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World Orphan Drug Congress USA

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Get regular updates on #WorldOrphanUSA!

June 9-11, 2026 | Boston Convention & Exhibition Center
Worldwide terrapinn.com/orphandrugsusa Joined July 2010
852 Following 4.6K Followers 5.7K Posts

Latest posts 5

World Orphan Drug Congress USA Verified account @OrphanConf
Last week, we wrapped up #WorldOrphanUSA 2025 โ€“ our largest and most impactful edition to date! ๐ŸŒŸ ๐ŸŒŸ

Our team wants to give a huge THANK YOU to our sponsors, exhibitors, speakers, and attendees for making this yearโ€™s event unforgettable. Your passion, insights, and commitment to the rare disease community have fueled every conversation, every connection, and every breakthrough moment.

Together, weโ€™re not just talking about change โ€“ we're making it happen. We can't wait to see the impact youโ€™ll carry forward from here! โค๏ธโ€๐Ÿฉน

๐˜‹๐˜ช๐˜ฅ ๐˜บ๐˜ฐ๐˜ถ ๐˜ฉ๐˜ฆ๐˜ข๐˜ณ? We're returning to Boston for our 2026 edition...
๐Ÿ‘‡
๐’๐€๐•๐„ ๐“๐‡๐„ ๐ƒ๐€๐“๐„
๐Ÿ—“๏ธ May 19-21, 2026
๐Ÿ“ Boston Convention & Exhibition Center

Stay tuned for more on next year's event โ€“ we can't wait to see you back in Boston for another amazing year!
World Orphan Drug Congress USA Verified account @OrphanConf
Good morning from Boston! Day 2 of #WorldOrphanUSA 2025 is underway ๐ŸŒŸ
After an incredible first day packed with insight, energy, and collaboration, weโ€™re diving into another full day of advancing the future of #OrphanDrug development and #raredisease care.

Weโ€™re starting strong with todayโ€™s main stage keynote sessions, featuring thought leaders who are challenging norms, shaping policy, and pushing innovation forward โ€“ including โ†ด

[8:30AM] ๐˜Š๐˜ฉ๐˜ข๐˜ช๐˜ณ ๐˜ฑ๐˜ฆ๐˜ณ๐˜ด๐˜ฐ๐˜ฏ ๐˜ฐ๐˜ฑ๐˜ฆ๐˜ฏ๐˜ช๐˜ฏ๐˜จ ๐˜ณ๐˜ฆ๐˜ฎ๐˜ข๐˜ณ๐˜ฌ๐˜ด | ๐“๐ซ๐š๐ง๐ฌ๐Ÿ๐จ๐ซ๐ฆ๐ข๐ง๐  ๐ญ๐ก๐ž ๐๐ž๐ฏ๐ž๐ฅ๐จ๐ฉ๐ฆ๐ž๐ง๐ญ ๐š๐ง๐ ๐ซ๐ž๐ ๐ฎ๐ฅ๐š๐ญ๐จ๐ซ๐ฒ ๐ฅ๐š๐ง๐๐ฌ๐œ๐š๐ฉ๐ž ๐ญ๐จ ๐ฆ๐ž๐ž๐ญ ๐ญ๐ก๐ž ๐ง๐ž๐ž๐๐ฌ ๐จ๐Ÿ ๐ฎ๐ง๐ญ๐ซ๐ž๐š๐ญ๐ž๐ ๐ซ๐š๐ซ๐ž ๐๐ข๐ฌ๐ž๐š๐ฌ๐ž๐ฌ ๐ข๐ง ๐ญ๐ก๐ž ๐Ÿ๐Ÿ๐ฌ๐ญ ๐œ๐ž๐ง๐ญ๐ฎ๐ซ๐ฒ
โœฎ CEO, @ultragenyx

