥 𝘣𝘺 @JNJNews [9:30AM] 𝘒𝘦𝘺𝘯𝘰𝘵𝘦 𝘗𝘢𝘯𝘦𝘭 | 𝐁𝐮𝐢𝐥𝐝𝐢𝐧𝐠 𝐭𝐡𝐞 𝐟𝐫𝐚𝐦𝐞𝐰𝐨𝐫𝐤: 𝐀 𝐬𝐮𝐬𝐭𝐚𝐢𝐧𝐚𝐛𝐥𝐞, 𝐞𝐟𝐟𝐞𝐜𝐭𝐢𝐯𝐞, 𝐚𝐧𝐝 𝐞𝐪𝐮𝐢𝐭𝐚𝐛𝐥𝐞 𝐬𝐨𝐥𝐮𝐭𝐢𝐨𝐧 𝐟𝐨𝐫 𝐎𝐫𝐩𝐡𝐚𝐧 𝐃𝐫𝐮𝐠𝐬 ✮ Moderator: EVP, Chief Medical Officer, @ultragenyx ✮ President, Head of R&D and COO, Rocket Phama ✮ CEO, @alliancerm [12:50PM] 𝘒𝘦𝘺𝘯𝘰𝘵𝘦 𝘗𝘢𝘯𝘦𝘭 & 𝘚𝘤𝘳𝘦𝘦𝘯𝘪𝘯𝘨 | 𝐓𝐡𝐞 𝐍𝐞𝐱𝐭 𝐅𝐫𝐨𝐧𝐭𝐢𝐞𝐫: 𝐓𝐡𝐞 𝐚𝐫𝐭 𝐨𝐟 𝐢𝐦𝐩𝐚𝐜𝐭𝐟𝐮𝐥 𝐬𝐭𝐨𝐫𝐲𝐭𝐞𝐥𝐥𝐢𝐧𝐠 𝐢𝐧 𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 ✮ Moderator: Global VP of Programme Partnerships, @BBCStoryWorks ✮Chief Patient Advocate & Head, Patient Advocacy Center of Excellence, @IAmBiotech (BIO) ✮ Global Head of Clinical Development and General Manager, Europe & USA, JCR Pharmaceuticals ✮ Director Of Development, Programme Partnerships, @BBC There's still time to join us today in Boston! 👉 Register below & get ready for bold ideas, honest dialogue, and the kind of connections that fuel long-term impact. terrapinn.com/bookwodc_tw

Rare Disease Advisor is in Boston, MA for #WorldOrphanUSA. Follow along for live updates, expert interviews, and our first-ever Rare Advocacy session! @OrphanConf #RDAatWODC

🥁 𝘋𝘳𝘶𝘮 𝘳𝘰𝘭𝘭 𝘱𝘭𝘦𝘢𝘴𝘦... Day 1 of the World Orphan Drug Congress USA 2025 is finally here! The #raredisease community is taking over Boston this week, and we’re kicking things off with a powerhouse lineup of visionary keynote sessions setting the tone for the days ahead. This morning, we’re diving into the most critical conversations shaping the future of the rare disease space ↴ [8:25am] 𝘖𝘱𝘦𝘯𝘪𝘯𝘨 𝘳𝘦𝘮𝘢𝘳𝘬𝘴 | 𝐓𝐨𝐝𝐚𝐲’𝐬 𝐩𝐨𝐥𝐢𝐜𝐢𝐞𝐬 𝐰𝐢𝐥𝐥 𝐦𝐚𝐤𝐞 𝐨𝐫 𝐛𝐫𝐞𝐚𝐤 𝐭𝐨𝐦𝐨𝐫𝐫𝐨𝐰’𝐬 𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞 𝐩𝐢𝐩𝐞𝐥𝐢𝐧𝐞𝐬 ✺ Chief Patient Advocate & Head, Patient Advocacy Center of