Rare Disease Report @RareDR
Breaking news, patient stories & FDA updates within the rare disease community. Listen to our podcast: https://t.co/xUkFDfCDUV, hosted by @GiulianaGrossi hcplive.com/clinical/rare-… @MDMagazine Joined April 2012-
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Big news in #RareDisease this morning! The US FDA has approved mavorixafor (XOLREMDI; @X4Pharma) as the first agent with an indication for patients with WHIM syndrome 12 years or older based on data from the 4WHIM trial. Learn more on @HCPLiveNews: hcplive.com/view/fda-appro…
That is it from us for #RareDiseaseDay2024! Thank you to all those who acknowledged the day and an even bigger thank you to all those pushing the field into the future! hcplive.com/clinical/rare-…
In August 2023: The US FDA approved pozelimab-bbfg (Veopoz) for the treatment of adult and pediatric patients 1 year of age and older with CHAPLE disease—the first therapy to receive such an indication. hcplive.com/view/fda-appro… #RareDiseaseDay #RareDisea
Gina Choi, MD, A hepatologist from UCLA, breaks down the unmet need and potential for improvement within the management of autoimmune hepatitis. hcplive.com/view/autoimmun… #RareDiseaseDay #RareDiseaseDay2024
In this interview: Safer, a patient with primary sclerosing cholangitis, discusses how symptoms and quality of life often go unaddressed in clinician-patient interactions. hcplive.com/view/what-clin… #RareDiseaseDay #RareDiseaseDay2024
From our This Year in Medicine series: @DrCaliff_FDA joins @HCPLiveNews to share perspectives on many topics, including gene and cell therapy development for rare diseases. Learn more: hcplive.com/view/reviewing… #RareDiseaseDay #RareDiseaseDay2024
From #ASH2023: Phase 3 data show etranacogene dezaparvovec (EtranaDez), a gene therapy from Hemgenix, significantly reduced annualized bleeding rates and the number of total bleeds in hemophilia B. hcplive.com/view/etranacog… #RareDiseaseDay #RareDiseaseDay2024 @CGT_Live
From October 2023: The US FDA approved an sNDA for vosoritide (VOXZOGO) to increase linear growth in pediatric patients ≤5 years of age with achondroplasia. hcplive.com/view/fda-appro… #RareDiseaseDay2024 #RareDiseaseDay
Algaille syndrome represents a difficult-to-treat #RareDisease in hepatology. In late 2023, data from the odevixibat program suggests the IBAT inhibitor could provide significant benefit for these patients. hcplive.com/view/odevixiba… #RareDiseaseDay2024
In January, @alkeuspharma announced positive interim data from the TEASE-3 clinical trial showing gildeuretinol slows the progression of Stargardt Disease, a rare ophthalmic disorder, for up to 6 years. hcplive.com/view/michael-g… #RareDiseaseDay
In the last 12 months, the management of #IgANephropathy has undergone seismic change, with the first agent (oral budesonide; Tarpeyo) receiving full approval in late 2023. Learn more about the space and ongoing development in our #IgAN resource center: hcplive.com/clinical/berge…
Today is #RareDiseaseDay2024! All day we are highlighting the most popular coverage this year from @HCPLiveNews! To kick off the day, here's a clip where @MasriAhmadMD calls attention to the need for reform in developing therapies for #RareDisease. hcplive.com/view/experts-p…
A #RareDisease win last night: The FDA has approved nirogacestat tablets (Ogsiveo) to treat progressing, painful desmoid tumors. Learn more about the systemic drug here: hcplive.com/view/fda-appro… @HCPLiveNews $SWTX
A year of research emphasizes the significance of collaboration, standardized approaches, and targeted therapies in rare pulmonary diseases. ow.ly/qIBr50Pmehl
The latest feature in The Unreached series delves into integrative care with expert perspectives from @GuyYoungMD, @DrTitiFasipe, and more. Read here: ow.ly/pOFQ50Pc49O @TexasChildrens @ChildrensLA @Nemours @SCDAAorg @GiulianaGrossi #integrativemedicine #chornicdisease
