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

📢 August was full of FDA approvals 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

🎙️ Episode 2 of Rare Endocrine Exchange is out now! In this episode, Dr. Margarita Ochoa-Maya and Dr. James Radke discuss why diagnostic delays occur and what can be done to help patients reach the right diagnosis sooner. 🎧 Listen now at the link below, or wherever you get your podcasts. checkrare.com/decoding-delay… #CheckRare #RareEndocrineExchange #RareDiseaseNews #RareEndocrine #Podcast

📚New CME program now available! Dive into the latest clinical research highlights in myasthenia gravis from the American Academy of Neurology Annual Meeting (AAN 2026) and earn CME credit. Enroll now at checkrare.com/learning/p-mya… #CheckRare #CME #CMEProgram #MyastheniaGravis

🔎New Learning Center- Congenital Adrenal Hyperplasia Learn more at checkrare.com/congenital-adr… #CheckRare #RareDisease #CongenitalAdrenalHyperplasia #CAH #RareGenetic

🎧 Episode 5 of The CheckRare Brief is available now! Listen at checkrare.com/new-fda-approv… or wherever you get your podcasts. #CheckRare #TheCheckRareBrief #RareDiseaseNews

PAI is characterized by inadequate production of cortisol and sometimes aldosterone, often caused by dysfunction in the adrenal cortex. Additionally, diagnosis can be challenging as early signs and features are vague and mimic other clinical problems. Learn more about the diagnosis and management of PAI with Mitchell Geffner, MD, at checkrare.com/pediatric-adre… #CheckRare #PediatricAdrenalInsufficiency #PAI #RareEndocrine