NIH-funded network fostering collaborative research among 21 teams of researchers, patients, and clinicians, each focused on a group of rare diseases.
United States
Joined October 2011
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- Following488
- Followers8.6K
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"For me, the @KTSupportGroup Annual Conference provided me w/the opportunity to be part of a #RareDisease community, to feel the reward that only a career combining #ClinicalCare & #MedicalResearch gives."—Dr. Michael Jeng, principal investigator, ARDVARC rarediseasesnetwork.org/news…
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The first-ever @rarediseasesnet Annual Report is now live! 🎉
Explore the report to learn about #RareDisease research consortium achievements, early career investigators, patients and patient advocacy groups, and other network accomplishments: rarediseasesnetwork.org/annu…
ALT Rare Diseases Clinical Research Network 2026 Annual Report
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We are welcoming eight new research groups to @rarediseasesnet! Each group will bring together scientists, clinicians, patients, families, and patient advocates to study a wide range of #RareDiseases. Read more about the new groups: rarediseasesnetwork.org/news…
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New research from the Rare Organic Acidemias Research Consortium (ROAR):
✅ Exploring Clinical and Mechanistic Data on Immune Dysregulation in #OrganicAcidemias
Listen and read more: rarediseasesnetwork.org/news…
New research from the North American #MitochondrialDisease Consortium (NAMDC):
✅ Assessing Exercise Training Protocols in #PrimaryMitochondrialDiseases
Listen and read more: rarediseasesnetwork.org/news…
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What are #NaturalHistory studies and why are they important for #RareDiseases?
Dr. Darius Ebrahimi-Fakhari of @BostonChildrens @harvardmed & the Spastic Paraplegia Centers of Excellence Research Network (SP-CERN) explains: healthawareness.co.uk/rare-d…
#RareDiseases2026
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New research from the #BrittleBoneDisorders Consortium (BBDC):
✅ Evaluating #LungDisease in Adults with #OsteogenesisImperfecta
Listen and read more: rarediseasesnetwork.org/news…
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🎧 New podcast episodes now available!
Featuring:
✅ #OsteogenesisImperfecta
✅ Primary #MitochondrialDiseases
✅ #OrganicAcidemias
Listen and subscribe: spreaker.com/podcast/rare-re…
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When will a person with #AmyotrophicLateralSclerosis (#ALS) begin to experience symptoms? Researchers have discovered new #biomarkers that can help predict the arrival of symptoms, allowing early intervention that could prevent permanent motor damage: rarediseasesnetwork.org/news…
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New research from the #SpasticParaplegia Centers of Excellence Research Network (SP-CERN):
✅ Investigating Genotype–Phenotype Correlations and Natural History of Early-Onset #SPG4
Listen and read more: rarediseasesnetwork.org/news…
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“The proposed work will have a major impact on advancing evidence-based treatment of invasive #mold infections in #immunocompromised children." —William Otto, MD
pfn-stride.rarediseasesnetwo…
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New research from the Consortium of #Eosinophilic #GastrointestinalDisease Researchers (CEGIR):
✅ Investigating the Use of #Dupilumab to Improve Outcomes for Individuals with #EosinophilicGastritis
Listen and read more: rarediseasesnetwork.org/news…
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“These #biomarkers give us a far better idea of the timing, allowing us to estimate the time to symptom onset with an average error of about 18 months. That’s something we can work with.”—Michael Benatar, MD, PhD, Principal Investigator, CReATe
#ALS #AmyotrophicLateralSclerosis
NEWS: New blood-based biomarkers predict when ALS symptoms will emerge.
Read the full story ➡️ bit.ly/4hiFpp9
ALT Blue background with white text that reads: "Press Release: New blood-based biomarkers predict when ALS symptoms will emerge." The National Institutes of Health (NIH) logo is at the bottom.
A new pilot study led by William Otto, MD, of @CincyResearch aims to use #ElectronicHealthRecord-based algorithms to improve identification of pediatric invasive mold infections in #immunocompromised children. Learn more about the PFN-STRIDE pilot study: rarediseasesnetwork.org/news…
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“Information gained from research like this brings us one step closer to providing much-needed treatment for our patients.”—Nirmala Gonsalves, MD
Learn more about CEGIR's recent research on dupilumab and #EosinophilicGastritis: rarediseasesnetwork.org/news…
New research from the Global #Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN):
✅ Developing a New Process to Improve Diagnostic Accuracy in Leukodystrophies
✅ Measuring Function in Children with Leukodystrophy
Listen and read more: rarediseasesnetwork.org/news…
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How does #HereditarySpasticParaplegia type 4 (#SPG4) change over time? To track down the answers, researchers from the Spastic Paraplegia Centers of Excellence Research Network (SP-CERN) have created the most detailed natural history of SPG4 to date: rarediseasesnetwork.org/news…
Rare Diseases Clinical Research Network retweeted
A new phase 2 clinical trial published in @LancetGastroHep found that investigational dupilumab improved gastric eosinophil counts and histologic and endoscopic outcomes in people with eosinophilic gastritis (EoG).
Learn more: scienceblog.cincinnatichildr…
In a first-of-its-kind study, researchers from the Consortium of #EosinophilicGastrointestinalDisease Researchers (CEGIR) found that #dupilumab improves outcomes for individuals with #EosinophilicGastritis. Read more: rarediseasesnetwork.org/news…
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Rare Diseases Clinical Research Network retweeted
🎙️Tune into #ONAIR as Mitch Gore welcomes author @TabithaOrth to discuss her touching children's book, Ima Rare. Explore Ima’s adventurous journey through a #raredisease Dx Perfect for all ages. Don't miss this heartfelt episode! Watch 🔗👉: youtu.be/Si1x-hkV4Ns #Pediatric
