https://rddc.tsinghua-gd.org/
RDDC Rare Disease Database | Facilitating research on genetic diseases, pathogenic genes and...
RDDC, Rare Disease Data Center, is dedicated to offering a comprehensive platform that covers genetics, diseases, and animal models, with the ultimate goal of...
rare diseasegenetic diseasesdatabaseresearchpathogenic
https://www.ibtimes.co.uk/jfk-john-f-kennedy-autoimmune-disease-aps-523880
JFK's Robust Public Image Hid Truth of Rare Disease | IBTimes UK
Nov 20, 2013 - Healthy, vigorous image of John F Kennedy did not match reality, say doctors on eve of assassination anniversary.
public imagerare diseasejfkrobusthid
https://975thefanatic.com/episodes/february-29-is-rare-disease-day-2/
February 29 is Rare Disease Day
Romy Braunstein, a Rare Disease patient, advocate and a founder of GettingUp.org discusses the over 10,000 rare diseases that impact over 35 million worldwide....
rare disease dayfebruary
https://www.onescdvoice.com/news-meeting/support-rare-disease-patients-impacted-covid-19-nord-launches-premium-limited-medical-relief-program/
In Support of Rare Disease Patients Impacted by COVID-19, NORD Launches Premium and Limited Medical...
get connected to the best sources of Sickle Cell Disease information and support online, all for free.
in support ofrare diseaseimpacted bypatientscovid
https://www.ispor.org/heor-resources/news-top/heor-news-desk/icer-examines-possible-policies-aimed-at-sustaining-future-rare-disease-drug-development
ISPOR - ICER Examines Possible Policies Aimed at Sustaining Future Rare Disease Drug Development
rare diseasedrug developmentisporicerexamines
https://www.spiritualsatanist.com/work-opportunities/job/remote-pah-rare-disease-account-strategist-at-merck-indianapolis-in-SVF6Z2I3aDc5bEtmcEUxMndlcW5Bd3k5dXc9PQ==
Remote PAH Rare-Disease Account Strategist job at Merck Indianapolis, IN, US -...
Remote PAH Rare-Disease Account Strategist job at Merck Indianapolis, IN, US ...insights. This position requires a mix of sales experience in healthcare and...
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https://elixir.mf.uni-lj.si/course/section.php?id=959
Section: Topic 2 | FAIR Rare Disease Registries 2022 | ELIXIR-SI
rare diseasesectiontopicfairregistries
https://www.researchprotocols.org/2024/1/e53362/citations
JMIR Research Protocols - Needs and Experiences With Health Care Providers of Adult Rare Disease...
Background: Rare diseases in Europe are defined as diseases with a prevalence of less than 5 per 10,000 people. Despite their individual rarity, the total...
jmir research protocolshealth care providersrare diseaseneedsexperiences
https://clarivate.com/life-sciences-healthcare/report/nrlfim0002-2018-biopharma-idiopathic-pulmonary-fibrosis-niche-rare-disease-landscape-forecast-us-eu5-2018/
Idiopathic Pulmonary Fibrosis | Niche & Rare Disease Landscape & Forecast | US/EU5 | 2018 |...
Idiopathic pulmonary fibrosis (IPF), although a rare disease, is one of the most common interstitial lung diseases (ILDs). It is characterized by an...
idiopathic pulmonary fibrosisrare diseasenichelandscapeforecast
https://researchfestival.nih.gov/2024/posters/identification-rare-disease-patients-ehr-systems-and-their-covid-19
Identification of Rare Disease Patients in EHR Systems and their COVID-19 Outcomes: A Retrospective...
rare diseaseehr systemsidentificationpatientscovid
https://acrpnet.org/2025/02/25/using-bayesian-statistics-to-support-rare-disease-research-innovations
Using Bayesian Statistics to Support Rare Disease Research Innovations - ACRP
Feb 25, 2025 - Randomized controlled clinical trials are considered the gold standard for understanding treatment safety and efficacy. Rare disease trials struggle to recruit...
rare disease researchbayesian statisticsusingsupportinnovations
https://www.distilnfo.com/lifesciences/2023/01/14/ipsen-to-acquire-albireo-for-rare-disease-portfolio/
Ipsen to Acquire Albireo for Rare Disease Portfolio - DistilINFO LifeSciences
Jan 14, 2023 - French biopharma Ipsen has agreed to acquire Boston-based Albireo Pharma for $42 per share, plus a contingent value right of $10 per share, the two...
rare diseaseipsenacquirealbireoportfolio
https://c-path.org/register-now-ai-powered-real-world-simulations-for-faster-and-value-based-rare-disease-drug-development/
View Now | AI-powered Real-world Simulations for Faster and Value-based Rare Disease Drug...
