Rare Disease Advisor @RareDisease_Adv
Trusted knowledge base of practical information and resources focused on treating and diagnosing #RareDisease. rarediseaseadvisor.com New York, NY Joined March 2021-
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The Brazilian Health Regulatory Agency (ANVISA) has approved #berotralstat (#Orladeyo®) for the prophylaxis of hereditary angioedema (HAE) attacks in adults and pediatric patients aged 12 years and older. Read more: brnw.ch/21wJkNW #RareDisease @BioCrystPharma
Researchers reported a case of a patient with polyneuropathy, organomegaly, endocrinopathy, monoclonal plasma cell disorder & skin changes syndrome who was initially misdiagnosed with & mistreated for chronic inflammatory demyelinating polyneuropathy. brnw.ch/21wJkIX
There seems to be a significant link between clinical metrics and performance on the Cerebellar Cognitive Affective Syndrome Scale (CCAS-S) in patients with Friedreich ataxia, as per a study published in the journal Cerebellum. Read more: brnw.ch/21wJkA8 #RareDisease
#DNTH103 improves neurotransmission in a preclinical model of myasthenia gravis, according to results presented at the 2024 @AANmember Annual Meeting. Read more: brnw.ch/21wJklQ #RareDisease #AANAM #Neurology @dianthustx
A new study has determined that patients with hereditary transthyretin-mediated amyloidosis (hATTR) due to a V1421 mutation have transthyretin amyloid polyneuropathy (ATTR-PN) more often than has been previously reported. Read more: brnw.ch/21wJk9G #RareDisease
Concomitant cases of Parkinson disease or myasthenia gravis are rare but could cause issues related to the therapeutic management of both diseases, as per a systematic review of case reports and case series published in @clinthe Read more: brnw.ch/21wJiHd #RareDisease
The identification of subclinical hypoventilation on time is of great importance in patients with Duchenne muscular dystrophy (DMD), according to a new study published in the Journal of Neurology. Read more: brnw.ch/21wJiBT #RareDisease #MedTwitter
Despite Hamas rocket attacks and war in Gaza, Dr. Avner Reshef, a leading expert on hereditary angioedema (HAE), has persistently treated patients with the rare disease for over six months. Learn more in the latest #RareCare podcast episode and article⬇️ brnw.ch/21wJitm
In @cureusjournals, physicians presented the rare case of a patient with a challenging presentation of granulomatosis with polyangiitis—a form of antineutrophil cytoplasmic antibody (ANCA)-associated vasculitis. Read more: brnw.ch/21wJig1 #CaseReport #RareDisease
#Zilucoplan displayed sustained safety, tolerability & efficacy in acetylcholine receptor autoantibody-positive (AChR+) generalized myasthenia gravis, patients over an extended phase 3 trial period, as reported in @TANeurolDisord Read more: brnw.ch/21wJhUD #RareDisease
The @US_FDA has approved fidanacogene elaparvovec-dzkt (#Beqvez™) as a one-time gene therapy to treat adult patients with moderate-to-severe hemophilia B, according to @pfizer. Read more: brnw.ch/21wJg7K #RareDisease #MedTwitter
An investigator-initiated trial is currently recruiting patients with relapsed or refractory autoimmune diseases, including ANCA-associated vasculitis, to evaluate the safety and efficacy of anti-CD19-CD3E-CAR-T cells. Read more: brnw.ch/21wJeVx #RareDisease
People affected by chronic inflammatory demyelinating polyneuropathy need close follow-up so that the medical team can ensure they receive the most appropriate therapies or medical interventions to prevent irreversible axonal degeneration. 📚Authorea brnw.ch/21wJe2l
Cognitive impairment in patients with neuromyelitis optica spectrum disorder (#NMOSD) is correlated with physical disability but not with several other factors, according to a Mexican study published in @SclerosisAnd, Read more: brnw.ch/21wJe2i #RareDisease #MedTwitter
#WorldOrphanUSA: Clinical experts and advocates call for improving children’s access to essential health care by streamlining the Medicaid provider screening & enrollment process for out-of-state providers Read more: brnw.ch/21wJdXb #RDAatWODC @OrphanConf @RareDiseases
