WORLDSymposia @WORLDSymposia
We're Organizing Research on Lysosomal Diseases worldsymposia.org Joined September 2014-
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Giacomo Chiesi, Head of Chiesi GRD, accepted a 2024 #WORLDSymposium New Treatment Award for velmanase alfa-tycv (Lamzede®), the first US ERT approved for non-CNS manifestations of #AlphaMannosidosis in adults and pediatric patients tinyurl.com/2p8b5uuc #LysosomalDisease
Diego Ardigo, Head of Global Research & Development of Chiesi Group, accepted a 2024 #WORLDSymposium New Treatment Award for Elfabrio® — an approved, safe, and effective ERT for adults with #FabryDisease. Learn more: tinyurl.com/2p8b5uuc #RareDiseases #LysosomalDisease
Bradley Campbell, CEO of Amicus Therapeutics, accepted a 2024 #WORLDSymposium New Treatment Award for the two-component therapy of cipaglucosidase alfa (Pombiliti®) and miglustat (Opfolda®) tinyurl.com/2p8b5uuc #RareDiseases #LysosomalDisease #CareAboutRare
Marking its 20th anniversary, #WORLDSymposium 2024 is celebrating significant milestones and special memories from the past two decades with a commemorative video. #RareDiseases #LysosomalDisease #CareAboutRare youtu.be/vWuq2Pqxxec
Congratulations to Alan Finglas, the recipient of the #WORLDSymposium 2024 Patient Advocate Leader Award! Alan's unwavering determination & practical approach have been instrumental in transforming MSD research. #RareDiseases #LysosomalDisease youtu.be/WCRCkkWYuiA
Elsa Shapiro received the 2024 Roscoe O. Brady Award at #WORLDSymposium on Feb 5. Dr. Shapiro is renowned for groundbreaking work in neurocognitive development measurement. Congratulations, Dr. Shapiro! #RareDiseases #LysosomalDisease #CareAboutRare youtu.be/yQ0nwySIZEc
#WORLDSymposia celebrated a pivotal moment, presenting the inaugural "Catalyst" award to Zachary Thomas. Because of his efforts, The Zachary Thomas Newborn Screening Act will be introduced to the Alabama state legislature this week. #RareDiseases #LysosomalDisease #CareAboutRare
NEW in 2024, WORLDSymposium will present the first "Catalyst" award to Zachary Thomas, an incredible rare disease policy advocate, at the Be The Catalyst Event at 6:00 PM PST on February 4, 2024. worldsymposia.org/catalyst-award/ #CareAboutRare #LysosomalDisease #RareDisease
#WORLDSymposia 2024 Speed Mentoring Event: A fantastic way to connect with incredible lysosomal disease leaders in a fun and casual setting. LAST CHANCE: Speed Mentoring sign-up closes TODAY #CareAboutRare #LysosomalDisease #RareDiseases worldsymposia.org/?p=14728
Meet 1:1 with Lysosomal Disease Experts during #WORLDSymposia 2024 Speed Mentoring Event. Sign-up closes this WEDNESDAY (Jan 10). #CareAboutRare #LysosomalDisease #RareDiseases worldsymposia.org/?p=14728
#WORLDSymposia Poster List Now Online. Over 450 abstracts will be presented at four WORLDSymposium 2024 poster sessions. Excellent opportunity to see, hear and discuss specific research topics directly with the abstract authors. worldsymposia.org/?p=94
LIVE in 30 Days! Early Registration Ends Today for WORLDSymposium 2024. Click here to register: cvent.me/LL4qVz #WORLDSymposia #RareDiseases #LysosomalDisease
Early Registration for #WORLDSymposia 2024 closes in one week: Friday, January 5, 2024. Click here to register: cvent.me/LL4qVz #CareAboutRare #RareDiseases #LysosomalDisease
NEW WORLDSymposium 2024 Full Program now online at WORLDSymposia.org. Includes ground-breaking presentations from industry leaders at Contemporary Forum on Thursday (2/8/2024), and Late-Breaking Science presentations on Friday (2/9/2024). #WORLDSymposia #LysosomalDisease
#WORLDSymposia Speed Mentoring: Incredible opportunity to meet w/ a wide variety of leaders and icons in lysosomal & rare disease space. All appts are pre-scheduled & space is limited. Register for preferred mentors today. bit.ly/3YKLM8W #CareAboutRare #LysosomalDisease
#WORLDSymposia Late-breaking abstract submission closes today, December 1, 2023 at 3:00 PM CST. This is the last opportunity to submit abstracts for poster and/or presentation at WORLDSymposium 2024. Submit abstracts today: tinyurl.com/an7rpwa8 #RareDiseases #LysosomalDisease