Individuals seeking contraception will be able to access norgestrel without consulting a health care provider, reducing barriers to contraception access. ow.ly/ra7950PaEzC #FDA #birthcontrol
Efanesoctocog alfa demonstrates promising results in new phase 3 data emphasizing the drug's potential to address unmet needs within the hemophilia community. ow.ly/FipW50P6YTk #ISTH2023 @isth #raredisease
Psychological factors also come into play, as patients may have varying appetites for risk and preferences for treatment regimens, Dr. Guy Young explains in an interview with @GiulianaGrossi ow.ly/721P50P6Xl9 @GuyYoungMD @ChildrensLA #hemophilia #genetherapy #prophylaxis
The American Indian/Alaska Native population showed the largest increase in maternal mortality ratios rising from 14-49.2, while the Black population consistently had the highest median state ratios, increasing from 26.7-55.4 ow.ly/V7t950P2O7o #FetalMedicine #Womenshealth
"Hemlibra is currently the only approved medicine for people with hemophilia A with or without factor VIII inhibitors that can be self-administered subcutaneously once weekly, every 2 weeks, or every 4 weeks," Dr. Juliana Biondo explains. ow.ly/hsXO50P28Oo #hemophilia #FDA
EURORDIS-Rare Disease.. @eurordis
32K Followers 2K 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.
Beacon for Rare Disea.. @RareBeacon
12K Followers 11K Following Beacon is a UK-based charity that is building a united rare disease community with patient groups at its heart. Previously known as Findacure.
Effie Parks @OnceUponAGene
5K Followers 3K Following Nuclear Advocate | Award Winning Podcaster | Speaker | Captain Connection | #RareDisease #RareMama to my sweet, Ford, who lives with #CTNNB1 🦓
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]
NORD @RareDiseases
41K Followers 3K Following National Organization for Rare Disorders (#NORD) is the voice of the #RareDisease community. Official US sponsor of #RareDiseaseDay.
Global Genes @GlobalGenes
30K Followers 6K Following Empowering the Next Generation Rare Disease Advocate Merged with RARE-X Dec. 2022. #CareAboutRare
Cambridge Rare Diseas.. @camraredisease
8K Followers 7K Following Improving #RareDisease lives. Creators of #RAREfest, #RAREsummit, Companies Forum & #UniqueFeetCam https://t.co/bWJ8DVF9iw
Ethan Perlstein @eperlste
17K Followers 2K Following ceo @PerlaraPBC (@ycombinator W16), ceo @epalrestat, solo vc @AngelList, evo pharmacologist, mTOR worshipper, HODLer since '14, cofounder of Kai & Luca, bio/acc
Patient Worthy @PatientWorthy
8K Followers 7K Following We're a resource for engaging, informative content and rare patient news, well done.
RDI @rarediseasesint
5K Followers 699 Following Rare Diseases International - the global alliance of rare disease patients & families
EveryLife Foundation @EveryLifeOrg
7K Followers 3K Following Nonprofit org. dedicated to advancing the development of treatment & diagnostic opportunities for rare disease patients through science-driven public policy.
FEDER | Enfermedades .. @FEDER_ONG
39K Followers 8K Following 👉🏻 Somos la esperanza de 3 millones de personas con #enfermedadesraras 🍀 Representamos a 418 organizaciones de pacientes y a 1.546 patologías poco frecuentes
Dr. Tracy Dixon-Salaz.. @TracyDixonSalaz
3K Followers 974 Following Mom. Rare Disease & LGS Advocate. Neuroscientist. The largest most neglected healthcare resource, worldwide, is the patient. Executive Director @LGS_Foundation
Stephanie Fischer @RarePOV
7K Followers 6K Following #Raredisease patient advocate & #stroke survivor.🦓 Member of @PARareDisease. Opinions are my own.
Genetic Alliance UK @GeneticAll_UK
16K Followers 2K Following National charity working for everyone affected by genetic, rare and undiagnosed conditions. We run the campaign @rarediseaseuk and support network @SWAN_UK.