[8:50AM] ๐˜’๐˜ฆ๐˜บ๐˜ฏ๐˜ฐ๐˜ต๐˜ฆ ๐˜—๐˜ข๐˜ฏ๐˜ฆ๐˜ญ | ๐๐ซ๐ข๐จ๐ซ๐ข๐ญ๐ข๐ž๐ฌ, ๐ฉ๐จ๐ฅ๐ข๐œ๐ข๐ž๐ฌ ๐š๐ง๐ ๐ข๐ฆ๐ฉ๐š๐œ๐ญ ๐จ๐ง ๐ฉ๐š๐ญ๐ข๐ž๐ง๐ญ๐ฌ ๐ฅ๐ข๐ฏ๐ข๐ง๐  ๐ฐ๐ข๐ญ๐ก ๐‘๐š๐ซ๐ž ๐ƒ๐ข๐ฌ๐ž๐š๐ฌ๐ž
โœฎ Moderator: Partner, Health Policy Strategies
โœฎ Chief Global Value and Access Officer, @JNJInnovMed
โœฎ President, Co-Founder, @CureFA_org
โœฎ Director of Policy and Regulatory Affairs, @RareDiseases (NORD)
๐˜š๐˜ฑ๐˜ฐ๐˜ฏ๐˜ด๐˜ฐ๐˜ณ๐˜ฆ๐˜ฅ ๐˜ฃ๐˜บ @JNJNews

[9:30AM] ๐˜’๐˜ฆ๐˜บ๐˜ฏ๐˜ฐ๐˜ต๐˜ฆ ๐˜—๐˜ข๐˜ฏ๐˜ฆ๐˜ญ | ๐๐ฎ๐ข๐ฅ๐๐ข๐ง๐  ๐ญ๐ก๐ž ๐Ÿ๐ซ๐š๐ฆ๐ž๐ฐ๐จ๐ซ๐ค: ๐€ ๐ฌ๐ฎ๐ฌ๐ญ๐š๐ข๐ง๐š๐›๐ฅ๐ž, ๐ž๐Ÿ๐Ÿ๐ž๐œ๐ญ๐ข๐ฏ๐ž, ๐š๐ง๐ ๐ž๐ช๐ฎ๐ข๐ญ๐š๐›๐ฅ๐ž ๐ฌ๐จ๐ฅ๐ฎ๐ญ๐ข๐จ๐ง ๐Ÿ๐จ๐ซ ๐Ž๐ซ๐ฉ๐ก๐š๐ง ๐ƒ๐ซ๐ฎ๐ ๐ฌ
โœฎ Moderator: EVP, Chief Medical Officer, @ultragenyx
โœฎ President, Head of R&D and COO, Rocket Phama
โœฎ CEO, @alliancerm

[12:50PM] ๐˜’๐˜ฆ๐˜บ๐˜ฏ๐˜ฐ๐˜ต๐˜ฆ ๐˜—๐˜ข๐˜ฏ๐˜ฆ๐˜ญ & ๐˜š๐˜ค๐˜ณ๐˜ฆ๐˜ฆ๐˜ฏ๐˜ช๐˜ฏ๐˜จ | ๐“๐ก๐ž ๐๐ž๐ฑ๐ญ ๐…๐ซ๐จ๐ง๐ญ๐ข๐ž๐ซ: ๐“๐ก๐ž ๐š๐ซ๐ญ ๐จ๐Ÿ ๐ข๐ฆ๐ฉ๐š๐œ๐ญ๐Ÿ๐ฎ๐ฅ ๐ฌ๐ญ๐จ๐ซ๐ฒ๐ญ๐ž๐ฅ๐ฅ๐ข๐ง๐  ๐ข๐ง ๐‘๐š๐ซ๐ž ๐ƒ๐ข๐ฌ๐ž๐š๐ฌ๐ž
โœฎ Moderator: Global VP of Programme Partnerships, @BBCStoryWorks
โœฎChief Patient Advocate & Head, Patient Advocacy Center of Excellence, @IAmBiotech (BIO)
โœฎ Global Head of Clinical Development and General Manager, Europe & USA, JCR Pharmaceuticals
โœฎ Director Of Development, Programme Partnerships, @BBC