Jun 27, 2024 - On March 16 Quinten Health CEO Billy Amzal, PhD, MBA, presented AI-powered Real-world Simulations for Faster and Value-based Rare Disease Drug Development....
view nowai poweredreal worldvalue basedrare disease
https://ega-archive.org/dacs/EGAC00001000566
DAC for Rare Disease Studies from the Broad Institute - EGA European Genome-Phenome Archive
DAC for Rare Disease Studies from the Broad Institute
rare diseasefrom thedacstudiesbroad
https://www.ncfinternational.it/gsk-to-transfer-its-portfolio-of-rare-disease-gene-therapies/
Gsk to transfer its portfolio of rare disease gene therapies - NCF International
Jun 12, 2018 - The strategic agreement between Gsk (https://www.gsk.com/) and Orchard Therapeutics (http://orchard-tx.com/) includes approved and investigational rare disease...
rare diseasegene therapiesgsktransferportfolio
https://www.pharmavoice.com/news/sharon-king-rare-disease-Aldevron/624962/
After losing her daughter to a rare disease, Sharon King is looking to rewrite the playbook of drug...
The communications pro is lending her community-building, storytelling expertise to the Bespoke Gene Therapy Consortium.
rare diseasesharon kingthe playbooklosingdaughter
https://www.progeriaresearch.org/fr/2025/05/27/rdd-2025-cimus/
Rare Disease Day 2025 I The Progeria Research Foundation
Aug 7, 2025 - The Center for Research in Molecular Medicine and Chronic Diseases (CiMUS) at the University of Santiago in Spain invited PRF co-founders to share their story...
rare disease dayresearch foundationprogeria
https://www.good.is/tag/rare-disease/
rare disease Archives - Good.is
rare diseasearchivesgood
https://www.biopharmadive.com/news/sanofi-venglustat-study-stopped-kidney-disease/601045/
Sanofi rare disease drug hits another setback | BioPharma Dive
The French pharma halted a late-stage trial of venglustat, one of its priority drug candidates, in a type of kidney disease, months after stopping testing in...
rare diseasebiopharma divesanofidrughits
https://oncodaily.com/blog/195430
Naveen Pemmaraju: ASH Hematology Session on Patient Care, Research and Education on Rare Disease -...
Dec 2, 2024 - Naveen Pemmaraju: ASH Hematology Session on Patient Care, Research and Education on Rare Disease / Anderson Cancer Center, ASH, ASH Hematology Session, BPDCN,
research and educationpatient carerare diseasenaveenash
https://www.khsu.org/regional-interests/2019-12-10/concord-woman-with-rare-disease-granted-reprieve-from-deportation
Concord Woman With Rare Disease Granted Reprieve From Deportation | KHSU
Aug 14, 2021 - Trump administration reverses policy and grants two-year protection to Guatemalan-born Maria Isabel Bueso, whose life depends on weekly medical treatments at...
rare diseaseconcordwomangrantedreprieve
https://www.fox9.com/news/pro-athlete-girl-fighting-rare-disease-are-best-friends
Pro athlete, girl fighting rare disease are best friends | FOX 9 Minneapolis-St. Paul
David "The Beast" Douglas is a professional weightlifter, Marine Corps veteran and motivational speaker, but - by his own admission - his strength is nothing...
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https://experts.umn.edu/en/organisations/neurodevelopmental-program-in-rare-disease/
Neurodevelopmental Program in Rare Disease - Experts@Minnesota
rare diseaseneurodevelopmentalprogramexpertsminnesota
https://themighty.com/topic/rare-disease/covid-19-pandemic-rare-disease-supporting-others/
On Rare Disease and Getting Through the COVID-19 Pandemic Together
Courtney R. writes about what the rare disease community can teach others about the COVID-19 pandemic.
rare diseasegettingcovidpandemictogether
https://pharma.economictimes.indiatimes.com/tag/rare+disease+treatment
Rare disease treatment - Latest rare disease treatment , Information & Updates - Pharma -ETPharma
ETPharma.com brings latest rare disease treatment news, views and updates from all top sources for the Indian Pharma industry.
