NNPDF @nnpdf
National Niemann-Pick Disease Foundation nnpdf.org Fort Atkinson, WI Joined February 2009-
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Global partnership in action 🌍💙 Thanks to @inpda and John Lee Taggart for capturing moments at our Family Support & Medical Conference. #NNPDF #NiemannPickDisease #Collaboration #INPDA #INPDR #PatientAdvocacy #Caregiver #PatientCommunity bit.ly/GlobalPartnerI…
For those who attended the conference in Orlando – we’d love to hear your thoughts! Please take a few minutes to complete your conference evaluation. Your feedback is greatly valued and helps us make future events even better. Thank you! Share with us: t.ly/a4Zue
Exciting news for the NPC community as Apertura Gene Therapy and the NIH advance early-stage gene therapy research for NPC1, bringing new hope to affected families and the future of treatment. Learn more at t.ly/D81tK #NiemannPick #NPC1 #RareDisease #GeneTherapy
While we put together the full photo gallery, here's a little taste of the weekend. 💙 📸 Have conference photos? Share them: t.ly/PHOTOS-NNPDFCo… We’d love to include them! And tell us: what stood out to you most from the weekend? #NiemannPick #NNPDFConference #NPCommunity
💙 Thank you to every family, researcher, and advocate who joined us in Orlando! 🎉 We are excited to announce the NNPDF Family Support & Medical Conference from July 7-11, 2027 in Milwaukee, WI! 👇 #NiemannPick #RareDisease #NNPDFConference #NNPDF2027
💙 Day 2 of the NNPDF Family Support & Medical Conference is off to a great start! After an incredible kickoff yesterday, we're excited for another day of connecting with families, sharing resources, and building community. #NNPDF #FamilySupport #RareDisease #Community
Happy 4th of July! Wishing you a safe, fun-filled holiday with family, friends, and moments worth celebrating. Have a wonderful Independence Day!
A Heartfelt Thank You to Our Outgoing Board Members 🎉 Our sincere gratitude goes to Liz Heinze, Michael Smith, Paul Merrigan, and Anthony Leoni for their years of commitment to our Board of Directors, for therir outstanding leadership and for the lasting impact they have made.
Congratulations to our new board members! NNPDF is proud to welcome this dedicated group of leaders and thank them for their commitment to advancing our mission of supporting families affected by Niemann-Pick. We look forward to providing hope, support, and progress together.
🩵 Your story matters! Zevra Therapeutics is inviting NPC patients, caregivers, and families to share their experiences. Help others feel less alone and raise awareness. Learn more: nnpdf.org/resources/surv… #NPC #RareDisease
🎉 Our Annual Family Support & Medical Conference is just one week away! Online registration closes July 3 (onsite registration available). View the full agenda at nnpdf.org/agenda/ . Email [email protected] if you are interested in volunteering in the Activity Zone!
📣 Please join us in welcoming Allie as NNPDF's new Family Services Manager! We're excited and look forward to the compassion, dedication, and support she'll bring to individuals and families affected by Niemann-Pick disease. Read Allie's bio at t.ly/zqFWC
The June 2026 NNPDF Newsletter is now available! Packed with important announcements, community updates, resources, upcoming opportunities, and the latest news impacting our Niemann-Pick community, there’s something for everyone to explore. Take a look at conta.cc/4v61XN6
🚨 GEORGIA RESIDENTS: Support rare disease newborn screening expansion! Contact your State Representative & Senator to help advance early diagnosis. Find your legislators at legis.ga.gov/find-my-legisl… Every step forward in newborn screening strengthens the rare disease community!