Researchers have unveiled a complex mechanism underlying antibody-mediated immune suppression (AMIS) and enhancement (AMIE), processes involved in hemolytic disease of the fetus and newborn (#HDFN). Read more: brnw.ch/21wJdEL #RareDisease #MaternalFetalMedicine
Regional measures of dentate nuclei substructure appear to be sensitive measures of disease duration and ataxia severity in patients with Friedreich ataxia (FA), according to a recently published study in Movement Disorders. Read more: brnw.ch/21wJdrW #RareDisease
#WorldOrphanUSA: After a 25-year journey advancing basic, translational & clinical science, the Friedreich ataxia community saw a drug approved by the @US_FDA last year to slow down the course of the disease. Read more: rarediseaseadvisor.com/reports/after-… #RDAatWODC @CureFA_org…
#WorldOrphanUSA: "These kids are not disposable." - Patricia Welton founder and CEO of @BeyondtheDx. @OrphanConf #RDAatWODC #OrphanDrugUSA #RareDisease
Alterations in the architecture of the protein synthesis machinery may be driving changes associated with translation in Huntington disease, according to a new study published in the journal Neurobiology of Disease. Read more: brnw.ch/21wJbEM #RareDisease #Neurology
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]
Abby Turnwald (she/he.. @PedsGCAbby
927 Followers 1K Following Pediatric genetic counselor, neurogenetics, advocate for the rare disease community, let’s talk about sibling health
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.
Angie B @quinky_dink
5K Followers 4K Following Chronic pain patient. Multiple autoimmune diseases. Patient Advocate. Fighting the fight of my life everyday.
Taylor Kane @rarelikeher
3K Followers 1K Following Patient engagement consultant, #raredisease advocate, author, speaker, founder @remember_girls, and more!
Patient Worthy @PatientWorthy
8K Followers 7K Following We're a resource for engaging, informative content and rare patient news, well done.
Lucy Burbury @lucyburbury
1 Followers 49 Following
Jackson @Jackson90570460
9 Followers 209 Following
Ebrima Drammeh @EbrimaEb32225
325 Followers 4K Following
melinda hughie blaloc.. @Mmhblalock
34 Followers 596 Following mama • wife • advocate • fundraiser • medical mama • Epilepsy | ASD • thyroid cancer 🫀 TAAD • 💜neuroscience, epilepsy 🧠 philanthropy / St. Jude
Gerry Nesbitt @ CLIRI.. @clirinx
460 Followers 747 Following Clinical Research IT 🇺🇸 🇨🇮 for Epilepsy & Rare Diseases ▪️Electronic data collection▪️Patient Portal▪️Genetic variant tools▪️Big Data▪️Gearóid 🇨🇮
MANZI Ndamukunze @MNdamukunze
436 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
Jason Tardio @JasonTardio
9 Followers 46 Following
Dr M.H. Khara @DrMHKhara
18 Followers 330 Following
JPA Health @JPAHealthComm
1K Followers 825 Following JPA Health specialists synchronize insights, ideas and incredible execution for results that impress. We make meaningful connections like no other agency.
Dermatology Advisor @DermAdvisor
696 Followers 49 Following https://t.co/BumMMJ4E2q offers dermatology specialists and other health care professionals a comprehensive knowledge base of practical information and resources
Dhaiwat Shukla @dhaiwat_shukla1
119 Followers 401 Following Consultant Rheumatologist@VS General Hospital French teacher @ Af d'Ahmedabad
Sharmila Kalgutkar @KsharmilaMT
5K Followers 2K Following Journalist @ TOI-महाराष्ट्र टाईम्स.UNFPA Laadali Yashaswini awardee Fellow !Formerly with Sakal Group .Investigative journo. Xavier's student Views are personal
Elsie Megan @poemsandparents
19 Followers 224 Following My cathartic and honest (poetic) ramblings about the ups and downs of parenting a child with severe learning disabilities.
Alström Syndrome UK @AS_UK
2K Followers 5K Following 'Alström Syndrome is a rare genetic condition affecting every organ in the body. ASUK provides support & guidance for those affected.' Tweets by Catherine Lewis
Nexogic @nexogichealth
298 Followers 3K Following Web App for medicos to create SEO based Digital Profiles, publish Medical Cases, Connect with Doctors and Hospitals via Patient referral tool, top Medical Jobs.