Genomics England @GenomicsEngland
30K Followers 2K Following We’re working to enable faster and deeper genomic research, to bring genomic healthcare to all who need it.
Medics4RareDiseases @M4RareDiseases
5K Followers 2K Following Medics4RareDiseases is driving an attitude change towards rare diseases amongst medical students and doctors in training #DareToThinkRare
FamilieSCN2A @FamilieSCN2A
2K Followers 1K Following Our vision is to find effective treatments and a cure for SCN2A disorders.
Rare Patient Voice @rarepatientvoic
2K Followers 4K Following We help clients find rare and non-rare disease patients and caregivers for studies, and connect patients and caregivers with paid research opportunities.
Rare Disease UK @rarediseaseuk
22K Followers 2K Following National campaign run by @GeneticAll_UK to improve the lives of those affected by rare conditions and all who support them. #RareDisease.
January Payne @januarypayne
1K Followers 2K Following Health communicator. Health equity advocate. Former health/medical journalist @washingtonpost @usnews. Dog mom. 🐶🐩🐕 Opinions are my own.
El Sheff @outofthebredbin
55 Followers 237 Following Anti-mag/CIDP, VVS, FND. The lines blur. Making memories while 'icing the cake'
Glycocast @Glycocast
19 Followers 92 Following A podcast/journey of families, sugars and rare disorders. Follow us at: https://t.co/AqY934HKa0
Israel Shamay 🗣️.. @Israel_Shamay
110 Followers 913 Following עיתונאי מקור ראשון, עורך-דין ומגיש פודקאסט האקטואליה בוא נחשוב. אשכול לינקים להאזנה: https://t.co/abBVYCg0yP Journalist, Lawyer, current affairs podcast host
Team PHenomenal Hope @teamphhope
5K Followers 807 Following Racing to make a difference in the lives of those with pulmonary hypertension. #LetMeBeYourLungs
J. Scott Parker @JayePsy
33 Followers 321 Following enjoyed 7 years of practically freelance humanities @ VCU but I am a philosopher musician...solipsistic? perhaps...namaste
Elsie Megan @poemsandparents
18 Followers 224 Following My cathartic and honest (poetic) ramblings about the ups and downs of parenting a child with severe learning disabilities.
Autumn Ryan @AutumnR53821595
142 Followers 872 Following Hayden’s wife, Beau & Charlie’s mom, Autism/PTHS mom, Proud Creek, Boomer Sooner, Blue Heelers rock, Love Conquers All
f4xoqcj0t8mch7j2 @f1d20dgj8dd
0 Followers 46 Following
Nathalie Vanden Eynde @VEyndeNathalie
9 Followers 138 Following
Denville Partners @denvillepartner
398 Followers 2K Following #Executivesearch firm focused on supporting clients in Biotechnology, Pharmaceuticals, and industry related nonprofits.
DNAUnited @DNAUnited2024
0 Followers 8 Following
Focus On Sickle Cell .. @UgandaSickle
250 Followers 1K Following FOSCA exists to provide a multi-faceted approach to combating SCD that includes testing, research, raising awareness, and providing psychosocial support.
ClinRheumatol @ClinRheumatol
7K Followers 3K Following Clinical Rheumatology - Journal of the International League of Associations for Rheumatology 2022 Impact Factor: 3.4 Editor-in-Chief: Prof. Adewale Adebajo
Global MedCon @GMedCon
2 Followers 30 Following Global MedCon is your Expert Consulting Partner in Medical Technology, Lifesciences, Genetics, Clinical & Market Research and Regulatory Solutions.
Juhyeon Hong @hongjuhyeon5
42 Followers 36 Following
Anne Micks @annedmicks
249 Followers 900 Following Chair Irish EDS 🇮🇪 Rare Disease Advocate| Board Member -Rare Diseases Ireland & Rare Ireland |Former Mgt Consultant. Views are my own.