🚨 CALIFORNIA PATIENTS, CAREGIVERS, AND ADVOCATES are encouraged to testify before the Senate Health Committee in support of AB 1887 which would reduce barriers like prior authorization and step therapy for rare disease patients. 🗓 July 1, 2026 ⏰ 12:00 PM PT 📍 Sacramento, CA
The countdown is on! ⏰ Join the Niemann-Pick community July 9–11 in Orlando for the 2026 NNPDF Family Support & Medical Conference. Connect with families, clinicians, researchers & advocates for a weekend of learning, support, and community. Visit nnpdf.org/conf
The Rare Artist Contest from the EveryLife Foundation for Rare Diseases, celebrates the creativity of people impacted by rare diseases in the U.S. Categories include music, poetry, visual art, and a new short-form video category. Enter by 7/20/2026 at rareartist.awardsplatform.com
Families affected by Niemann-Pick disease rely on Medicaid for healthcare and support services. @RareDiseases has released resources explaining future Medicaid work requirements and what they may mean for members of the rare disease community. Learn more: t.ly/8xVAR
Beren Therapeutics has shared an update outlining its commitment to the NPC community with plans to expand programs supporting families throughout the diagnosis and care journey. Review the full update at bit.ly/4v2KBkZ #NiemannPick #NPC #RareDisease #ClinicalResearch
Cyclo Therapeutics, I... @Cyclodexpert
662 Followers 141 Following Dedicated to developing life-changing medicines for patients and families living with challenging diseases
Beacon for Rare Disea... @RareBeacon
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Cologna Lab @ColognaLab
1K Followers 783 Following biological mass spectrometry, uic, lipids, proteins, neurodegeneration, niemann-pick type c
NPUK @NiemannPickUK
786 Followers 326 Following Niemann-Pick UK is a charity providing support to those affected by Niemann-Pick Disease a group of rare, inherited, life-limiting diseases.
Zevra Therapeutics @zevratx
780 Followers 82 Following We are a commercial-stage company committed to redefining what is possible in bringing life-changing therapies to people living with rare diseases. $ZVRA
Global Genes @GlobalGenes
29K Followers 6K Following Empowering the Next Generation Rare Disease Advocate. Merged with RARE-X Dec. 2022. #CareAboutRare
Stephanie Fischer @RarePOV
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RARE Revolution Magaz... @RareRevolutionM
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Dawn Stites @dawncstites
37 Followers 237 Following
Heather M. Moran @Health_Mama
194 Followers 254 Following 🍏Certified Health Coach, MSEd, 💪Wellness Advocate & Entrepreneur ⭐️ Mom 🤱 Shopping Guru 🛍️Deal Finder 🎉Achieving Dreams ❤️ Happiness 😀= Family & Friends
Linda L Quinn @llquinn2
4 Followers 139 Following
APPEL - Enfermedades ... @peruappel
58 Followers 102 Following Asociación Peruana de Pacientes con Enfermedades de Depósito Lisosomal. Org. Sin fines de lucro 🌐 https://t.co/OcW0FH1H8i 2024
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Curant Health @CurantHealth
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Sruifui @Sruifui576908
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Elsa Rodrigues @ElsaRod_Sci
94 Followers 241 Following [email protected] and Associate Professor @FFULisboa.
Tawdo @Tawdo25069
66 Followers 3K Following
Marlies Hendrickx @Marliesxo
156 Followers 210 Following
Daphne @DaphneKretz222
41 Followers 290 Following I love all animals, vegetarian,disabled due to CADASIL, former Teacher, xAI plz save my brother & from Terminal Gene NOTCH 3 Chromosome 19. Save Rain Forest !!
Smeauez @SmeauezprQHC
64 Followers 2K Following
Cathrine Fog-Tonnese @fog_tonnesen
6 Followers 54 Following
Nturrore @NturrorezPgM
38 Followers 4K Following
Cathy Trzaskawka (Tra... @ctraz
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Thoughshy @ThoughshynZ5lp
43 Followers 3K Following
Jeronimo Lukin @Jer0nimoLukin
515 Followers 2K Following 🇦🇷⚽🏆 🐞| Neuroscience 🧠 | Postdoc @DeRubeisLab
Mikey Wright @mor79103
122 Followers 235 Following
E. Aselage @AselageE99014
0 Followers 3 Following
Liz Heinze @Heinzemomof4
0 Followers 5 Following
Ledesma Lab @LedesmaLab
36 Followers 48 Following Understanding the role of lipids in neuronal physiology and pathology at @CBM_CSIC_UAM #Neuroscience #Lipidosis #NiemannPick
BRETON d'Origine impa... @Romaxi45
133 Followers 1K Following
Behind the Mystery @btmcaresforrare
241 Followers 108 Following Rare diseases, real stories, powerful impact. Behind the Mystery™ raises awareness & inspires change. Watch on @lifetimetv!