Late-breaking Abstract Submission Closes this Friday - Only 4 days left to submit a late-breaking abstract and the last chance to have platform presentation at WORLDSymposium 2024. Visit tinyurl.com/an7rpwa8 to submit your abstracts today! #WORLDSymposia #LysosomalDisease
Congratulations to Alan Finglas, founder of MSD Action Foundation & SavingDylan.com. Finglas to receive 2024 Patient Advocate Leader (PAL) Award at WORLDSymposium 2024 on Tuesday, February 6, 2024 #WORLDSymposia #RareDiseases #careaboutrare tinyurl.com/ym4p5nn9
WORLDSymposium 2024 abstract submission closes on Monday - Only 3 days left to submit an abstract and the last chance to have abstract included in the February MGM Journal. Visit tinyurl.com/an7rpwa8 to submit your abstracts today! #WORLDSymposia #CareAboutRare #RareDisease
#WORLDSymposia is seeking mentors for the 2024 Speed Mentoring session Monday, February 5 from 5:15-6:15 PM. If you have a passion for teaching and want to help foster the next generation of #LysosomalDisease researchers, sign up here: tinyurl.com/mr2rhtmf #RareDisease
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GTです。 @gtEeZop2isdrEgt
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champs @kejr35778464
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RARE Revolution Magaz.. @RareRevolutionM
12K Followers 8K Following Digital magazine giving a voice to those affected by rare conditions and the charities that support them. Contact us: [email protected]
EURORDIS-Rare Disease.. @eurordis
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MPS Society UK, here .. @MPSSocietyUK
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461 Followers 205 Following The IGA represents the interests of Gaucher patients and non-profit Gaucher patient groups worldwide. Find us on Threads: https://t.co/Fv7UMsqkyv
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299 Followers 132 Following #LDRTC is a #NonprofitOrganization with a vision to provide high quality care for patients and families with #LysosomalStorageDisorders and other #RareDiseases
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NIH @NIH
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NINDS Translate @NINDStranslate
3K Followers 244 Following Information on the National Institute of Neurological Disorder and Stroke (NINDS) Division of Translational Research [Privacy Policy: https://t.co/uXZ2BEHgFz ]
Phoenix Nest Inc. @PhoenixNestInc
104 Followers 98 Following Creating treatments for Sanfilippo Syndrome. #CUREmps
C. Javier Alméciga D.. @donisaid
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mnhealth @mnhealth
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1K Followers 584 Following Dedicated to driving research, enabling access to effective treatments and improving the quality of life for children affected by Sanfilippo syndrome.
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2K Followers 531 Following We are a global biotech company focused on discovering, developing & delivering high-quality medicines for people living with rare diseases.
urddad-foundation @TerenceHoey
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1K Followers 688 Following 501c3 nonprofit - Mission: Advocate for & fund research directed toward a cure or treatment options for children with Sanfilippo Syndrome. Video at https://t.co/0WOT6g9S6G
TREND Community @trendcommunity
427 Followers 247 Following TREND Community is a digital health company using artificial intelligence to capture current perspectives of rare communities from their social data streams.
Christine @ChristineWNGF
201 Followers 133 Following Tweets by Christine White, President. #raredisease @NGFCanada
jayne gershkowitz @jgersh1115
308 Followers 182 Following
Deborah V. Requesens @dvrequesens
160 Followers 146 Following Director Jumpstart Program at Orphan Disease Center University of Pennsylvania
During @WORLDSymposia, @atrevino had the opportunity to participate in a new program titled, “The Patient Voice: Is Anyone Listening?” to discuss our work with @NAMICommunicate to help support the mental health of people living with #Fabry disease. Watch this video to learn more.
