October is Rett Syndrome Awareness Month 💜 Rett syndrome is a neurodevelopmental condition characterized by normal psychomotor development during the first 6 to 18 months of life, followed by the development of severe problems with language and communication, learning, coordination, and other brain functions. This month, join us in recognizing the importance of continued research, multidisciplinary care, patient advocacy, and education. Learn more at checkrare.com/rett-syndrome-… #RettSyndromeAwarenessMonth #RettSyndrome #Rett #RareDisease

🔎 Understanding Arginine Vasopressin Deficiency (AVP-D) Previously known as central diabetes insipidus, AVP-D is a rare condition that affects the body’s ability to regulate water balance. In this CheckRare overview, Christopher Romero, MD, discusses the symptoms, diagnostic challenges, and management of AVP-D, highlighting the importance of awareness and early recognition to help improve patient outcomes. View the full overview: checkrare.com/overview-of-ar… #CheckRare #RareDisease #AVPD #RareDiseaseAwareness #Endocrinology #RareDiseases

This week's episode of The CheckRare Brief is live! Listen at checkrare.com/ultragenyx-sel… or wherever you get your podcasts. #TheCheckRareBrief #RareDisease #RareDiseaseNews

Episode 10 of The CheckRare Brief is out now! Listen at checkrare.com/therapies-for-… or wherever you get your podcasts. #CheckRare #TheCheckRareBrief #RareDisease #Podcast

🔎 What are the common signs and symptoms of Rett syndrome? This Rett Syndrome Awareness Month, learn more about this rare neurodevelopmental condition and join us in recognizing the importance of continued research, multidisciplinary care, patient advocacy, and education. Read more at checkrare.com/rett-syndrome-… #RettSyndromeAwarenessMonth #RettSyndrome #Rett #RareDisease

💊 September was a busy month in rare disease regulatory decisions. Learn more about each of these approvals at checkrare.com/2026-orphan-dr… #CheckRare #RareDisease #FDADecisions #FDAApprovals #RareDiseaseApprovals #RareDiseaseTreatment

This week on The CheckRare Brief, we are joined by Eugene Lee, Head of @CMIMediaGroup's Rare Disease Division, for an industry perspective on some of the biggest developments in rare disease. Tune in to learn about CMI Media Group's Specialized Rare Disease Center of Excellence and this week's latest in FDA approvals. Listen now at checkrare.com/new-rare-disea… #TheCheckRareBrief #CMIMediaGroup #Podcast #RareDisease

CheckRare is excited to once again report on the latest in Neuromuscular and Electrodiagnostic Medicine from the 2026 @AANEMorg Annual Meeting in Orlando. Keep and eye out for our coverage from the MGFA Scientific Session. #AANEMinOrlando #MyastheniaGravis #MGstrong

What role could menin inhibition play in newly diagnosed NPM1-mutated AML? A Phase 3 study is evaluating revumenib + intensive chemotherapy versus chemotherapy alone, with event-free survival and MRD complete remission among the primary endpoints. The use of revumenib in this newly diagnosed setting is investigational and is not currently FDA-approved for this use. Hear Dr. Joshua Zeidner explain the rationale and what researchers hope to learn. Read/watch: checkrare.com/revumenib-chem… #AML #NPM1 #Leukemia #Hematology #CancerResearch #RareCancer

🔎 Beyond AChR: Recognizing and Treating Less-Recognized Forms of Myasthenia Gravis Not all myasthenia gravis (MG) looks the same. MuSK Ab+, LRP4 Ab+, and triple seronegative MG can present unique diagnostic and treatment challenges—and recognizing these subtypes can help inform more individualized care. This CME activity from CheckRare explores: - Subtype-specific clinical features and diagnostic approaches - Disease mechanisms and treatment considerations - When patients may be suboptimally controlled on traditional therapies - How emerging, targeted approaches may inform treatment planning 🎓 Earn 0.75 AMA PRA Category 1 Credits™ while learning from neuromuscular experts Neelam Goyal, MD and Christyn Edmundson, MD. 👉 Explore the activity and enroll: checkrare.com/learning/p-bey… #MyastheniaGravis #MG #Neurology #CME #MedicalEducation #ContinuingMedicalEducation #RareDisease #Neuromuscular

💡Rare Disease Spotlight: Congenital Adrenal Hyperplasia Learn more about this rare disease at checkrare.com/congenital-adr… #CheckRare #RareDisease #CongenitalAdrenalHyperplasia

🎙️ Episode 8 of The CheckRare Brief is live! Tune in at checkrare.com/fda-approves-n… or wherever you get your podcasts.

💙💚 Join us in celebrating Rare Cancer Day 💚💙 @targetcancer #RareCancerDay

🎙️ Listen to Episode 7 of The CheckRare Brief, out now! Available at checkrare.com/multimedia/the… or wherever you get your podcasts #TheCheckRareBrief #Podcast #RareDiseaseNews

🔎 Could you recognize systemic mastocytosis earlier? With diagnosis often taking nearly 5 years, recognizing the early signs of systemic mastocytosis (SM) is critical. Join hematology expert Daniel J. DeAngelo, MD, PhD for a 45-minute accredited CME activity focused on: - Recognizing early clinical signs and symptoms of SM - Applying current diagnostic criteria and tools - Improving timely referral and testing Designed for physicians across hematology, dermatology, gastroenterology, immunology, and family practice. 📚 Expand your clinical approach to identifying SM and help shorten the path to diagnosis. Enroll now at checkrare.com/learning/p-sys… #CME #SystemicMastocytosis #RareHematology #MedicalEducation #RareDisease

This weekend! Join us at the #CureFest2026 for Childhood Cancer event 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. Last chance to register at curefestusa.org/ #CureFest2026

🎧 Episode 6 of The CheckRare Brief is available now! Listen at checkrare.com/new-huntington… or wherever you get your podcasts. #CheckRare #TheCheckRareBrief #RareDiseaseNews

🩸 CheckRare is at #SOHO2026 in Houston covering important data on hematologic malignancies. This event brings together HCPs to learn about the advances in leukemias, lymphomas, myeloma, myelodysplastic neoplasms, and cellular therapies.

🔬 Can serum proteomics help predict treatment response in myasthenia gravis? New research combining proteomic profiling and machine learning identified treatment-specific protein signatures associated with 6-month clinical improvement. The findings highlight the potential of biomarkers to support treatment selection, risk stratification, and a more personalized approach to MG care, while offering new insights into the biology underlying different treatment responses. Learn more at checkrare.com/predicting-tre… #MyastheniaGravis #MG #RareNeurology #RareNeuromuscular #RareDisease #PrecisionMedicine #ClinicalResearch

Do you want to make a real difference? Help us support Lipodystrophy United’s EL-PFDD. A donation of any amount can support a person and, eventually, a cure! Donate here: lipodystrophyunited.org/form… #LU-PFDD #Lipodystrophy #LipodystrophyUnited #25storiesfor25K