rare diseaselatest informationtreatmentupdatespharma
https://medcraveonline.com/JPNC/median-cervical-cleft-a-rare-disease-case-report-in-a-newborn.html
Median cervical cleft: A rare disease: Case report in a newborn - MedCrave online
rare diseasecase reportmediancervicalcleft
https://www.illumina.com/areas-of-interest/genetic-disease/rare-disease-genomics/rare-disease-wgs.html
Rare Disease Whole-Genome Sequencing
Whole-genome sequencing is the most comprehensive test for rare disease, with the potential for superior diagnostics and outcomes.
whole genome sequencingrare disease
https://www.advarra.com/blog/tag/rare-disease-research/
rare disease research Archives | Advarra
rare disease researcharchives
https://healthcare-digital.com/technology-and-ai/rare-disease-day-bruce-bloom-of-healx-on-technology-ai
Rare Disease Day: Bruce Bloom of Healx on technology & AI | Healthcare Digital
rare disease dayon technologyai healthcarebrucebloom
https://www.hrb.ie/news-stories/hrb-advancing-rare-disease-research/
HRB - Advancing rare disease research | HRB | Health Research Board
Apr 16, 2025 - To celebrate International Rare Disease Day, the Health Research Board (HRB) highlights its support and investment for Rare Disease research. This includes...
rare disease researchhrbadvancinghealthboard
https://cdn.jwplayer.com/previews/5u1x4sQp-Oib7I9Ot
Cure Hunters #7: Hunting Rare Disease
rare diseasecurehuntershunting
https://pharmasource.global/content/news/cdmo-news/andelyn-biosciences-launches-the-andelyn-foundation-to-support-rare-disease-families/
Andelyn Biosciences Launches The Andelyn Foundation to Support Rare Disease Families - PharmaSource
Mar 11, 2026 - Andelyn Biosciences has announced the launch of The Andelyn Foundation, a nonprofit organization created to support families living with rare and ultra-rare...
andelyn biosciencesthe foundationsupport rarelaunchesdisease
https://digitalcommons.library.uab.edu/all-news/8908/
"Innovative technology applied to rare disease research focus of annual" by Alicia Rohan
By Alicia Rohan, Published on 02/18/20
rare disease researchinnovative technologyappliedfocusannual
https://www.taiwannews.com.tw/topic/rare%20disease
rare disease related news | Taiwan News - Voice of the People, Bridge to the World
Taiwan News is the most widely visited English-language portal for news about Taiwan, offering the outside world a revealing look at all things Taiwan
rare diseaserelated newsthe peopletaiwanvoice
https://www.iomcworld.com/proceedings/caenorhabditis-elegans-as-a-rare-disease-characterization-model-59568.html
Caenorhabditis elegans as a rare disease characterization model | 59568
International Online Medical Council journals provide an efficacious channel for researchers, students, and faculty members to publish works while maintaining...
caenorhabditis elegansrare diseasecharacterizationmodel
https://health.ucdavis.edu/vprp/news/headlines/uc-davis-health-designated-as-a-nord-rare-disease-center-of-excellence/2025/12
UC Davis Health designated as a NORD Rare Disease Center of Excellence
UC Davis Health is now designated as a NORD Rare Disease Center of Excellence for its leadership in providing research and expert care for people with rare...
uc davis healthcenter of excellencerare diseasedesignatednord
https://www.pacb.com/blog/solving-rare-disease-with-smrt-sequencing/
The Pathologist: Solving Rare Disease with SMRT Sequencing - PacBio
May 11, 2020 - An article by PacBio's Luke Hickey offers a look at how various types of sequencing have been important for solving rare diseases, including an overview of the...
rare diseasepathologistsolvingsmrtsequencing
https://www.oaepublish.com/rdodj/search
Search For Articles | Rare Disease and Orphan Drugs Journal - OAE
Search for keywordstitleauthor nameDOI to find the article of Rare Disease and Orphan Drugs Journal you want.
search for articlesrare diseaseorphan drugsjournaloae
https://castbox.fm/channel/Rare-Disease-Discussions-id3466013?country=us
Rare Disease Discussions | Listen Free on Castbox.
News and clinical perspective including CME programs focused on rare diseases. CheckRare focuses on rare and neglected diseases.Millions of podcasts for...
