Rare Diseases Clinical Research Network

New research from the Network for Advancing #SexChromosomeAneuploidy Research Readiness (NASCARR) ✅ Creating a #PatientRegistry to Improve Health Outcomes for Individuals with #SexChromosomeAneuploidies  Listen and read more: #NASCARR" target="_blank" rel="nofollow noopener">rarediseasesnetwork.org/news/research-…

New research from the #MyastheniaGravis Rare Disease Network (MGNet): ✅ Investigating the Impact of Demographic Trends on Disease Prognosis in Myasthenia Gravis Listen and read more: #MGNET" target="_blank" rel="nofollow noopener">rarediseasesnetwork.org/news/research-…

The Batten Disease Clinical Research Consortium has awarded the 2026 Early Career Investigator Grant to Aikaterini Chatzipli, PhD, of @BostonChildrens and @harvardmed, and the 2026 Pilot Project Grant to Ruchira Singh, PhD, of @UR_Med! Learn more: rarediseasesnetwork.org/news/consortiu…

New research from The IMPACT Study: ✅ Advancing Treatment in #CongenitalThromboticThrombocytopenicPurpura (#cTTP) Listen and read more: #IMPACT" target="_blank" rel="nofollow noopener">rarediseasesnetwork.org/news/research-…

🎧 New podcast episodes now available! Featuring: ✅ #CongenitalThromboticThrombocytopenicPurpura ✅ #MyastheniaGravis ✅ #SexChromosomeAneuploidies ✅ #ATP1A3-Related Disorders ✅ #OrnithineTranscarbamylase (#OTCDeficiency) Listen and subscribe: spreaker.com/podcast/rare-r…

🚨 New funding opportunities in #MyastheniaGravis from @MGNet__! 💬 Support for myasthenia gravis research and career development ✅ Scholar Program ✅ Pilot Grant Program Learn more and apply: mgnet.rarediseasesnetwork.org/funding-opport…

New research from the Global #Leukodystrophy Initiative Clinical Trials Network (GLIA-CTN): ✅ Investigating #LanguageSkills in Children with #AlexanderDisease Listen and read more: #GLIA" target="_blank" rel="nofollow noopener">rarediseasesnetwork.org/news/research-…

🎉 April is National #PrimaryImmunodeficiency Awareness Month! ➡️ Learn more about primary immunodeficiency: rarediseasesnetwork.org/diseases/168/p… ➡️ Check out our research: pidtc.rarediseasesnetwork.org ➡️ Get involved with our patient advocacy partner @immunedeficienC: primaryimmune.org

Meet Andrew D. Tran, MD! Dr. Tran is a faculty member @uamshealth & @archildrens. With the Advancing Rare Disorders: #VascularMalformation Research Network with CaNVAS (ARDVARC), his work focuses on using #AI & #MachineLearning to improve patient safety. rarediseasesnetwork.org/news/consortiu…

🎉 Today is International #Pompe Day! ➡️ Learn more about #PompeDisease: rarediseasesnetwork.org/diseases/164/p… ➡️ Check out our research: ldn.rarediseasesnetwork.org

"I'm working out the way to cope, counting every dose of hope. I'm trying to be who I want to be, living with a #RareDisease." The @MGNet__ team created a song about the struggles of living with a rare disease, like #MyastheniaGravis: youtube.com/watch?v=UqOI0t…

🚨 New funding opportunity! ✅ IMPACT Early Career Immersion Award ➡️ Supporting early career scholars with a 12-month translational or clinical immersion experience in #ThromboticMicroangiopathies (TMAs) 🔗 Learn more and apply: grants.rarediseasesnetwork.org/grants/IMPACT-…

April is #UreaCycleDisorders (UCD) Awareness Month! ➡️ Learn more about UCD: ucdc.rarediseasesnetwork.org/diseases-studi… ➡️ Check out our research: ucdc.rarediseasesnetwork.org ➡️ Get involved with our patient advocacy partner @CureUCD: nucdf.org

🎧 New podcast episodes now available! Featuring: ✅ #AlexanderDisease ✅ Progressive #MovementDisorders Listen and subscribe: spreaker.com/podcast/rare-r…

Patient stories matter. 💙 NPKUA is proud to support @amcporg's In Their Words Patient Journey Collection, sharing real experiences to drive patient‑centered care. Watch Valerie’s story: tinyurl.com/4w62yn2r #AMCPLearn #RareDisease #ManagedCarePharmacy #PKU

New research from the #UreaCycleDisorders Consortium (UCDC): ✅ Evaluating a New Food Photography App for Measuring Dietary Intake in Urea Cycle Disorders Listen and read more: #UCDC" target="_blank" rel="nofollow noopener">rarediseasesnetwork.org/news/research-…

New research from the #SpasticParaplegia Centers of Excellence Research Network: ✅ Assessing Health-Related #QualityOfLife in Children with Rare Forms of #HereditarySpasticParaplegia ✅ Investigating the ATG9A Ratio as a Diagnostic Tool for AP-4-HSP #SPCERN" target="_blank" rel="nofollow noopener">rarediseasesnetwork.org/news/research-…

New research from the North American #MitochondrialDisease Consortium (NAMDC): ✅ Using a New Statistical Technique for #Accelerometer Data to Assess a Treatment for Mitochondrial Disease Listen and read more: #NAMDC" target="_blank" rel="nofollow noopener">rarediseasesnetwork.org/news/research-…

Calling all phenylketonuria (PKU) researchers! National PKU Alliance’s annual research grant program is now open. Visit npkua.grantplatform.com for more details and to apply. 📅 The deadline to submit proposals is Monday, June 15, 2026 at 11:59pm ET. Apply today!

New research from the Global #Leukodystrophy Initiative Clinical Trials Network: ✅Exploring the Use of Glial Fibrillary Acidic Protein as a #Biomarker in #AlexanderDisease ✅Reviewing Pathology and Interventions in Cerebral X-Linked #Adrenoleukodystrophy #GLIA" target="_blank" rel="nofollow noopener">rarediseasesnetwork.org/news/research-…