Blood MedEd @bloodmeded
211 Followers 861 Following Here for sharing information & bringing awareness to 🩸 disorders &🩸diseases. RTs ≠ endorsements. Help us grow, follow us! #hemophilia #cancer #scd #dvt
me @Silence_And_I
44 Followers 219 Following
Manchester Rare Condi.. @mft_iMRare
602 Followers 234 Following Official Manchester Rare Conditions Centre account. Improving lives of people with rare conditions
Owoeye David @davoshalom
772 Followers 2K Following God Lover. Infection Prevention & Control Specialist. Sickle Cell Warrior & Advocate. Author. https://t.co/9z1lvAfJYh…
Jinok Lee @Jinokleesnubh
0 Followers 1 Following
Mike @mike286347
87 Followers 5K Following
Chromocell @ChromocellThera
31 Followers 114 Following Chromocell is a biotechnology company developing & commercializing novel, non-opioid, non-addictive therapeutics to alleviate pain & associated conditions $CHRO
ɈɿoƆ ǝꓷ γnnǝ�.. @KennyDeCort
424 Followers 880 Following Overheid: 🇧🇪 https://t.co/xMlEIFUS4t BE-Alert: https://t.co/1bTCYIZiAF Zelfmoordlijn: https://t.co/OG4akuwHIA Het weer: https://t.co/mlf2TsLbfR
SHANTANU SENGUPTA @SHANTAN03364656
109 Followers 2K Following
JARDIN_Joint-Action @jardin_EU_JA
90 Followers 131 Following JARDIN is created to improve the accessibility of the ERNs for people living with rare diseases or complex conditions in EU member states.
zelalem Geletu, MD @GeletuZelalem
50 Followers 346 Following
Skeletal Dysplasia @skeletal_dys
9 Followers 65 Following R&D on pharmaceutical treatments, approved + in trial, and advancements in surgical treatment options for #skeletaldysplasia – e.g., #dwarfism | @ach_research
Salivary Gland Cancer.. @SGCancerUK
653 Followers 705 Following Working for those with all types of #SalivaryGlandCancer, in the UK & beyond. Patient-clinician collaboration to support, further research & improve outcomes.
Sherook @docsherook
101 Followers 2K Following Hematologist in Progress. Fellow traveller. Rider. car and bike enthusiast. Freethinker. in love with nature and myself. Humanity is my religion. Eden's Dad.
cillianboushel @cillianbou36694
260 Followers 1K Following
Arafhart Kibirige @ArafhartK
34 Followers 252 Following
GPSolis @solis_gp
28 Followers 182 Following Senior Researcher - Molecular Cell Biology #Science #CellBiology #GPCR #GNAO1 #RareDisease #EERR
Rob Hynds @robhynds
2K Followers 2K Following Scientist studying epithelial biology, stem cells and cancer @EpiCENTR_ICH. ⚽️ @BCFC 🏃🏻♂️ @LondonHeathside
Michelle Mayer @MichelleAMayer
11 Followers 174 Following
megweatherly @megweatherly
5 Followers 94 Following
Emma McEvoy @DrEmmaMcEvoy
1K Followers 2K Following Assistant Professor in Law at Dublin City University, School of Law and Government. Tweets on procurement law, health procurement, social enterprise
jlrxabar9fl9wmb @xvx80fcb5454oi
0 Followers 224 Following If you want to make friends, you can contact me anytime TG：https://t.co/u0q65Spmxf
Lauren Rollit @lauren_rollit
113 Followers 1K Following MA Public History candidate @CU_History, alum @JSchool_CU & @HUMSCarleton. Views my own. she/her 🏳️🌈
Jack Trujillo @JackTru16084055
44 Followers 48 Following
Hadeel Jambi @JambiHadeel
11 Followers 84 Following
EURORDIS-Rare Disease.. @eurordis
32K Followers 2K 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.