There's still time to join us today in Boston! ๐Ÿ‘‰ Register below & get ready for bold ideas, honest dialogue, and the kind of connections that fuel long-term impact.
terrapinn.com/bookwodc_tw
World Orphan Drug Congress USA Verified account @OrphanConf
๐Ÿฅ ๐˜‹๐˜ณ๐˜ถ๐˜ฎ ๐˜ณ๐˜ฐ๐˜ญ๐˜ญ ๐˜ฑ๐˜ญ๐˜ฆ๐˜ข๐˜ด๐˜ฆ... Day 1 of the World Orphan Drug Congress USA 2025 is finally here!

The #raredisease community is taking over Boston this week, and weโ€™re kicking things off with a powerhouse lineup of visionary keynote sessions setting the tone for the days ahead. This morning, weโ€™re diving into the most critical conversations shaping the future of the rare disease space โ†ด

[8:25am] ๐˜–๐˜ฑ๐˜ฆ๐˜ฏ๐˜ช๐˜ฏ๐˜จ ๐˜ณ๐˜ฆ๐˜ฎ๐˜ข๐˜ณ๐˜ฌ๐˜ด | ๐“๐จ๐๐š๐ฒโ€™๐ฌ ๐ฉ๐จ๐ฅ๐ข๐œ๐ข๐ž๐ฌ ๐ฐ๐ข๐ฅ๐ฅ ๐ฆ๐š๐ค๐ž ๐จ๐ซ ๐›๐ซ๐ž๐š๐ค ๐ญ๐จ๐ฆ๐จ๐ซ๐ซ๐จ๐ฐโ€™๐ฌ ๐‘๐š๐ซ๐ž ๐ƒ๐ข๐ฌ๐ž๐š๐ฌ๐ž ๐ฉ๐ข๐ฉ๐ž๐ฅ๐ข๐ง๐ž๐ฌ
โœบ Chief Patient Advocate & Head, Patient Advocacy Center of Excellence, @IAmBiotech (BIO)

[8:45am] ๐˜’๐˜ฆ๐˜บ๐˜ฏ๐˜ฐ๐˜ต๐˜ฆ ๐˜—๐˜ข๐˜ฏ๐˜ฆ๐˜ญ | ๐„๐š๐ซ๐ฅ๐ฒ ๐ข๐ง๐ญ๐ž๐ซ๐ฏ๐ž๐ง๐ญ๐ข๐จ๐ง: ๐“๐ก๐ž ๐œ๐ซ๐ข๐ญ๐ข๐œ๐š๐ฅ ๐ฉ๐š๐ญ๐ก ๐ข๐ง ๐ซ๐š๐ซ๐ž ๐๐ข๐ฌ๐ž๐š๐ฌ๐ž๐ฌ
โœบ Moderator: Health & Medical Reporter and Writer, @NBCNews
โœบ General Manager, US Rare Disease, @Sanofi
โœบ CEO, @RareDiseases (NORD)

[9:05am] ๐˜’๐˜ฆ๐˜บ๐˜ฏ๐˜ฐ๐˜ต๐˜ฆ ๐˜—๐˜ข๐˜ฏ๐˜ฆ๐˜ญ | ๐‘๐ž๐ฉ๐จ๐ฌ๐ข๐ญ๐ข๐จ๐ง๐ข๐ง๐  ๐๐ฎ๐ ๐ฌ ๐Ÿ๐จ๐ซ ๐‘๐š๐ซ๐ž ๐ƒ๐ข๐ฌ๐ž๐š๐ฌ๐ž๐ฌ: ๐”๐ง๐ฅ๐ž๐š๐ฌ๐ก๐ข๐ง๐  ๐ญ๐ก๐ž ๐ฉ๐จ๐ญ๐ž๐ง๐ญ๐ข๐š๐ฅ ๐จ๐Ÿ ๐ฌ๐ก๐ž๐ฅ๐ฏ๐ž๐ ๐š๐ฌ๐ฌ๐ž๐ญ๐ฌ ๐ข๐ง ๐ฉ๐ก๐š๐ซ๐ฆ๐š ๐š๐ง๐ ๐›๐ข๐จ๐ญ๐ž๐œ๐ก
โœบ Moderator: CEO, @ChildrensTumor
โœบ Former Chief Patient Officer & Executive Vice President, @pfizer
โœบ Charles E. and Susan T. Harris Professor, @MITSloan
โœบ Visiting Fellow, @DukeMargolis
โœบ Associate Professor, Markets, Public Policy, and Law, @BU_Tweets

