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

CNBC's @BeckyQuick, rare disease mom, co-anchor of Squawk Box, and host of CNBC Cures, moderates a high-level CEO discussion on "The Next Decade of Rare Disease: A CEO Leadership Dialogue." The panel features: - Renee Gala, President and CEO, @JazzPharma - Pamela Gavin, MBA, President and CEO, @RareDiseases - Alexander Hardy, President and CEO, @BioMarin - Tarek Rabah, President and CEO, @OtsukaUS #NORDSummit #RareDisease #RareDiseaseLeadership

NIH Director Jay Bhattacharya, MD, PhD, discusses the importance of including patients, families, and caregivers in the NIH's work. " We need to give greater attention to the viewpoints from patients and families." #NORDSummit #RareDisease #PatientVoice

FDA Acting Commissioner Kyle Diamantas discusses how the FDA plans to incorporate the voices and experiences of people living with rare diseases into its decision-making. #NORDSummit #RareDisease #PatientVoice

CheckRare is proud to once again partner with the @RareDiseases NORD Breakthrough Summit, connecting with leaders who are advancing the rare disease community. We look forward to learning from inspiring voices like Pam Gavin, President & CEO of NORD. #NORDSummit #RareDisease #BecauseRareMatters

CheckRare is excited to report on the latest rare kidney disorder developments from ASN Kidney Week 2026 in Denver, Colorado. Keep an eye out for our coverage from the meeting #KidneyWk

Are you tuning in to the NORD Breakthrough Summit, October 26 and 27? You can still register to virtually attend this annual gathering of the national rare disease community—featuring CNBC’s Becky Quick and the leaders of the FDA and NIH—from wherever you are right now. Join us: nordsummit.org/?utm_source=c…

🧬 Advancing research in hypochondroplasia Phase 3 results from the CANOPY-HCH-3 trial show that vosoritide improved annualized growth velocity compared with placebo in children with hypochondroplasia. Researchers also reported improvements in arm span. These findings represent an encouraging step toward understanding new potential treatment options for people living with hypochondroplasia. Learn more from CheckRare: checkrare.com/efficacy-of-vo… #Hypochondroplasia #Vosoritide #RareDiseaseResearch #ClinicalTrials #RareDiseaseAwareness #RareMusculoskeletal

💜 October is Rett Syndrome Awareness Month The Rett syndrome landscape is always evolving, and better diagnostic understanding and novel treatment options are continuing to improve patients outcomes. Learn about the latest in treatments and clinical developments at checkrare.com/rett-syndrome-… #RettSyndromeAwarenessMonth #RettSyndrome #Rett #RareDisease

There’s still time to register for next week’s 2026 NORD Breakthrough Summit, in Washington, D.C. and online October 26-27! Join the nation’s top health officials, biopharma and medtech innovators, venture investors, and leaders of hundreds of patient organizations for conversations that will shape the future of rare disease medicine. Register now: nordsummit.org/?utm_source=c…

Episode 12 of The CheckRare Brief is live! Listen at checkrare.com/ultragenyx-get… or wherever you get your podcasts.

🔬 New developments in achondroplasia research! @BridgeBioPharma Phase 3 clinical trial results for infigratinib show improvements in annualized growth velocity compared with placebo, along with positive changes in height scores and body proportions in children with achondroplasia. With FDA review underway, these findings mark an important milestone in the search for new treatment options. Learn more from CheckRare: checkrare.com/efficacy-of-in… #Achondroplasia #RareDisease #ClinicalResearch #RareDiseaseAwareness #DrugDevelopment #RareMusculoskeletal

💡Rare Disease Spotlight: Severe Primary IGF-1 Deficiency Learn more about this rare disease at checkrare.com/primary-igf-1-… #CheckRare #RareDisease #PrimaryIGF1Defiency #RareEndocrine #RareGenetic

CheckRare is at #CHEST2026 in Phoenix 🌵 Stay tuned for conference coverage!

🔎 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