Rare Disease Day @rarediseaseday
44K Followers 3K Following 29 February 2024 is Rare Disease Day. Raising awareness for patients, families and carers around the world that are affected by rare diseases. #RareDiseaseDay
Beacon for Rare Disea.. @RareBeacon
12K Followers 11K Following Beacon is a UK-based charity that is building a united rare disease community with patient groups at its heart. Previously known as Findacure.
NORD @RareDiseases
41K Followers 3K Following National Organization for Rare Disorders (#NORD) is the voice of the #RareDisease community. Official US sponsor of #RareDiseaseDay.
Global Genes @GlobalGenes
30K Followers 6K Following Empowering the Next Generation Rare Disease Advocate Merged with RARE-X Dec. 2022. #CareAboutRare
Ethan Perlstein @eperlste
17K Followers 2K Following ceo @PerlaraPBC (@ycombinator W16), ceo @epalrestat, solo vc @AngelList, evo pharmacologist, mTOR worshipper, HODLer since '14, cofounder of Kai & Luca, bio/acc
EveryLife Foundation @EveryLifeOrg
7K Followers 3K Following Nonprofit org. dedicated to advancing the development of treatment & diagnostic opportunities for rare disease patients through science-driven public policy.
FEDER | Enfermedades .. @FEDER_ONG
39K Followers 8K Following 👉🏻 Somos la esperanza de 3 millones de personas con #enfermedadesraras 🍀 Representamos a 418 organizaciones de pacientes y a 1.546 patologías poco frecuentes
Stephanie Fischer @RarePOV
7K Followers 6K Following #Raredisease patient advocate & #stroke survivor.🦓 Member of @PARareDisease. Opinions are my own.
Genetic Alliance UK @GeneticAll_UK
16K Followers 2K Following National charity working for everyone affected by genetic, rare and undiagnosed conditions. We run the campaign @rarediseaseuk and support network @SWAN_UK.
Genomics England @GenomicsEngland
30K Followers 2K Following We’re working to enable faster and deeper genomic research, to bring genomic healthcare to all who need it.
Medics4RareDiseases @M4RareDiseases
5K Followers 2K Following Medics4RareDiseases is driving an attitude change towards rare diseases amongst medical students and doctors in training #DareToThinkRare
Rare Patient Voice @rarepatientvoic
2K Followers 4K Following We help clients find rare and non-rare disease patients and caregivers for studies, and connect patients and caregivers with paid research opportunities.
Rare Disease UK @rarediseaseuk
22K Followers 2K Following National campaign run by @GeneticAll_UK to improve the lives of those affected by rare conditions and all who support them. #RareDisease.
David Fajgenbaum, MD @DavidFajgenbaum
11K Followers 3K Following @Penn physician-scientist-patient who 'cured thyself' | Advancing @CureCastleman + @EveryCure | National Bestseller #ChasingMyCure https://t.co/26OS13etrF
RDLA @RareAdvocates
6K Followers 3K Following A program of the EveryLife Foundation committed to growing the patient advocacy community and working collaboratively, thereby amplifying the patient voice!
NCATS @ncats_nih_gov
16K Followers 702 Following Official account of @NIH's National Center for Advancing Translational Sciences. Privacy Policy: https://t.co/f5qzfKqqaR engagement ≠ endorsement
CORD @raredisorders
5K Followers 730 Following CORD Mission: Provide a strong common voice to advocate for health policy and a healthcare system that works for those with rare disorders.#Canada4Rare
Monica Dudley-Weldon @mlweldon5
3K Followers 3K Following DC’s Most Eligible Bachelorette | Son 6th diagnosed #SYNGAP1 |Author | KOL | Fnd @syngap1_Fnd | Legal Analyst | Life Sciences | Investor VAMO @pharmaboardroom
Unique @Unique_charity
8K Followers 2K Following Unique supports & informs anyone born w/a rare chromosome or gene disorder, their families & carers. Eurordis Patient Organisation Award winner. https://t.co/GkIBlYg5c5
Andrew Korda @KordaAndrew
10 Followers 39 Following
MRC Unit The Gambia a.. @mrcunitgambia
8K Followers 352 Following We are leading health research in West Africa to save lives and improve health across the world.