Avance Clinical @AvanceCRO
169 Followers 570 Following Avance Clinical is a full-service global CRO for Biotechs - specializing in delivering quality clinical trials, with globally accepted data
Tiffany Dages @TiffanyDages
990 Followers 4K Following Lifelong vEDS warrior🦓 7th gen Floridian 🍊mom 💐hospice volunteer🤗 Dem💙
༄✿Sana Queen♛�.. @SanaQueen_PTI
1K Followers 1K Following
DNAUnited @DNAUnited2024
0 Followers 8 Following
Marni Cartelli @Purrfectly_Rare
3K Followers 2K Following Rare Disease Patient, mom, & advocate. Danny's Dose Alliance Board Member. Know who you are & live a way you can be proud of.
n-Lorem Foundation @n_lorem
1K Followers 572 Following Discovering, developing, and providing personalized experimental ASO medicines to treat nano-rare patients — for free, for life
Dr. Sabin Adhikari @DrSabinAC5908
151 Followers 1K Following Resident Doctor, Department of Clinical Oncology @ NAMS, Bir Hospital Breaking the stereotype Jaya Desh Nepal🇳🇵
KenWarringtonMD @MdWarrington
3K Followers 963 Following Professor and Rheumatologist l Vasculitis Clinic Director @MayoClinic Rochester | Researcher #vasculitis #rheumatology #MayoClinicRheumatology #travel 🇲🇹🇺🇸
Hemophilia Free India @HemophiliaX
159 Followers 317 Following a dream.. that is.. life without pain
leen alkalbani @alkalbani_leen
11 Followers 134 Following MD; Neurology Research Fellow 🧠#Aspiringchildneurologist
Arun Senniappan @Arunsenniappan
65 Followers 657 Following
Susan Berry @BerryS730
1K Followers 1K Following Medical Genetics, especially inherited disorders of metabolism. Tweets are personal. @IBEMC1 @NBSTRN @MNAAP @Region4Genetics #tweetiatrician #NewbornScreening
Sleep-Wake Advisor @SleepWakeAdv
16 Followers 39 Following Sleep-Wake Advisor is aimed at helping clinicians with the latest research and resources to assist in the diagnosis and treatment of sleep-wake disorders.
Peer Med Zebra | Rare.. @peermedzebra
72 Followers 295 Following An initiative of @PeerMedicalFDN advocating for intersectionality in care for patients with Rare Diseases so that no one is left behind!
SmashingPompe @SmashingPompe
25 Followers 49 Following Pompe, it's in my DNA! I am Rare! Pompe Disease is Me! I am a Warrior! I am Fighting the Battle! I am Smashing Pompe! I am a Pompe Champ!
J Harp @GodADesk
210 Followers 6K Following Proverbs 1:7 KJV The fear of the Lord is the beginning of knowledge: but fools despise wisdom and instruction.
Adrienne @adriennelj
2K Followers 5K Following Being a lady is like being powerful; if you have to tell people that you are, then you aren't. ~~ Margaret Thatcher
Sam @k33pster
6 Followers 492 Following
Rachel Miller-Stainsb.. @RMillerStainsby
16 Followers 70 Following Health Information & Engagement Manager @myrovlytis 🧬 MSc Health Psychology 🧠 Patient Advocacy 🩺 She/Her | VMO
Stine Krag Brunvold @stine_krag
14 Followers 53 Following Communications Manager I Graphic Designer I NMD Pharma
teamconnor4cadasilcur.. @teamconnor4cada
39 Followers 107 Following The Connor family is dedicated to raising awareness and funds to for CADASIL research. Help us reach goal!
M Morais @MMorais1
29 Followers 539 Following
Prabhat Sinha @ThePksinha
2K Followers 1K Following Director- Public and Government Affairs - Boehringer Ingelheim India ( views personal)
Kirsten L. Schneider .. @leaferi
3K Followers 5K Following Nature at night, cars are not people, lover of law—not lawyers, human beings are inherently equal. https://t.co/E8FQenPcq6
Offthegrid🇺🇸✝.. @offthegrid01
271 Followers 1K Following
Real Mr Jimmy @jalkhayer
25K Followers 3K Following Management Consultant and Book Author 🛩 Doctorate under development US.FL {ما شاء الله} اعتقد اني ضايع ما بين السعودية 🇸🇦 و البحرين 🇧🇭
Mohammad Sukkar @msukkar2014
91 Followers 175 Following
Biotechvest @razals
11 Followers 101 Following
zelalem Geletu, MD @GeletuZelalem
50 Followers 346 Following
Jack Hall @Hall8Jack
1K Followers 3K Following Biotech, Commodities, etc. A like is not necessarily a like.
