CheckRare

🚨2026 orphan drug PDUFA & FDA approval dates are here! Get the latest timeline and status for rare disease therapies: checkrare.com/2026-orphan-dr… #RareDisease #OrphanDrugs #FDA #PDUFA

In young patients with short stature, growth hormone deficiency is the most common cause. However, in a small subset of patients, the underlying problem may be an abnormality in insulin-like growth factor-1 (IGF-1) levels. Learn more about the differences between these two rare conditions with Dr. Robert Rapaport at checkrare.com/primary-igf-1-… #CheckRare #IGF1Deficiency #RareEndocrine #GrowthHormoneDeficiency

The latest in Cutaneous T-Cell Lymphoma: Julia Scarisbrick, MD, discusses the the PROCLIPI study and challenges in the diagnosis of CTCL at checkrare.com/prospective-cu… Lauren Shea, MD, discusses important aspects of medical treatment and stem-cell transplantation in patients with CTCL at checkrare.com/medical-treatm… #CheckRare #CTCL #RareCancer #RareOncology #ClinicalTrials #DrugDevelopment

🎗️ #CureFest2026, hosted by the Arms Wide Open Childhood Cancer Foundation takes place this September 18-20 in Washington, DC! Featuring speeches, performances, games, activities, and a memorial shoe display, CureFest provides a platform to connect and foster collaboration with patients, families, caregivers, physicians, researchers, and elected representatives. Register at curefestusa.org @CureFestUSA

💡Rare Disease Spotlight: PLGD-1 Learn more about this rare disease with our Learning Center checkrare.com/plasminogen-de… #CheckRare #RareDisease #PLGD #PLGD1 #RareGenetic #RareOphthalmology

June 2026 FDA Milestones in Rare Disease Care! Stay up to date with our 2026 PDUFA Dates and FDA Approvals page at checkrare.com/2026-orphan-dr… #RareDisease #FDAApproval #MedicalBreakthroughs #CheckRare

💡Rare Disease Spotlight: Pediatric Adrenal Insufficiency Learn more about this rare disease at checkrare.com/pediatric-adre… #CheckRare #RareDisease #PediatricAdrenalInsufficiency #RareEndocrine

Arthur Beisang, MD, Department of Pediatrics at Gillette Children’s Specialty Healthcare in Saint Paul, Minnesota, discusses treatment of Rett syndrome. checkrare.com/daybue-trofine… #CheckRare #RettSyndrome #RareDiseaseTreatment #RareGenetic #RareNeurology

🎗️ The @CureFestUSA for Childhood Cancer event, hosted by the Arms Wide Open Childhood Cancer Foundation takes place September 18-20 in Washington, DC! Register at curefestusa.org #CureFest2026

Christopher Romero, MD, discusses #AVPD, previously known as central diabetes insipidus. Learn the reason behind the name change, presentation, diagnosis, and management at checkrare.com/overview-of-ar… #CheckRare #RareGenetic #RareEndocrine #RareNeurology

This past weekend, CheckRare was once again honored to cover ENDO 2026. Stay tuned for our expert interviews with world renowned thought leaders, advocates, and rare patients and caregivers. #ENDO2026

June is CAH Awareness Month 🩵 Learn more about this rare disease and its diagnosis, management, and research advancements at checkrare.com/june-is-cah-aw… #CheckRare #CAHAwarenessMonth #CAH #CongenitalAdrenalHyperplasia #RareEndocrine

John Day, MD, PhD, Director of Neuromuscular Medicine at Stanford University, discusses the development of Itvisma gene therapy for patients with spinal muscular atrophy (SMA). checkrare.com/itvisma-gene-t… #CheckRare #RareMusculoskeletal #RareNeurology #SMA #SpinalMuscularAtrophy #DrugDevelopment

CheckRare is excited to once again serve as a media partner for the World Orphan Drug Congress. This event brings together leaders, innovators, advocates, and experts from across the rare disease community to share insights, discuss emerging trends, and explore opportunities to advance patient care and education. If you're attending, we'd love to connect. Stop by Booth #640 to learn more about how CheckRare supports rare disease education and awareness. #RareDisease #WorldOrphanDrugCongress #RareDiseaseCommunity #PatientAdvocacy @orphan_drugs

James Howard Jr., MD, Professor of Neurology at the University of North Carolina at Chapel Hill, discusses results from the ADAPT SERON clinical trial of efgartigimod IV in patients with seronegative generalized myasthenia gravis (gMG). Data from this trial was pivotal in efgartigimod’s recent FDA approval. Learn more at checkrare.com/results-from-t… #CheckRare #MyastheniaGravis #RareNeurology #RareAutoimmune #RareMusculoskeletal #ClinicalTrial #DrugDevelopment

Even though the ligneous lesions on the eye are generally the first symptom of PLGD-1, the deficiency can present in many different ways (and in virtually any organ system with mucous membranes). Learn about the key features of disease at checkrare.com/diagnosis-and-… #CheckRare #PLGD #RareGenetic #RareDisease

📢 May 2026 FDA Milestones in Rare Disease Care! Stay up to date with our 2026 PDUFA Dates and FDA Approvals page at checkrare.com/2026-orphan-dr… #RareDisease #FDAApproval #MedicalBreakthroughs #CheckRare

Today is officially World #COL6Day! 🎉 Join us in celebrating the inaugural World Collagen 6 Day, dedicated to promoting global compassion, research, and care for those affected by COL6-related myopathies. Join the Virtual Congress: col6.world/applicationform #CheckRare #RareDisease #COL6Day #COL6Myopathy #RareGenetic #RareNeurology #RareMusculoskeletal

🎗️Join us at the @CureFestUSA for Childhood Cancer event, hosted by the Arms Wide Open Childhood Cancer Foundation this September 18-20 in Washington, DC! Featuring speeches, performances, games, activities, and a memorial shoe display, CureFest provides a platform to connect and foster collaboration with patients, families, caregivers, physicians, researchers, and elected representatives. Register at curefestusa.org #CureFest2026

💡Rare Disease Spotlight: Prader-Willi Syndrome Learn more about this rare disease with our Learning Center checkrare.com/prader-willi-s… #CheckRare #RareDisease #PraderWilli #RareGenetic

The #CheckRare team has been hard at work at #ASCO26 attending clinical data sessions. This was an exciting conference with a ton of presentations on the latest in #RareCancer!