Scleroderma Awareness.. @SclerodermaKC
397 Followers 415 Following Scleroderma Awareness of Kansas City to educate and make people aware of scleroderma in their community.
Institute for Health .. @IHME_UW
63K Followers 4K Following A research center at @UW quantifying global health challenges like #COVID19: https://t.co/VbwttwAVL4 & making the #GBDstudy: https://t.co/oZEYdt6DF5 #PopulationHealth
Sidney Kimmel Cancer .. @KimmelCancerCtr
11K Followers 2K Following An NCI-designated comprehensive cancer center working to eradicate cancer in Philadelphia and beyond.
Haydar Frangoul @hfrangoul
283 Followers 277 Following Pediatric Hematologist-Oncologist | Medical Director of Pediatric Transplant | Sarah Cannon Research Institute
HCPLive Rheumatology @HCPLiveRheum
8K Followers 874 Following HCPLive Rheumatology provides clinically impactful news and updates for HCPs treating rheumatic disease.
HCPLive Cardiology @HCPLiveCardio
993 Followers 588 Following HCPLive Cardiology provides clinically impactful news and updates for practitioners in cardiovascular medicine.
Barry Byrne @barrybyrne1
337 Followers 231 Following Barry Byrne, M.D., Ph.D. is Director of the Powell Center at the University of Florida. The Center is focused on rare disease research and therapy.
Dorababu SannaReddy @DoraSannaReddy
45 Followers 552 Following
KirstC @wipaweeee
402 Followers 2K Following 💉💉💉 💉MFA USYD, Artist Design #autoimmunewarrior, deaf 🤟 she/her, Disability and Rare Disease activist 😷
GW SMHS @GWSMHS
9K Followers 5K Following The George Washington University School of Medicine & Health Sciences (#GWSMHS). Follow/RT ≠ endorsement.
CDKL5 Art For HopeLov.. @ArtForHLC
445 Followers 2K Following Marissa, proud board member of @CDKL5_IFCR 💚 Creating art with my son who lives with #CDKL5. Facebook & Instagram @artforhopelovecure 🎨
Osama El Safi @OsamaElSafi5
541 Followers 3K Following Professor of pediatric hematology and oncology
dr.akhilesh khare @drakhileshkhar1
148 Followers 1K Following pediatrician and pediatric endocrinology fellow
alexandra geary @Alextremophile
507 Followers 732 Following @[email protected] Bioinformatician CRDG lab (@nickywhiffin) human genetic disease and evolution. (she/her)
Jack Lepping @jack_lepping
118 Followers 309 Following
Whiwon Lee @WhiwonLee
78 Followers 364 Following genetic counselor, genomics enthusiast, PhD candidate in health services research, she/her
Brandi @brandi_whine
270 Followers 3K Following MS & Narcolepsy Advocate. HSCT Veteran Northwestern 2015. Twitter OG, Weather g33k. WCNC Spotter. Duke. Sharks. Frenchie mom. Tiffany Blue. Jimmy Ear Wold.
Rare Aware @rare_aware
386 Followers 779 Following Our RARE ACTIVist programme needs YOU to help us raise awareness and funds for our cause, our patients, and ultimately the rare disease community.
Jessica McNair, MSN, .. @jessiccaniccole
178 Followers 903 Following Board Certified Family Nurse Practitioner 🥼
Halim Çetinel @drcetinel
268 Followers 3K Following
Ilyas Ibrahim @Ilyas_Archim
82 Followers 1K Following Interested in hematopoiesis and hematopoietic stem cells.
AHN @AHNtoday
11K Followers 1K Following We’re a 14 hospital health system dedicated to providing exceptional care in PA and NY. 👩⚕️💚👨⚕️ We're proud to be part of Highmark Health.