Shematologist, MD @acweyand
84K Followers 6K Following Peds hematologist oncologist,mother, dreamer, pacifist, justice seeker.Clueless Mother https://t.co/HblMm3s8OM Cream Influencer #CakeForBreakfast https://t.co/4SLDqCFsMq
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 🦓
NORD @RareDiseases
41K Followers 3K Following National Organization for Rare Disorders (#NORD) is the voice of the #RareDisease community. Official US sponsor of #RareDiseaseDay.
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]
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
Abby Turnwald (she/he.. @PedsGCAbby
927 Followers 1K Following Pediatric genetic counselor, neurogenetics, advocate for the rare disease community, let’s talk about sibling health
Global Genes @GlobalGenes
30K Followers 6K Following Empowering the Next Generation Rare Disease Advocate Merged with RARE-X Dec. 2022. #CareAboutRare
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.
Angie B @quinky_dink
5K Followers 4K Following Chronic pain patient. Multiple autoimmune diseases. Patient Advocate. Fighting the fight of my life everyday.
Taylor Kane @rarelikeher
3K Followers 1K Following Patient engagement consultant, #raredisease advocate, author, speaker, founder @remember_girls, and more!
ASH @ASH_hematology
69K Followers 2K Following The American Society of Hematology (ASH) is the world's largest professional society concerned with the causes and treatments of blood disorders.
Patient Worthy @PatientWorthy
8K Followers 7K Following We're a resource for engaging, informative content and rare patient news, well done.
Laurent ARNAUD @Lupusreference
17K Followers 596 Following MD, PhD | Professor of #Rheumatology | #Lupus #SLE | #Digitalhealth | President of @SLEuroSociety | @ERN_ReCONNET Disease Coordinator for Lupus & RP 🇪🇺
Rare STRIDES® @RareStrides
54 Followers 102 Following Rare STRIDES® is an innovative technology company focused on improving quality of life for patients. Our Mission: Connecting Care to Those with Rare.
Jason Tardio @JasonTardio
9 Followers 46 Following
JPA Health @JPAHealthComm
1K Followers 825 Following JPA Health specialists synchronize insights, ideas and incredible execution for results that impress. We make meaningful connections like no other agency.
Dermatology Advisor @DermAdvisor
696 Followers 49 Following https://t.co/BumMMJ4E2q offers dermatology specialists and other health care professionals a comprehensive knowledge base of practical information and resources
Siblings with a Missi.. @SibsMission
91 Followers 102 Following Siblings with a Mission supports siblings and families of individuals with complex health conditions through the power of stories and conversation.
Maria Mouyis @MariaMouyis
249 Followers 325 Following General Physician and Rheumatologist with a Specialist Interest in Maternal Medicine.
IFOPA @ifopa
849 Followers 782 Following Fund research to find a cure for FOP while supporting individuals and families through education, public awareness and advocacy.
DISORDER: The Rare Di.. @DisorderRare
949 Followers 217 Following Two rare disease dads took their films for Menkes Disease and USP7 & built them into a festival for all films on rare disease. https://t.co/Qjsl21HRH4
ASBPE @ASBPE
2K Followers 1K Following ASBPE is the professional association for #editors, #writers, #freelancers, art directors & #designers employed in the #B2B press.
Ana Mingorance @CNSdrughunter
3K Followers 1K Following 🧠 🧬 Neuroscientist. Looking for new medicines for CDKL5, SCN1A, SHANK3, DHPS and a few others.
Liz Kennerley (she/he.. @LifeAccrdingLiz
1K Followers 2K Following #chronicillness #patient and Federal Health Policy #Advocate @SimmonsAlumni, @remember_girls Board member. Views my own.