Excellence, @IAmBiotech (BIO) [8:45am] 𝘒𝘦𝘺𝘯𝘰𝘵𝘦 𝘗𝘢𝘯𝘦𝘭 | 𝐄𝐚𝐫𝐥𝐲 𝐢𝐧𝐭𝐞𝐫𝐯𝐞𝐧𝐭𝐢𝐨𝐧: 𝐓𝐡𝐞 𝐜𝐫𝐢𝐭𝐢𝐜𝐚𝐥 𝐩𝐚𝐭𝐡 𝐢𝐧 𝐫𝐚𝐫𝐞 𝐝𝐢𝐬𝐞𝐚𝐬𝐞𝐬 ✺ Moderator: Health & Medical Reporter and Writer, @NBCNews ✺ General Manager, US Rare Disease, @Sanofi ✺ CEO, @RareDiseases (NORD) [9:05am] 𝘒𝘦𝘺𝘯𝘰𝘵𝘦 𝘗𝘢𝘯𝘦𝘭 | 𝐑𝐞𝐩𝐨𝐬𝐢𝐭𝐢𝐨𝐧𝐢𝐧𝐠 𝐝𝐮𝐠𝐬 𝐟𝐨𝐫 𝐑𝐚𝐫𝐞 𝐃𝐢𝐬𝐞𝐚𝐬𝐞𝐬: 𝐔𝐧𝐥𝐞𝐚𝐬𝐡𝐢𝐧𝐠 𝐭𝐡𝐞 𝐩𝐨𝐭𝐞𝐧𝐭𝐢𝐚𝐥 𝐨𝐟 𝐬𝐡𝐞𝐥𝐯𝐞𝐝 𝐚𝐬𝐬𝐞𝐭𝐬 𝐢𝐧 𝐩𝐡𝐚𝐫𝐦𝐚 𝐚𝐧𝐝 𝐛𝐢𝐨𝐭𝐞𝐜𝐡 ✺ Moderator: CEO, @ChildrensTumor ✺ Former Chief Patient Officer & Executive Vice President, @pfizer ✺ Charles E. and Susan T. Harris Professor, @MITSloan ✺ Visiting Fellow, @DukeMargolis ✺ Associate Professor, Markets, Public Policy, and Law, @BU_Tweets [9:45am] 𝘒𝘦𝘺𝘯𝘰𝘵𝘦 𝘗𝘢𝘯𝘦𝘭 | 𝐕𝐢𝐬𝐢𝐨𝐧 𝟐𝟎𝟑𝟎: 𝐂𝐡𝐚𝐫𝐭𝐢𝐧𝐠 𝐚 𝐛𝐫𝐢𝐠𝐡𝐭 𝐟𝐮𝐭𝐮𝐫𝐞 𝐟𝐨𝐫 𝐫𝐚𝐫𝐞 𝐦𝐞𝐝𝐢𝐜𝐢𝐧𝐞 ✺ Moderator: Executive Director, Global Head of Rare Disease, CoE, @Parexel ✺ Director of Strategic Coalitions, Rare Disease Innovation Hub, @US_FDA ✺ Co-Founder, @CnryAdvisors ✺ SVP, Patient Affairs, @Sarepta ✺ CEO, @reaganudall There's still time to join the world's most important event for advancing Orphan Drug development and rare disease patient care. Act now 🎟️ Get your ticket today [𝘭𝘪𝘮𝘪𝘵𝘦𝘥 𝘢𝘷𝘢𝘪𝘭𝘢𝘣𝘪𝘭𝘪𝘵𝘺] and join us in Boston today & tomorrow ➜ terrapinn.com/bookwodc_tw *𝘗𝘢𝘵𝘪𝘦𝘯𝘵 𝘢𝘥𝘷𝘰𝘤𝘢𝘵𝘦𝘴 𝘢𝘯𝘥 𝘩𝘰𝘴𝘱𝘪𝘵𝘢𝘭 𝘱𝘳𝘢𝘤𝘵𝘪𝘵𝘪𝘰𝘯𝘦𝘳𝘴 𝘢𝘵𝘵𝘦𝘯𝘥 𝘧𝘰𝘳 𝘍𝘙𝘌𝘌 #WorldOrphanUSA 🗓️ April 22-24 📍 Boston Convention & Exhibition Center

📣 Meet the Day 1 Chairs of #WorldOrphanUSA 2025! Tomorrow, these industry leaders are bringing their expertise, energy, and vision to the forefront – guiding the conversations that matter most in rare disease and orphan drug innovation. ✦ 𝐈𝐧𝐧𝐨𝐯𝐚𝐭𝐢𝐨𝐧 & 𝐏𝐚𝐫𝐭𝐧𝐞𝐫𝐬𝐡𝐢𝐩𝐬 → VP, Head of US Medical Affairs, ITF Therapeutics LLC ✦ 𝐂𝐥𝐢𝐧𝐢𝐜𝐚𝐥 𝐃𝐞𝐯𝐞𝐥𝐨𝐩𝐦𝐞𝐧𝐭 & 𝐑𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐲 → Senior Director, Global Medical Affairs, @Alnylam ✦ 𝐑𝐚𝐫𝐞 𝐀𝐝𝐯𝐨𝐜𝐚𝐜𝐲 → Executive Director, @SistersHopeALSP ✦ 𝐀𝐝𝐯𝐚𝐧𝐜𝐞𝐝 𝐓𝐡𝐞𝐫𝐚𝐩𝐢𝐞𝐬 → VP, Medical Affairs, Rare Diseases and Pediatrics, @premierresearch ✦ 𝐂𝐨𝐦𝐦𝐞𝐫𝐜𝐢𝐚𝐥 → Managing Director, @HavasMediaGroup (Havas Life Rare) ✦ 𝐄𝐚𝐫𝐥𝐲 𝐀𝐜𝐜𝐞𝐬𝐬 𝐏𝐫𝐨𝐠𝐫𝐚𝐦𝐬 → Director, Expanded Access Program, @argenxglobal ✦ 𝐀𝐈 & 𝐃𝐢𝐠𝐢𝐭𝐚𝐥 𝐇𝐞𝐚𝐥𝐭𝐡 → Rare Disease Patient Digital Technology Advisor, Bartlett Rare Disease Advocacy Join these industry leaders this week in Boston as they drive the dialogue shaping the future of rare and orphan drug development – from clinical breakthroughs to commercialization and policy. There's still time to register! 🎟️𝘚𝘦𝘤𝘶𝘳𝘦 𝘺𝘰𝘶𝘳 𝘴𝘱𝘰𝘵 𝘣𝘦𝘭𝘰𝘸 𝘣𝘦𝘧𝘰𝘳𝘦 𝘵𝘪𝘤𝘬𝘦𝘵𝘴 𝘴𝘦𝘭𝘭 𝘰𝘶𝘵: 🔬 𝘗𝘩𝘢𝘳𝘮𝘢 𝘢𝘯𝘥 𝘣𝘪𝘰𝘵𝘦𝘤𝘩𝘴 – register now and get 𝐬𝐩𝐞𝐜𝐢𝐚𝐥 𝐭𝐢𝐜𝐤𝐞𝐭 𝐩𝐫𝐢𝐜𝐢𝐧𝐠: terrapinn.com/bookwodc_tw 🏥 𝘗𝘢𝘵𝘪𝘦𝘯𝘵 𝘢𝘥𝘷𝘰𝘤𝘢𝘵𝘦𝘴 𝘢𝘯𝘥 𝘩𝘰𝘴𝘱𝘪𝘵𝘢𝘭 𝘱𝘳𝘢𝘤𝘵𝘪𝘵𝘪𝘰𝘯𝘦𝘳𝘴 – secure one of the last remaining free VIP passes: terrapinn.com/bookwodc_tw 👥 Save BIG with our discounted group rates and bring your team: terrapinn.com/freewodc_tw Pre-Conference Day: April 22 Event Day 1: April 23 Event Day 2: April 24 Venue: Boston Convention & Exhibition Center

Don't miss any highlights from #WorldOrphanUSA 2025! Follow our LinkedIn page below to catch LIVE coverage the next 3 days in Boston 📸 linkedin.com/company/world-o… 🗓️ April 22-24 📍 Boston Convention & Exhibition Cetner