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https://flexcareers.com/jobs/444554650-rare-disease-manager
Rare Disease Manager at CSL - FlexCareers
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https://pharmaphorum.com/digital/combatting-longstanding-challenges-rare-disease-detection-innovative-deep-learning-models
Combatting longstanding challenges in rare disease detection with innovative deep learning models |...
deep learning modelsrare diseasechallengesdetectioninnovative
https://newyork.news12.com/two-brothers-one-rare-disease-and-a-race-against-time
News 12 | New York | Two brothers, one rare disease and a race against time
Anthony Spina has been battling a rare genetic disease since childhood. His younger brother, Nicholas, is facing the same crisis.
race against timenew yorktwo brothersrare diseasenews
https://www.ktnv.com/news/national/connecticut-mom-becomes-voice-for-families-affected-by-rare-genetic-disease
Mom becomes voice for families affected by rare disease
Dec 29, 2020 - The rare genetic condition is SMA, or Spinal Muscular Atrophy, which is the leading genetic cause of death in infants and toddlers.
for familiesrare diseasemombecomesvoice
https://www.insightaceanalytic.com/request-sample/3563
Request Sample - AAV Vector Gene Therapy for Rare Disease Market Size, Scope and Trends 2026 to 2035
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https://www.medicaldesignsourcing.com/tags/rare-disease/
rare disease Archives - Medical Design Sourcing
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https://irdirc.org/the-economist-suffering-in-silence-assessing-rare-disease-awareness-and-management-in-asia-pacific/
The Economist: SUFFERING IN SILENCE: ASSESSING RARE DISEASE AWARENESS AND MANAGEMENT IN...
rare disease awarenessthe economistin silencesufferingassessing
https://www.businessstudies.org/Course/Details/1152
"Certified: Rare Disease Prevention Strategies Course" (59 characters)
Rare Disease Prevention Strategies Certificate Program: Gain expertise in identifying and preventing rare diseases. Actionable insights, expert instructors,...
rare diseaseprevention strategiescertifiedcoursecharacters
https://ijisrt.com/role-of-gene-editing-in-rare-disease-management-emerging-therapeutic-paradigms
Role of gene editing in rare disease management emerging therapeutic paradigms| International...
role ofgene editingrare diseasemanagementemerging
https://www.chemdiv.com/company/media/pharma-news/2024/fda-amplifies/
FDA Amplifies Cross-Center Collaboration with the Creation of the Rare Disease...
FDA Amplifies Cross-Center Collaboration with the Creation of the Rare Disease Innovation Hub
the creationrare diseasefdaamplifiescross
https://skyroam.id/rayakan-world-rare-disease-day-2024-dash-sports-hadirkan-track-tribe-showdown/
Rayakan World Rare Disease Day 2024, Dash Sports Hadirkan "Track Tribe Showdown" - SkyRoam
Mar 2, 2024 - LigaNusantara.com - Dash Sports hadirkan ajang kompetisi lari di lintasan yang diselenggarakan di tiga kota, yaitu Jakarta, Bandung dan Yogyakarta dalam
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https://www.kkh.com.sg/giving/rare-disease-fund
Rare Disease Fund | KK Women's and Children's Hospital
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https://business.optum.com/en/insights.insights.real-world-data-fills-rare-disease-data-gaps.html
White Paper: Real-World Data Fills Rare Disease Data Gaps
Read how this creates an opportunity for researchers and product developers to fill the data divide to develop effective treatment options.
real world datawhite paperrare diseasefillsgaps
https://eurobloodnet.eu/news/163/european-rare-disease-research-coordination-and-support-action-erica-project-has-been-evaluated-positively-with-the-involvement-of-the-24-erns-for-building-on-the-strength-of-the-individual-erns-and-create-a-platform-that-integrates-research-and-inno
European Rare dIsease research Coordination and support Action (ERICA) Project, has been evaluated...
rare disease researchand supporterica projecthas beeneuropean
https://healthtree.org/mds/community/articles/rare-disease-day-2023-mds
Rare Disease Day 2023 - HealthTree for Myelodysplastic Syndromes
There are 300 million people in the world living with a rare disease. February 8, 2023 is #RareDiseaseDay.
rare disease daymyelodysplastic syndromes
https://researchportalplus.anu.edu.au/en/publications/time-to-diagnosis-for-a-rare-disease-managing-medical-uncertainty/
Time to diagnosis for a rare disease: managing medical uncertainty: A qualitative study - The...
time torare diseasequalitative studydiagnosismanaging
https://in.marketscreener.com/news/soleno-withdraws-eu-rare-disease-drug-application-ahead-of-neurocrine-takeover-ce7e51d3d08ef722
Soleno withdraws EU rare disease drug application ahead of Neurocrine takeover | MarketScreener...