Alström Syndrome UK @AS_UK
2K Followers 5K Following 'Alström Syndrome is a rare genetic condition affecting every organ in the body. ASUK provides support & guidance for those affected.' Tweets by Catherine Lewis
The Ehlers-Danlos Soc.. @TheEDSociety
24K Followers 6K Following The global nonprofit organization dedicated to change and progress in the world of Ehlers-Danlos syndromes (EDS) and hypermobility spectrum disorders (HSD).
Parexel @Parexel
13K Followers 1K Following Improving the lives of patients every day #withheart. #biotech #healthcare #lifesciences #innovation
n-Lorem Foundation @n_lorem
1K Followers 572 Following Discovering, developing, and providing personalized experimental ASO medicines to treat nano-rare patients — for free, for life
Uplifting Athletes @UpliftingAth
5K Followers 3K Following Nonprofit organization harnessing the power of sport to build a community that invests in the lives of people impacted by rare diseases.
Marni Cartelli @Purrfectly_Rare
3K Followers 2K Following Rare Disease Patient, mom, & advocate. Danny's Dose Alliance Board Member. Know who you are & live a way you can be proud of.
Dr. Nara Michaelson M.. @narologist
1K Followers 1K Following MS/Neuroimmunology Fellow @MGH @BWH 👩🏻⚕️Sylvia Lawry/ABY Fellow 🧡 Cornell Neuro @WCMCNeurology 🧠 MIT Alum @MIT👩🏻🎓Dartmouth MD 💊#NeurologyRF Editor 📚
Rare Epilepsy Network @RareEpilepsy
2K Followers 563 Following Rare Epilepsy Network (REN) is working with urgency to collaboratively improve outcomes of rare epilepsy patients & families via research and advocacy.
Leah Acosta, MD, MPH,.. @acostalmd
943 Followers 409 Following @VUMedicine @VUMCNeuro Inspirations: behavioral neurology via clinical education & humanistic physicians via narrative medicine @greenjournal & art Posts=mine
Liver International @LiverInt
7K Followers 255 Following We publish high-quality original research in Hepatology / Co-EICs @lucavalenti75 @AghemoAlessio @Alexfornergon, #SoMe Ed @jturnesv / IF=6.7 / RT ≠ endorsment
Matthew Armstrong �.. @Drfattyliver
2K Followers 124 Following Liver/Transplant medic, married, father to awesome twins
The Liver Dude @MichaelHenegha8
3K Followers 370 Following Professor of Hepatology. Consultant Hepatologist. Specialist in Autoimmune Liver Disease and Liver Transplantation. King's College Hospital, London, UK.
PBC Foundation @PBCFoundation
3K Followers 2K Following We provide support for life to those affected by autoimmune liver disease Primary Biliary Cholangitis,(formerly Primary Biliary Cirrhosis) #thebiggerpicture
@raregivers @raregivers
238 Followers 86 Following Raregivers sources research, relief and inspiration for families living with rare diseases.
Erynn Johnson @johnson_erynn
28 Followers 81 Following Biochemist and metabolism junkie | skeletal muscle and rare neuromuscular disease biology | passionate about metabolism for lifestyle optimization
Engage Health Inc. @EngageHealthInc
85 Followers 182 Following Engage Health is an experienced health research firm serving the pharmaceutical, biotechnology and medical device sectors.
Michael A. Lopez, MD,.. @lopezamike
1K Followers 2K Following Pediatric neuromuscular / Muscle disease researcher. #ChildNeurology #Advocacy. Views my own. He/Him, 🏳️🌈, #BLM
Kyle Bryant @KyleABryant
2K Followers 3K Following @TheAtaxian, Speaker, Director of @rideATAXIA for @CureFA_org. Author of #ShiftingIntoHighGear. Co-host @2DDPodcast. #CureFA #RareDisease
Christina Megli @ChristinaMegli
768 Followers 527 Following she/hers, Obstetrician, MFM, repro ID, physician scientist, mom, loves dogs and the placenta, tweets are my own
Method Tuuli, MD, MPH.. @MethodTuuli
1K Followers 853 Following MFM Physician-Scientist-Executive; Professor and OBGYN Dept Chair, @Brown University; Chief of OBGYN@Women&Infants Hospital **views expressed here are my own**
Ai-ris Yonekura Colli.. @airisyc
433 Followers 574 Following Maternal Fetal Medicine @BidmcMFM, Translational Immunology @BIDMC_CVVRTrial, Sister 👩🏻🏫🙋🏻♀️👩🏻🎤, Mommy 👧🏻👧🏻👧🏻, Educator, Advocate, Ai=love
Cynthia Gyamfi, MD, M.. @drcynthiagyamfi
4K Followers 2K Following Samuel SC Yen Endowed Chair and Professor of OBGYN & Repro Sci at UC San Diego in La Jolla Tweets=mine
Dr. Erika Werner @ErikaWernerMD
1K Followers 216 Following Chair of Obstetrics and Gynecology at Tufts MFM/mom who loves clinical research & seeking creative ways to improve health and limit costs. Opinions are my own.