[9:45am] ๐˜’๐˜ฆ๐˜บ๐˜ฏ๐˜ฐ๐˜ต๐˜ฆ ๐˜—๐˜ข๐˜ฏ๐˜ฆ๐˜ญ | ๐•๐ข๐ฌ๐ข๐จ๐ง ๐Ÿ๐ŸŽ๐Ÿ‘๐ŸŽ: ๐‚๐ก๐š๐ซ๐ญ๐ข๐ง๐  ๐š ๐›๐ซ๐ข๐ ๐ก๐ญ ๐Ÿ๐ฎ๐ญ๐ฎ๐ซ๐ž ๐Ÿ๐จ๐ซ ๐ซ๐š๐ซ๐ž ๐ฆ๐ž๐๐ข๐œ๐ข๐ง๐ž
โœบ Moderator: Executive Director, Global Head of Rare Disease, CoE, @Parexel
โœบ Director of Strategic Coalitions, Rare Disease Innovation Hub, @US_FDA
โœบ Co-Founder, @CnryAdvisors
โœบ SVP, Patient Affairs, @Sarepta
โœบ 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 ๐ŸŽŸ๏ธ Get your ticket today [๐˜ญ๐˜ช๐˜ฎ๐˜ช๐˜ต๐˜ฆ๐˜ฅ ๐˜ข๐˜ท๐˜ข๐˜ช๐˜ญ๐˜ข๐˜ฃ๐˜ช๐˜ญ๐˜ช๐˜ต๐˜บ] and join us in Boston today & tomorrow โžœ terrapinn.com/bookwodc_tw
*๐˜—๐˜ข๐˜ต๐˜ช๐˜ฆ๐˜ฏ๐˜ต ๐˜ข๐˜ฅ๐˜ท๐˜ฐ๐˜ค๐˜ข๐˜ต๐˜ฆ๐˜ด ๐˜ข๐˜ฏ๐˜ฅ ๐˜ฉ๐˜ฐ๐˜ด๐˜ฑ๐˜ช๐˜ต๐˜ข๐˜ญ ๐˜ฑ๐˜ณ๐˜ข๐˜ค๐˜ต๐˜ช๐˜ต๐˜ช๐˜ฐ๐˜ฏ๐˜ฆ๐˜ณ๐˜ด ๐˜ข๐˜ต๐˜ต๐˜ฆ๐˜ฏ๐˜ฅ ๐˜ง๐˜ฐ๐˜ณ ๐˜๐˜™๐˜Œ๐˜Œ

#WorldOrphanUSA
๐Ÿ—“๏ธ April 22-24
๐Ÿ“ Boston Convention & Exhibition Center
World Orphan Drug Congress USA Verified account @OrphanConf
๐Ÿ“ฃ Meet the Day 1 Chairs of #WorldOrphanUSA 2025!
Tomorrow, these industry leaders are bringing their expertise, energy, and vision to the forefront โ€“ guiding the conversations that matter most in rare disease and orphan drug innovation.