Apr 7, 2026 - Soleno Therapeutics said on Tuesday it has voluntarily withdrawn the European marketing application for its rare metabolic disorder drug following acquirer...
rare diseasesolenowithdrawseudrug
https://journalforclinicalstudies.com/jj-bows-out-of-horizon-bid-as-amgen-and-sanofi-eye-cash-plays-for-rare-disease-drugmaker/
J&J bows out of Horizon bid as Amgen and Sanofi eye cash plays for rare disease drugmaker - Journal...
Dec 7, 2022 - The battle to absorb rare disease drugmaker Horizon Therapeutics will be waged with cash.
out ofrare diseasejbowshorizon
https://biopharmaboardroom.com/news/22/3182/odylia-therapeutics-marks-rare-disease-day-with-focus-on-funding-collaboration.html
Odylia Therapeutics Marks Rare Disease Day with Focus on Funding & Collaboration
Odylia Therapeutics Hosts Rare Disease Day Event to Drive Innovation in Funding and Collaboration
rare disease dayfocus onfunding collaborationtherapeuticsmarks
https://www.birmingham.ac.uk/research/centres-institutes/rare-disease-studies/lifearc-centre
LifeArc Centre for Acceleration of Rare Disease Trials - University of Birmingham
The LifeARC consortium aim to boost the capacity and efficiency of rare disease trials across the UK.
rare diseasecentreaccelerationtrialsuniversity
https://www.massbio.org/news/recent-news/disorder-the-rare-disease-film-festival-expands-festival/
DISORDER: THE RARE DISEASE FILM FESTIVAL EXPANDS FESTIVAL - MassBio
Mar 19, 2020 - Disorder: The Rare Disease Film Festival (Disorder: RDFF) is a first of its kind event showcasing a myriad of films from around the world that address the...
rare diseasefilm festivaldisorderexpandsmassbio
https://briefglance.com/articles/ultragenyx-bets-on-rare-disease-innovation-amidst-competitive-landscape
Ultragenyx Bets on Rare Disease Innovation Amidst Competitive Landscape - BriefGlance.com
Jan 11, 2026 - Biopharmaceutical firm Ultragenyx prepares to report Q3 earnings, highlighting its focus on novel therapies for rare genetic diseases. We examine the co...
rare diseasecompetitive landscapebetsinnovationamidst
https://www.expresspharma.in/fda-approves-first-therapy-for-children-with-debilitating-and-disfiguring-rare-disease/
FDA Approves First Therapy for Children with Debilitating and Disfiguring Rare Disease - Express...
Apr 16, 2020 - The FDA granted approval of Koselugo to AstraZeneca Pharmaceuticals
therapy for childrenrare diseasefdaapprovesfirst
https://www.certara.com/video/how-biosimulation-is-transforming-rare-disease-drug-development/
How Biosimulation is Transforming Rare Disease Drug Development | Certara
rare diseasedrug developmenttransforming
https://healthprofessionalradio.com.au/biopharma-global/
Biopharma Global and Rare Disease Research Approvals
Jul 14, 2017 - One of the leading health radio in Australia, United Kingdom,USA, and Southeast Asia. Health Radio which promotes health awareness to Health Professionals and...
rare disease researchbiopharmaglobalapprovals
https://icts.wustl.edu/icts-supports-rare-disease-research-and-awareness/
ICTS Supports Rare Disease Research and Awareness | Clinical & Translational Sciences | Washington...
rare disease researchictssupportsawarenessclinical
https://www.anews.com.tr/health/2025/02/28/rare-disease-day-2025-raising-awareness-for-400-million-affected-globally
Rare Disease Day 2025: Raising awareness for 400 Million affected globally
Approximately 7,000 rare diseases affect 400 million people worldwide, with 75% of those impacted being children. Rare Disease Day, observed on February...
rare disease dayraising awarenessmillionaffectedglobally
https://theshortcoat.com/tag/rare-disease-diagnosis/
rare disease diagnosis Archives | The Short Coat
rare diseasediagnosisarchivesshortcoat
https://newsletter.genevahealthfiles.com/replug-balancing-health-rights-and/
[Replug] Balancing Health Rights and Resource Constraints: The Dilemma of Rare Disease Treatment in...