𝘛𝘩𝘦 𝘸𝘢𝘪𝘵 𝘪𝘴 𝘧𝘪𝘯𝘢𝘭𝘭𝘺 𝘰𝘷𝘦𝘳... #WorldOrphanUSA 2025 kicks off today with our Pre-Conference Day! 🎊 With a jam packed schedule filled with interactive workshops, presentations, and fireside chats , you won't want to miss this. Take a glimpse into what's in store ↴ [1pm] Workshop | 𝘉𝘳𝘦𝘢𝘬𝘪𝘯𝘨 𝘪𝘯𝘵𝘰 𝘌𝘶𝘳𝘰𝘱𝘦: 𝘗𝘢𝘳𝘵𝘯𝘦𝘳𝘴𝘩𝘪𝘱𝘴 𝘵𝘩𝘢𝘵 𝘱𝘳𝘰𝘱𝘦𝘭 𝘜.𝘚. 𝘣𝘪𝘰𝘵𝘦𝘤𝘩𝘴 𝘵𝘰 𝘰𝘳𝘱𝘩𝘢𝘯 𝘥𝘳𝘶𝘨 𝘴𝘶𝘤𝘤𝘦𝘴𝘴 ✴︎ VP Access & Reimbursement, @Sciensus_Group ✴︎ Director of Rare Clinical Services, @Sciensus_Group ✴︎ VP, Medical Affairs, @Sciensus_Group ✴︎ VP, Rare & Orphan Strategy, @Sciensus_Group ✴︎ SVP Commercial Strategy & Operations, @sentynl ✴︎ Volunteer, Rare Disease Patient *𝘚𝘱𝘰𝘯𝘴𝘰𝘳𝘦𝘥 𝘣𝘺 𝘚𝘤𝘪𝘦𝘯𝘴𝘶𝘴 [2pm] Workshop | 𝘛𝘩𝘦 𝘤𝘰𝘮𝘮𝘦𝘳𝘤𝘪𝘢𝘭 𝘧𝘰𝘳𝘦𝘤𝘢𝘴𝘵 𝘢𝘴 𝘢 𝘴𝘵𝘳𝘢𝘵𝘦𝘨𝘺-𝘥𝘳𝘪𝘷𝘦𝘳 ✴︎ Director, @BluePrintOrphan ✴︎ President, @BluePrintOrphan *𝘚𝘱𝘰𝘯𝘴𝘰𝘳𝘦𝘥 𝘣𝘺 𝘉𝘭𝘶𝘦𝘗𝘳𝘪𝘯𝘵𝘖𝘳𝘱𝘩𝘢𝘯 [2pm] Presentation | Access & Pricing | 𝘕𝘢𝘷𝘪𝘨𝘢𝘵𝘪𝘯𝘨 𝘵𝘩𝘦 𝘤𝘰𝘮𝘱𝘭𝘦𝘹𝘪𝘵𝘪𝘦𝘴 𝘰𝘧 𝘢𝘤𝘤𝘦𝘴𝘴𝘪𝘯𝘨 𝘦𝘮𝘦𝘳𝘨𝘪𝘯𝘨 𝘵𝘩𝘦𝘳𝘢𝘱𝘪𝘦𝘴 𝘢𝘤𝘳𝘰𝘴𝘴 𝘴𝘵𝘢𝘵𝘦 𝘣𝘰𝘳𝘥𝘦𝘳𝘴 ✴︎ RN, Vice President - Emerging Discoveries, @BostonChildrens [3pm] Presentation | Patient Data | 𝘐𝘭𝘭𝘶𝘮𝘪𝘯𝘢𝘵𝘪𝘯𝘨 𝘶𝘭𝘵𝘳𝘢-𝘳𝘢𝘳𝘦 𝘥𝘪𝘴𝘦𝘢𝘴𝘦 𝘫𝘰𝘶𝘳𝘯𝘦𝘺𝘴: 𝘏𝘢𝘳𝘯𝘦𝘴𝘴𝘪𝘯𝘨 𝘳𝘦𝘢𝘭-𝘸𝘰𝘳𝘭𝘥 𝘥𝘢𝘵𝘢 𝘢𝘯𝘥 𝘢𝘥𝘷𝘢𝘯𝘤𝘦𝘥 𝘢𝘯𝘢𝘭𝘺𝘵𝘪𝘤𝘴 𝘵𝘰 𝘪𝘥𝘦𝘯𝘵𝘪𝘧𝘺 𝘵𝘩𝘦 𝘶𝘯𝘥𝘪𝘢𝘨𝘯𝘰𝘴𝘦𝘥 ✴︎ President, @Egetis_EGTX [3:20pm] Presentation | Next Gen Therapies | 𝘔𝘰𝘥𝘦𝘭𝘪𝘯𝘨 𝘢𝘯𝘥 𝘚𝘪𝘮𝘶𝘭𝘢𝘵𝘪𝘰𝘯 (𝘔&𝘚) 𝘪𝘯 𝘯𝘦𝘹𝘵-𝘨𝘦𝘯 𝘵𝘩𝘦𝘳𝘢𝘱𝘪𝘦𝘴: 𝘈𝘤𝘤𝘦𝘭𝘦𝘳𝘢𝘵𝘪𝘯𝘨 𝘪𝘯𝘯𝘰𝘷𝘢𝘵𝘪𝘰𝘯 𝘵𝘩𝘳𝘰𝘶𝘨𝘩 𝘪𝘯𝘴𝘪𝘨𝘩𝘵 ✴︎ Chief Clinical Development Officer, @Polaryx777 Interested in being a part of the world's most important Orphan Drug and rare disease event? Good news – 𝐭𝐡𝐞𝐫𝐞'𝐬 𝐬𝐭𝐢𝐥𝐥 𝐭𝐢𝐦𝐞 𝐭𝐨 𝐫𝐞𝐠𝐢𝐬𝐭𝐞𝐫! 🎟️ Get your ticket below ↴ ⭐️ 𝐒𝐞𝐜𝐮𝐫𝐞 𝐨𝐧𝐞 𝐨𝐟 𝐨𝐮𝐫 𝐥𝐚𝐬𝐭 𝐚𝐯𝐚𝐢𝐥𝐚𝐛𝐥𝐞 𝐭𝐢𝐜𝐤𝐞𝐭𝐬: terrapinn.com/bookwodc_tw ⭐️ Patient advocates & hospital practitioners, we've 𝐚𝐝𝐝𝐞𝐝 𝟓𝟎 𝐦𝐨𝐫𝐞 𝐜𝐨𝐦𝐩𝐥𝐢𝐦𝐞𝐧𝐭𝐚𝐫𝐲 𝐕𝐈𝐏 𝐩𝐚𝐬𝐬𝐞𝐬 for the first individuals that apply. Get yours now: terrapinn.com/freewodc_tw Pre-Conference Day | April 22 Main Event Days | April 23 & 24 Venue | Boston Convention & Exhibition Center