Anushka Chelliah MD @anushkachelliah
438 Followers 399 Following Maternal Fetal Medicine Specialist, Obstetrician and Gynecologist. Mom and Wife. #womeninmedicine
Moti Gulersen @GulersenMoti
675 Followers 379 Following MFM at Jefferson. Trained by Northwell Health. Opinions are my own.
Blair J. Wylie @blairwylie
1K Followers 1K Following Founding Director, The Collaborative for Women's Environmental Health @ColubiaOBGYN MFM doc, repro env health epidemiologist & global maternal health rsrcher
Tony Shanks @alshanks
1K Followers 2K Following MFM by day. Fan of sports, food and music at night. Opinions my own.
Minhaz Sarker, MD @MinhazSarkerMD
84 Followers 100 Following MFM Fellow Physician @UCSD_ObGyn • Former Resident Physician @IcahnMountSinai • Photographer at Minhaz Sarker Photography • My Tweets/My Thoughts • He/Him
Omar Young MD (he/him.. @DocOhms
2K Followers 2K Following Vice-Chair of Education @UNCOBGYN /APD @UncOBGynRes. Co-creator of @BroDocTalk1. Political junkie| Tweets are my own. #GetVaccinated #blacklivesmatter #tennis
Ebony B. Carter, MD, .. @ebonycartermd
2K Followers 579 Following high-risk OB, mother of 3, wife, physician-scientist, AE Equity @ OB/GYN, promoter of reproductive health equity, tickler of ebony & ivory keys, all tweets mine
Mike Pishvaian @MPishvaian
5K Followers 1K Following GI Onc at Hopkins, focusing on #PancreaticCancer, #PrecisionMedicine. Striving to improve patient outcomes. Originator of #TumorBoardTuesday @TumorBoardTues
Benjamin Weinberg, MD.. @benweinbergmd
2K Followers 812 Following GI Medical Oncologist, Associate Professor of Medicine @Georgetown @RueschCenter @LombardiCancer, Ex-@SidwellFriends @Penn @KeckMedUSC keyboardist @BasementRiot
Dr. jordan berlin @jordanberlin5
2K Followers 358 Following Oncologist at Vanderbilt University Medical Center focusing on gastrointestinal cancers and early phase drug development. I like dogs. Views are my own.
Blue Faery @BlueFaeryLiver
2K Followers 2K Following Blue Faery's mission is to prevent, treat and cure primary #livercancer, specifically Hepatocellular Carcinoma, through #research, #education and #advocacy.
Caroline H. Hopkins @Ch_Hops
1K Followers 3K Following Health journalist • Reporter @PrecOncNews • Contributor @NBCNews • Stories in @nytimes @voxdotcom @NatGeo • @ColumbiaJourn alum • [email protected]
Dr Amol Akhade @SuyogCancer
3K Followers 121 Following Oncology care , to give or not to give chemo , data analysis , oncology pharamocovigilance , enthusiastic critic, twitts are not twists
Valuable insights from Ovid's COO, Jason Tardio, shared during a panel at this year's @OrphanConf. #WorldOrphanUSA
A major challenge that drug developers face in the development of rare cancer medicines is the accessibility of biomarker testing. Jim Palma of @targetcancer emphasizes that education is key not just for patients but also providers. #WorldOrphanUSA
Closing this morning's sessions at #WorldOrphanUSA discussing "Challenge and Opportunity with the Current Regulatory and Legislative Landscape in the US." The EveryLife's VP of Policy Jamie Sullivan, was joined by Rachel Smith, Mark Trusheim, Kinnari Patel and Peter Marks.