Feb 17, 2026 - REPLUG Newsletter Edition #260 [The Files In-Depth]
health rightsthe dilemmarare diseasereplugbalancing
https://www.ksl.com/article/19112429
Family prepares for next step in girls' recovery from rare disease | KSL.com
Just before Christmas, KSL News aired a \nreport on two Utah sisters who were trying to \novercome a rare genetic disease. Both girls \nhave now hopefully had...
next steprare diseasefamilypreparesgirls
https://yespunjab.com/maharashtra-reports-first-death-of-rare-disease-guillain-barre-syndrome/
Maharashtra reports first death of rare disease Guillain-Barre Syndrome - Yes Punjab News
Jan 28, 2025 - Maharashtra reports its first death due to Guillain-Barre Syndrome (GBS) in Pune. The state is on alert with 73 cases reported, as health authorities take...
death ofrare diseasepunjab newsmaharashtrareports
https://recordati.com/rare-disease-day-2016-02-29-110000-business-financial-news/
RARE DISEASE DAY, - 2016-02-29 11:00:00 - Business & Financial News - Recordati
rare disease dayfinancial newsbusinessrecordati
https://truemedtx.com/
TrueMed Therapeutics Israel - Rare Disease and Niche Specialty Care Company in Israel
Dec 10, 2025 - TrueMed Therapeutics Israel is a Premier Rare Disease and Niche Specialty Care Company in Israel. we deliver rare dedication to wellness.
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https://www.wcgclinical.com/insights/archive/therapeutic-area/rare-disease/
Rare Disease Archives | WCG
rare diseasearchiveswcg
https://biohealthinnovation.org/rare-disease-racers-speak-out-stand-tall-at-racefor7-events-in-the-u-s-and-india/
Rare disease racers speak out, stand tall at Racefor7 events in the U.S. and India - BioHealth...
May 22, 2025 - Visibility, speaking out, courage and organization. Forty years ago, these were the things that brought the Orphan Drug Act to life. On Sunday, February 24,...
rare diseasespeak outand indiaracersstand
https://www.utwente.nl/en/news/2024/11/1842761/tailor-made-treatments-for-ultra-rare-disease-patients
Tailor-made treatments for ultra-rare disease patients
In a new study, a team of researchers, including Anneliene Jonker, has published the first roadmap for developing genetic therapies for ultra-rare diseases....
tailor madeultra raretreatmentsdiseasepatients
https://www.m3india.in/contents/clinical_news/rare-disease-study-uncovers-amygdala-s-impact-on-generosity-and-social
Rare disease study uncovers amygdala's impact on generosity and social
Are there areas of the brain that regulate prosocial, altruistic behaviour? Together with colleagues from the universities in Lausanne, Utrecht and Cape Town...
rare diseaseand socialstudyamygdalaimpact
https://www.amgen.ca/en-ca/stories/categories/rare-disease
Rare Disease
rare disease
https://www.pharmanow.live/marketing/specialty-rare-disease-gtm
Go-To-Market Strategies For Specialty And Rare Disease Therapies
Explore how pharma companies must redesign go-to-market strategies for specialty and rare diseases using precision, patient-centricity, and data-driven...
go to marketrare diseasestrategiesspecialtytherapies
https://medinform.jmir.org/2024/1/e60665/tweetations
JMIR Medical Informatics - An Automatic and End-to-End System for Rare Disease Knowledge Graph...
Background: Rare diseases affect millions worldwide but sometimes face limited research focus individually due to low prevalence. Many rare diseases do not...
medical informaticsrare diseaseknowledge graphjmirautomatic
https://www.fox13now.com/news/national/parents-hope-football-signed-by-chiefs-will-help-fund-cure-for-childs-rare-disease
Signed Chiefs football to help fund rare disease cure
Jan 22, 2020 - A mother and father are hoping a football signed by every Kansas City Chiefs player will help save their son.
to helprare diseasesignedchiefsfootball
https://www.easp.es/project/implementing-outcomes-based-managed-entry-agreements-for-rare-disease-treatments-nusinersen-and-tisagenlecleucel/
Implementing Outcomes-Based Managed Entry Agreements for Rare Disease Treatments: Nusinersen and...
managed entry agreementsrare diseaseimplementingoutcomesbased
https://www.ptcbio.com/
PTC Therapeutics: Pioneering Rare Disease Treatments
May 7, 2026 - Explore PTC Therapeutics' commitment to developing innovative treatments for rare diseases.
ptc therapeuticsrare diseasepioneeringtreatments
https://news.microsoft.com/source/features/work-life/separated-by-an-ocean-two-people-connect-through-gaming-similar-passions-and-a-rare-disease/
Separated by an ocean, two people connect through gaming, similar passions and a rare disease -...