Rareatives @rareatives
113 Followers 492 Following Share Your Rare | Amplifying Rare Disease Voices 🎙️ 📖 Sharing stories from the 1 in 10 🧬 New patient-led publication #RareDisease #PatientVoicesMatter
Charles Marques Loure... @CharlesLourenco
68 Followers 472 Following Clinical Geneticist Neurogenetics and Hereditary Metabolic Disorders Specialist
Pediatric Neuroscienc... @PedNeuroscience
412 Followers 317 Following A clinical collaboration between Dell Children’s Medical Center, a part of Ascension Seton, and UT Health Austin, the clinical practice of Dell Medical School
Katie Norris @knorris06
22 Followers 117 Following
JPearson @Jp1J
1K Followers 710 Following Alzheimer Scotland - Director of Policy and Practice at Alzheimer Scotland. Views are my own.
Niemannpickindia @NPD_IND
4 Followers 29 Following
Spino Serebellar Atax... @Konur48728623
174 Followers 3K Following Neuroscience Research. Cerebellum Research S.C.A Group Turkey https://t.co/y9Mx5osjXr
Lindsay Marszal @LindsayMarszal
29 Followers 230 Following Pediatric Palliative Care and Complex Care Physician🦓 I also spend a good portion of my day being bossed around by 🐈⬛🐈⬛🐈⬛
WaynD01 @WaynD01
374 Followers 1K Following Finding undervalued small cap late stage biotechs with no dilution potential for big gains. https://t.co/aY0y1aWkDc
T Lee @rtoddlee
0 Followers 66 Following
angel jose pereira @angelpereira450
937 Followers 7K Following
Trina Paulk @PaulkTrina
2 Followers 31 Following
Christine white @Christinew26587
1 Followers 38 Following
MANZI Ndamukunze @MNdamukunze
549 Followers 3K Following Renforcement des Capacités - AT en finance inclusive (capacity building-TA in i.f.) Engaged for Persons Living with a Rare Disease (PLWRD) with CENTRE-ALLIANCE
hallotronjhon @hallotronj91845
0 Followers 84 Following
HDAwareness2024 @HDAwareness2024
368 Followers 4K Following Awareness leads to great things; Please Get involved what's left of my Family Thanks You. #huntingtondisease #juvenilehuntingtonsdisease #CureHD #CureJHD too.
Calvin Hawe @calvin_hawe
140 Followers 2K Following 🟪 MSTP Student @NUFeinbergMed, '26- 🟦 Chem Research Associate @OctantBio, '24-'26 🟩 Biochem with Neuro focus @NDscience, '20-'24 🟧 https://t.co/SEE3Pzg9Lr
NPFR @NiemannPickFR
5 Followers 81 Following
Gurleen Course5 @GCourse563329
0 Followers 20 Following
Gaucher Community All... @gaucheralliance
12 Followers 24 Following GCA is a non-profit organization supporting Gaucher patients and families through peer-to-peer aid, education, advocacy, resources, and networking.
champs @kejr35778464
140 Followers 1K Following champs ist eine Aktion um Pädiater auf die seltene Erkrankung "Mukopolysaccharidose" (MPS) aufmerksam zu machen.
Mcwewe @Mcwewe199732
5 Followers 30 Following
AbbyStrong Fights NPC @AbbyNPC
9 Followers 74 Following Abby suffers from an ultra rare genetic diagnosis called Niemann-Pick Disease type C. NPC is one of over 100 diseases that cause terminal childhood dementia.
Cyclo Therapeutics, I... @Cyclodexpert
662 Followers 141 Following Dedicated to developing life-changing medicines for patients and families living with challenging diseases
Rare Disease Day @rarediseaseday
42K Followers 3K Following 28 February 2027 is Rare Disease Day. Raising awareness for patients, families and carers around the world that are impacted by rare diseases. #RareDiseaseDay
Ethan Perlstein 1-to-... @eperlste
19K Followers 2K Following ceo @1000cures, ceo @PerlaraPBC (w16 @ycombinator), founder @epalrestat, founder @endrarediseases, founder @smer28rapa
NPUK @NiemannPickUK
786 Followers 326 Following Niemann-Pick UK is a charity providing support to those affected by Niemann-Pick Disease a group of rare, inherited, life-limiting diseases.