Sylvester Comprehensi.. @SylvesterCancer
8K Followers 2K Following South Florida’s Only NCI-Designated Cancer Center. Moving discoveries from research labs to patients and out to the community. #OnlySylvester
Keith N. Fargo, PhD @KeithNFargo
822 Followers 126 Following Director of Scientific Initiatives, Lewy Body Dementia Association
CMT Research Foundati.. @CMTRF
771 Followers 2K Following The CMT Research Foundation has one mission: Deliver treatments and cures for #CMT. We fund the most promising research solely focused on drug development.
SwanBio Therapeutics @SwanBioTx
226 Followers 187 Following Fueled by a unique company culture, we are researching & developing life-changing gene therapies for people with devastating inherited neurological conditions.
Relapsing Polychondri.. @RP_Organization
1K Followers 2K Following The RP Foundation’s purpose is to facilitate awareness, education, and research to improve the quality of life for patients with relapsing polychondritis (RP).
BARE @BAREInc_org
287 Followers 411 Following Our mission is to drive research and education for better outcomes in treatment and management of care within the Biliary Atresia community.
GNmd @1987gala
106 Followers 644 Following The doctor by heart, Clinical Biochemical Geneticist. specialist in rare disorders, IEM, renal genetic disorders
RareNewEngland @RareNewEngland
705 Followers 493 Following Rare New England's Mission is to bring together New England patients and families touched by rare and complex disorders. We build foundations for support, creat
The Rare Disorder Pod.. @RareDisorderPod
134 Followers 37 Following A podcast dedicated to raising awareness for rare diseases and disorders 5K+ active listeners, 1K+ followers, 35 ep. & growing | Host @ShivaniGVyas
GradyHealth @GradyHealth
9K Followers 3K Following Atlanta can't live without Grady. Grady Hospital + 7 Neighborhood Centers Leaders in Trauma, Stroke, Burn, Heart, Cancer, Ortho, HIV
ISurvivor Lisa Deck @lbdeck
2K Followers 1K Following ❤ Mom & Wife * Experienced Speaker & Patient Advocate * #StrokeSurvivor * Founder Sisters@Heart * #Moyamoya warrior; #RareDisease Fighter; #Heart Spokeswomen
Andra Stratton @livinlavidalopo
1K Followers 554 Following Co-Founder of Lipodystrophy United Program Associate for #RareAsOne @CZIScience. Always hungry. Sometimes funny. Opinions are my own.
Giuliana Grossi @GiulianaGrossi
190 Followers 737 Following Editor 💬 @AJMC_Journal Reporting disparities in healthcare at the Center on Health Equity & Access ✌🏻my tweets
MUSC Health @MUSChealth
15K Followers 1K Following For the ninth year in a row, MUSC Health University Medical Center in Charleston has been named the top hospital in South Carolina by U.S. News & World Report.
FSR @StopSarcoidosis
3K Followers 632 Following The Foundation for Sarcoidosis Research (FSR) is the leading international organization dedicated to finding a cure for sarcoidosis and improving care.
Moira Gunn @biotechnation
3K Followers 325 Following Host of BioTech Nation, the biotech segments from Tech Nation on NPR Talk. Subscribe thru any podcast outlet at https://t.co/b3R5ja7gvh
Infection Control Tod.. @ICT_magazine
7K Followers 656 Following Infection Control Today provides timely, informative news, articles and educational opportunities to infection prevention and control professionals.
Patrick Campbell @RealPatCampbell
704 Followers 646 Following Managing editor: @HCPLiveNews. Producer: #DiabetesDialogue & #DontMissABeat Formerly: @njdotcom | @starnewsgroup | @poconorecord | Opinions are my own. #Ramily
Kevin Kunzmann @NotADoctorKevin
814 Followers 710 Following @HCPLiveNews. @ContempPeds. @ContempOBGYN. Writing on medicine, tweeting about it too. Got my degree from @RowanUniversity, and my anxiety from the @Mets.