While accepting a @WORLDSymposia New Treatment Award for work to support the #AlphaMannosidosis community, Giacomo Chiesi, Head of #ChiesiGlobalRareDiseases, reaffirmed our commitment to deliver innovative solutions for people living with #RareDiseases. b.link/63zrxnpy
We were excited to receive a @WORLDSymposia New Treatment Award recognizing our work to develop a therapeutic option for people living with Fabry disease. Diego Ardigò, Head of R&D, shares a message of gratitude for the #Fabry community. b.link/oqury314
Giacomo Chiesi, Head of #ChiesiGlobalRareDiseases, shares highlights from our experience at @WORLDSymposia – including the special recognition we received at the New Treatment Award ceremony for regulatory approvals in #Fabry disease & #AlphaMannosidosis: r.chiesi.com/00v0fv0g
Join us this #RareDiseaseDay for a story of collaboration & hope. Together, we're pioneering a clinical trial spanning eight #RareDiseases, with the goal of ensuring all babies are born healthy. Thanks @WORLDSymposia & our great partners youtu.be/x8dKAZUGBDc #LysosomalDisease
#ICYMI We were proud to receive two New Treatment Awards at the 20th Annual @WORLDSymposia in San Diego! Our team was grateful for this special opportunity to celebrate important achievements for the #Fabry disease and #AlphaMannosidosis communities.
Thank you Livia Finoti from Children's Hospital of Philadelphia for this important iPSC work on MSD. This will enable further MSD research. It was wonderul to see this important poster presented at @WORLDSymposia in San Diego. Thank you @AhrensNicklas #TreatCureMSD
Aaaand that’s a wrap. The sixth of @chocchildrens / @CHOCDocs platform presentations at the 2024 #worldsymposia is finished. Also completed a commentary re: reliance of #raredisease tx on industry. Shareholder value is NOT aligned w interests of pts and their families
2024 #WORLDSymposia New Treatment Award was presented to @ChiesiGRD for enzyme replacement therapies (ERTs) for people living with #Fabry disease and #AlphaMannosidosis This is an important recognition on how we can make a greater impact to improve #patients’ lives #RareDiseases
CheckRare was delighted to attend this year's #WORLDSymposia in San Diego. Be on the lookout for our coverage in the coming weeks, featuring interviews with KOL and Dean Suhr of MLD Foundation #CheckRare #RareDisease
Sophie Thomas and the team from our sister charity, @MPSSocietyUK , are at #WORLDSymposium in San Diego. Read more below.
Hello from a very wet and windy San Diego! It’s been a great start to #WORLDSymposium. Our team have been busy presenting research findings and attending sessions. Read about what they're up to: buff.ly/49upm0d
An excellent talk by @rayywang76 @WangLysolab at #WORLDSYMPOSIUM on gene therapy for MPS I
My first international conference at #WORLDsymposium in San Diego was an absolute blast!! Learned so much & excited to dive into next steps re the pathophys of small vessel disease in the heart & brain in #fabrydisease 🫀🧠🧬
Today was the final day of the 20th Annual WORLDSymposium in San Diego. BDSRA President & CEO Amy Fenton Parker brings you up to speed on the latest news and information related to the Batten disease community. #BattenAdvocatesForACure #WORLDSymposium #BattenDisease
In the #WORLDSymposium 2024 in San Diego, learning about new therapies in #LysosomalDiseases for our patients, with a very nice multidisciplinary Spanish Delegation #TimeforTraslationalScience #Metabolics #Genetics #RareDiseases #CareAboutRare Celebrating #WomenInScienceDay
The conclusion of the 20th Annual WORLDSymposium in San Diego marks a significant milestone in the pursuit of knowledge and advancements for the Batten disease community. #WORLDSymposium #BattenDiseaseResearch #KnowledgeSharing
Orchard Therapeutics’ arsa-cel, approved as Libmeldy in Europe, a gene-edited cell therapy intended to treat MLD, has enabled restoration of ARSA enzyme activity among patients with the late juvenile (LJ) form of MLD. #WORLDSymposium cgtlive.com/view/orchard-t…
That’s a wrap on @WORLDSymposia 2024! It's an honor to work alongside @FabryOrg and other organizations to change the way diseases are viewed, for they are so much more than symptoms alone. Learn more about our work uncovering insights in #FabryDisease: bit.ly/4bJKqlp
Amazing results so far. We were delighted to see this @WORLDSymposia in San Diego. Thanks to @hrbireland @HRCIreland for helping to support this superb research project
@Vi_Pham20 presents some amazing results on ex vivo gene therapy for MSD. This was co-funded by @hrbireland & MSD Action Foundation @ the @AhrensNicklas & Rivella labs at Children's Hospital of Philadelphia. Vi was awarded with a very prestigious young investigators award also👏
We were honored to be presented with 2 New Treatment Awards at @WORLDSymposia based on regulatory approvals in #Fabry disease & #AlphaMannosidosis. This recognition is a testament to our commitment to deliver innovative therapies & solutions for people affected by rare diseases.
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