Hana and Patricia Weltin from Beyond the Diagnosis unveil their latest masterpiece featuring a portrait of Yiannis at #WorldOrphanUSA
Looking for support as you navigate #myastheniagravis as a young adult? MAYA offers engagement opportunities, events, and discussions to help you live a healthy and positive lifestyle with MG. Learn more here: - ow.ly/T2Il50Riz7u #MGStrong #RareDisease #MGWarriors
“I enjoy being a #PH Support Group Leader because it’s a way to give back to my community,” says Evelyn Mitchell. “Bringing our group together helps us all with the opportunity to share and learn together.” #volunteerweek
Our first sloth arrived for Emmalee 🥰 She is so excited! #FindYourTribe #PompeDisease #NewbornScreening #RareDisease
The EveryLife's Jamie Sullivan and Julie Breneiser from @gorlin_alliance discuss "Patient-first innovation: FDA shifting rare diseases endpoints" #WorldOrphanUSA
Our Chief Operating Officer, Sarah Glass, is the chair of of today's World Orphan Drug Congress USA 2024 session and kicked off the event with Opening Remarks!
Diagnosed with #SMA at just one year old, #MDA National Ambassador Ira Walker’s journey showcases resilience and determination. With promising new treatments, he’s on a mission to unite the #neuromuscular community and inspire change. Read his story in @RareDisease_Adv.
Spinal muscular atrophy #PatientAdvocate Ira Walker Selected as @MDAorg’s 2024 ‘National Ambassador’ 💡Discover more about Ira and his hopes for the future of SMA care: brnw.ch/21wISwg #MDAConference #MDAorg #RDAatMDA #neurology
Here is a roundup of our most recent #hemophilia-related articles: ❗ SDOHs May Affect Medication Adherence Among Patients With Hemophilia brnw.ch... ❗ Structural Variant Disrupting F8 Gene Identified in Families With Severe Hemophilia A…
Join us in raising awareness for #WorldHemophiliaDay with @RareDiseaseAdvisor. We stand united in support of those affected by #hemophilia and those who care for them.
Happy #WorldHaemophiliaDay from all the team at the Irish Haemophilia Society! Today, we are delighted to launch our new Introduction to Haemophilia booklet, which you can view online at this link: haemophilia.ie/wp-content/upl…
We applaud this important research to help advance the understanding of Friedreich ataxia and the progression of the condition in children.
The Friedreich Ataxia Rating Scale balance and gait subscore, also called the upright stability score, may be a better outcome measure in pediatric patients with Friedreich ataxia. Annals of Clinical and Translational Neurology Read more: brnw.ch/21wIJDJ #RareDisease
Tomorrow is International Pompe Disease Day, April 15th! I have a Passion for spreading awareness about Pompe.Check out @MDAorg video! #Smashingpompe #mdaambassador #TikTok tiktok.com/t/ZTLyWsC35/
FDA and 15 private organizations have joined forces to launch the Bespoke Gene Therapy Consortium. This Consortium will improve the landscape for AAV gene therapy development for extremely rare diseases on multiple fronts. (1/2) nih.gov/news-events/ne…
MGFA volunteer Tom Bartlett turns the challenges of living with #myastheniagravis into laughter-filled lessons. From DIY disasters to everyday hurdles, his story teaches us the strength found in humor. Read the full article from @RareDisease_Adv: ow.ly/vf6850R2mN7
#MDA once again hosted the annual gathering of the Neuromuscular Advocacy Collaborative at the #MDAconference with MDA’s Vice President of Public Policy & Advocacy, @PMelmeyer. The meeting brought together leaders from 20+ neuromuscular advocacy organizations. @MDA_Advocacy
Treatment with #givinostat for ambulatory boys with #DMD led to noticeable improvements at 18 months, according to a poster from #MDA2024. Get the details from researchers who presented at @MDAorg 👇 bit.ly/3wFPneR @RareDisease_Adv
The 2024 #MDAconference is well underway in #Orlando! We kicked off the conference with opening remarks from MDA’s President and CEO, Donald S. Wood, PhD, followed by a Keynote Address from #ALS patient and advocate, @limpbroozkit.
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