Gavin Corcoran @gavincorcoranmd
252 Followers 618 Following New medicines for serious diseases; helping people to live healthier lives - LGBTQ especially. Chief R&D Officer at Sio Gene Therapies. All opinions are mineThe Foundation for Sarcoidosis Research (FSR) is pleased to announce the appointment of Calvin Harris Jr., CPA and Joel D. Rosen, Esq. to the FSR Board of Directors. Read the full press release: loom.ly/AO7hvTs #sarcoidosis
We’re presenting late-breaking Phase 3 data for children under 12 with severe #hemophilia A at #ISTH2023. Learn more about these results and how we’re breaking barriers in hemophilia. spkl.io/601749IcX
Breaking News: Dr. Margaret Ragni explains the potential benefits of Roctavian for Hemophilia A, including reduced bleeds and enhanced quality of life. #Roctavian #Hemophilia #RareDisease ow.ly/MVCw50P1W9X @GiulianaGrossi @RareDR
A new analysis of @CDCgov data shows up to 1 in 5 US adults have been diagnosed with #depression as of 2020. Concerningly, a greater rate of depression is observed in younger adults. Read more about the data: ow.ly/POwR50OZVpQ
The 3rd edition of Qazi Corner goes live today! Stay tuned for 5 new articles, reviews and analyses from our quarterly #gastro expert contributors today. First up, @RavShahMD of @ClevelandClinic reviews upadacitinib data for Crohn's disease: ow.ly/vLCp50P0rXL
#BREAKING: The @US_FDA has approved the first #genetherapy for the treatment of adults with severe #HemophiliaA without antibodies to AAV5 detected by an approved test, giving @BioMarin's valoctocogene roxaparvovec-rvox (Roctavian) the go-ahead. Read: ow.ly/TbSY50P0V62
We’re off to the races at @SocietyPedsDerm annual #PreAAD2023! Stay tuned for live interviews later today!
Polypharmacy, defined as ≥5 concomitant drugs, was significantly higher in patients with #PsoriaticArthritis when compared with controls (49% vs 17%, respectively). ow.ly/Nc9950NkxwM
A clinical trial is testing whether a smartphone app called SPARC, which prompts users to perform meditative breathing exercises and contains disease-relevant education modules, can help ease fatigue and improve life quality for people w/ #sarcoidosis: loom.ly/YnH791s
Check out the latest #cardiology case report from Dr. Pregerson! ow.ly/llv250Nh392
Higher baseline Disease Activity Index for Psoriatic Arthritis (DAPSA) was associated with reduced probability of achieving low disease activity or remission. Learn more: ow.ly/4v5950Nh9K3 #PsoriaticArthritis
I'm continuously inspired by the stories I get to share and the overall strength of the rare disease community 🤍 Happy #RareDiseaseDay2023 🦓
Raising Awareness on #RareDiseaseDay @KerryLeeson with her Son, Kion headed to the Parliamentary Rare Disease Event in Westminster, London. Find out more about what is happening for Rare Disease Day on the ASUK website alstrom.org.uk/rare-disease-d…
Today is #RareDiseaseDay! 🦓 NORD is so thankful for everyone in our #RareDisease community, and we're honored every day to advocate alongside you. Tag us in your posts throughout the day as you celebrate! Learn more at this link and share it with others: bit.ly/RDD2023
This month our team gathered to show our support for families living with #RareDiseases in honor of #RareDiseaseDay. Our commitment to developing therapies for people living with #ENPP1 Deficiency & #ABCC6 Deficiency remains unwavering.
At @AAAAI_org today, our team is setting up for an interview and associate editor @GiulianaGrossi is at the camera! Follow for more coverage of the 2023 #AAAAI23 conference in San Antonio, TX! #allergy #immunology ow.ly/ypET50N353Y
Did you know that many women aren't aware they have hemophilia until they experience postpartum bleeding?
Today, during an illuminating conversation with an expert hematologist, I learned that hemophilia in women and girls is severely overlooked, much like sickle cell trait.
Today, during an illuminating conversation with an expert hematologist, I learned that hemophilia in women and girls is severely overlooked, much like sickle cell trait.
Day 1 of #AAAAI23. Will we pin 1,000 cranes for rare disease by the end of the meeting? @AAAAI_org @TakedaPharma
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