โœฆ ๐ˆ๐ง๐ง๐จ๐ฏ๐š๐ญ๐ข๐จ๐ง & ๐๐š๐ซ๐ญ๐ง๐ž๐ซ๐ฌ๐ก๐ข๐ฉ๐ฌ โ†’ VP, Head of US Medical Affairs, ITF Therapeutics LLC
โœฆ ๐‚๐ฅ๐ข๐ง๐ข๐œ๐š๐ฅ ๐ƒ๐ž๐ฏ๐ž๐ฅ๐จ๐ฉ๐ฆ๐ž๐ง๐ญ & ๐‘๐ž๐ ๐ฎ๐ฅ๐š๐ญ๐จ๐ซ๐ฒ โ†’ Senior Director, Global Medical Affairs, @Alnylam
โœฆ ๐‘๐š๐ซ๐ž ๐€๐๐ฏ๐จ๐œ๐š๐œ๐ฒ โ†’ Executive Director, @SistersHopeALSP
โœฆ ๐€๐๐ฏ๐š๐ง๐œ๐ž๐ ๐“๐ก๐ž๐ซ๐š๐ฉ๐ข๐ž๐ฌ โ†’ VP, Medical Affairs, Rare Diseases and Pediatrics, @premierresearch
โœฆ ๐‚๐จ๐ฆ๐ฆ๐ž๐ซ๐œ๐ข๐š๐ฅ โ†’ Managing Director, @HavasMediaGroup (Havas Life Rare)
โœฆ ๐„๐š๐ซ๐ฅ๐ฒ ๐€๐œ๐œ๐ž๐ฌ๐ฌ ๐๐ซ๐จ๐ ๐ซ๐š๐ฆ๐ฌ โ†’ Director, Expanded Access Program, @argenxglobal
โœฆ ๐€๐ˆ & ๐ƒ๐ข๐ ๐ข๐ญ๐š๐ฅ ๐‡๐ž๐š๐ฅ๐ญ๐ก โ†’ 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 โ€“ from clinical breakthroughs to commercialization and policy. There's still time to register! ๐ŸŽŸ๏ธ๐˜š๐˜ฆ๐˜ค๐˜ถ๐˜ณ๐˜ฆ ๐˜บ๐˜ฐ๐˜ถ๐˜ณ ๐˜ด๐˜ฑ๐˜ฐ๐˜ต ๐˜ฃ๐˜ฆ๐˜ญ๐˜ฐ๐˜ธ ๐˜ฃ๐˜ฆ๐˜ง๐˜ฐ๐˜ณ๐˜ฆ ๐˜ต๐˜ช๐˜ค๐˜ฌ๐˜ฆ๐˜ต๐˜ด ๐˜ด๐˜ฆ๐˜ญ๐˜ญ ๐˜ฐ๐˜ถ๐˜ต:

๐Ÿ”ฌ ๐˜—๐˜ฉ๐˜ข๐˜ณ๐˜ฎ๐˜ข ๐˜ข๐˜ฏ๐˜ฅ ๐˜ฃ๐˜ช๐˜ฐ๐˜ต๐˜ฆ๐˜ค๐˜ฉ๐˜ด โ€“ register now and get ๐ฌ๐ฉ๐ž๐œ๐ข๐š๐ฅ ๐ญ๐ข๐œ๐ค๐ž๐ญ ๐ฉ๐ซ๐ข๐œ๐ข๐ง๐ : terrapinn.com/bookwodc_tw

๐Ÿฅ ๐˜—๐˜ข๐˜ต๐˜ช๐˜ฆ๐˜ฏ๐˜ต ๐˜ข๐˜ฅ๐˜ท๐˜ฐ๐˜ค๐˜ข๐˜ต๐˜ฆ๐˜ด ๐˜ข๐˜ฏ๐˜ฅ ๐˜ฉ๐˜ฐ๐˜ด๐˜ฑ๐˜ช๐˜ต๐˜ข๐˜ญ ๐˜ฑ๐˜ณ๐˜ข๐˜ค๐˜ต๐˜ช๐˜ต๐˜ช๐˜ฐ๐˜ฏ๐˜ฆ๐˜ณ๐˜ด โ€“ secure one of the last remaining free VIP passes: terrapinn.com/bookwodc_tw

๐Ÿ‘ฅ 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