Global Genes @GlobalGenes
29K Followers 6K Following Empowering the Next Generation Rare Disease Advocate. Merged with RARE-X Dec. 2022. #CareAboutRare
Stephanie Fischer @RarePOV
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RARE Revolution Magaz... @RareRevolutionM
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CheckRare @CheckRare
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AllStripes @_allstripes
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Professor Mark Walter... @MarkWalterfang
690 Followers 890 Following MBBS Hons PhD DMedSci FRANZCP Neuropsychiatrist and Professor of Neuropsychiatry. Specialising in neurometabolic disorders, cognition, imaging.
Howard Lutnick @howardlutnick
333K Followers 141 Following 41st United States Secretary of Commerce. Amazing wife and 4 great kids.
U.S. FDA @US_FDA
591K Followers 103 Following Our posts are FDA Approved! Privacy Policy - https://t.co/LbTJXYRXVP FDA Authentic Accounts - https://t.co/w2LNbzG7M0
ScreenPlus @ScreenPlusNY
183 Followers 174 Following We are the largest newborn pilot screening program in the United States. Our goal is to identify babies with rare disorders earlier #2025NBS #rarediseases
RDLA @RareAdvocates
6K Followers 2K Following A program of the EveryLife Foundation committed to growing the patient advocacy community and working collaboratively, thereby amplifying the patient voice!
Patient Worthy @PatientWorthy
8K Followers 7K Following We're a resource for engaging, informative content and rare patient news, well done.
FNP España @FNP_Espana
217 Followers 82 Following ¡Bienvenid@s a la Fundación #NiemannPick de España! Investigación científica, divulgación, y orientación familiar. Por una causa, LA VIDA.
Breaking Down Barrier... @BarriersDown
1K Followers 3K Following BDB is a network of organisations working together to improve the lives of people from marginalised communities and addressing health inequalities.
International Niemann... @inpdr_registry
206 Followers 308 Following We document the Niemann-Pick patient experience to advance research and improve lives.
PA Rare Disease Advis... @PARareDisease
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Hope the Zebra @hopethezebra
492 Followers 404 Following Bringing the rare disease community together by sharing Hope, laughter and support. [email protected]
Asociación ASMD Espa... @AsmdSpain
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Association Français... @niemann_pick
44 Followers 178 Following
Saving Belle & Abby @belleandabby
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National MPS Society @MPSSociety
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PPALS @PPALSorg
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Regulatory Affairs Pr... @RAPSorg
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Firefly Fund @Firefly_Fund
228 Followers 416 Following A nonprofit that provides crucial support for research and education necessary to accelerate cures for rare genetic diseases that affect children.
MPS España @mps_lisosomales
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NPC @npcnow
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IntraBio @IntraBio
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Will Evans @willrhevans
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CenterJewishGenetics @jewishgenetics
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jscreen @MyJScreen
827 Followers 1K Following jscreen is a national, non-profit, public health initiative dedicated to preventing genetic diseases & promoting healthy families.
FDA Patient Info @FDAPatientinfo
4K Followers 99 Following The FDA Public Engagement Staff covers a range of topics and conducts numerous activities that are of interest to patients. This account is now archived.
Zhulin Lab @zhulinlab
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World Health Organiza... @WHO
12.7M Followers 2K Following We are the @UN’s health agency working for #HealthForAll Always check our latest tweets for updated advice/information.
Mayo Clinic @MayoClinic
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Ahad Rahim @ProfAhadRahim
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Regan Sherman @ReganESherman
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EURORDIS-Rare Disease... @eurordis
31K Followers 1K Following An alliance of over 1,000 patient organisations working across borders and diseases to improve the lives of all people living with rare diseases.
#makingmemories #npc @prideinlleyton
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Baby Genes Inc @BabyGenesInc
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Addi & Cassi Hempel @AlzheimersTwins
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Hugh Hempel @HughHempel
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NPRF @niemannpickrf
202 Followers 154 Following The NPRF was a charity dedicated to supporting research and trials into Niemann-Pick type C, a rare metabolic disease. It was closed in April 2024You might like