Jun 14, 2023 - As a child, Megan Shaw was always falling. She bruised easily, seemed to be accident-prone and fainted a lot. As a teenager, she found out she had...
ocean tworare diseaseseparatedpeopleconnect
https://raredisease.net/living/social-media-comments
Calling Out With Grace: Social Media as a Rare Disease Advocate
A rare disease patient advocate shares tips for reflecting before commenting on social media and how one interaction made a difference for patients.
calling outsocial mediarare diseasegraceadvocate
https://020110.blogspot.com/2014/02/rare-disease-day_28.html
today is a miracle: Rare Disease Day
Happy Rare Disease Day! We (meaning everyone involved and spreading the word about Rare Disease Day) are trending on Facebook, which ...
rare disease daytodaymiracle
https://www.labmedica.com/molecular-diagnostics/articles/294809650/long-read-rna-sequencing-platform-improves-rare-disease-diagnosis.html
Long-Read RNA Sequencing Platform Improves Rare Disease Diagnosis - molecular-diagnostics -...
Long-Read RNA Sequencing Platform Improves Rare Disease Diagnosis
long readrna sequencingrare diseasemolecular diagnosticsplatform
https://www.annchildneurol.org/journal/view.php?doi=10.26815/acn.2022.00129
Ahn, Kim, Lim, Kim, and Chae: Variable Phenotypes of ZC4H2-Associated Rare Disease in Six Patients
rare diseasekimlimvariablephenotypes
https://kunduz.com/questions-and-answers/a-woman-with-achondroplasia-autosomal-dominant-rare-disease-that-exhibits-dwarfism-married-a-man-who-was-normal-height-she-gave-birth-to-a-child-afflicted-with-achondroplasia-what-is-the-chance-that-173930/
[ANSWERED] A woman with achondroplasia autosomal dominant rare disease - Kunduz
a womanrare diseaseansweredachondroplasiaautosomal
https://www.hunimed.eu/news/29-february-rare-disease-day/
29 February: Rare Disease Day - Hunimed
Oct 14, 2025 - Rare Disease Day is the most important date for all people diagnosed with a rare disease, as well as for their families, healthcare professionals,
rare disease dayfebruary
https://bullishdaily.com/news/neurocrine-2-9b-soleno-acquisition-rare-disease-consolidation
Neurocrine Biosciences' $2.9B Soleno Acquisition Signals Biotech Consolidation in Rare Disease...
Neurocrine Biosciences' $2.9B cash buyout of Soleno Therapeutics adds VYKAT XR, the first FDA-approved therapy for Prader-Willi hyperphagia, bolstering its...
neurocrine biosciencesrare diseasesolenoacquisitionsignals
https://www.uclahealth.org/news/release/PNP2022
Determining how nucleoside metabolism regulates contrasting immune functions in the rare disease...
A new study sheds light on the immunoregulatory mechanisms of the metabolic enzyme purine nucleoside phosphorylase (PNP), providing insights for future...
rare diseasemetabolismimmunefunctions
https://thetab.com/2024/12/18/what-is-sarcoidosis-the-rare-disease-dennis-thinks-he-has-in-no-good-deed
What is sarcoidosis, the rare disease Dennis thinks he has in No Good Deed?
Dec 18, 2024 - Dennis has a huge health scare in Netflix's No Good Deed when he thinks he has sarcoidosis, but what is the rare disease?
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https://xtalks.com/canadian-biotech-startup-239/
Canadian Biotech Startup Tackles Blood Cancer and Rare Disease - Xtalks
Apr 23, 2025 - A new Toronto-based biotech company has announced two novel ex vivo treatments for blood cancer and a rare disease, using gene therapy. AvroBio is a joint...
biotech startupblood cancerrare diseasecanadiantackles