COR2ED - Rare Diseases Medical Conversation

Think narcolepsy: Why early recognition and referral matter in an evolving treatment landscape

Join experts Dr Markus Schmidt and Dr Anna Heidbreder as they discuss the importance of early recognition and referral in narcolepsy type 1 (NT1), particularly as new targeted treatments begin to emerge.  Key discussion points include:  * Emerging therapies: How OX2R agonists may transform narcolepsy care, with Phase 3 overporextin data showing significant improvements in wakefulness and cataplexy with no serious AEs reported.  * Diagnostic red flags: Recognising excessive daytime sleepiness, cataplexy, sleep paralysis, and hallucinations to help reduce the current 7–10 year delay in diagnosis.  * Clinical pitfalls: Avoiding common misdiagnoses, such as attributing symptoms to lifestyle-related fatigue in younger patients or sleep apnoea in older adults.  * Specialist referral: The importance of timely referral for specialist assessment and objective sleep testing to support earlier access to emerging therapies.  By identifying these symptom clusters earlier, clinicians may improve long-term outcomes and quality of life for people living with NT1.    Key clinical takeaways:   * New treatment options are emerging: OX2R agonists represent a shift toward targeting the underlying biology of NT1, with the potential to significantly improve patient outcomes  * Early recognition is critical: Narcolepsy is often under-recognised and misdiagnosed, so clinicians should maintain a high index of suspicion, particularly in patients with excessive daytime sleepiness  * Act early and refer appropriately: Timely referral to specialists is essential to confirm diagnosis, optimise management, and ensure patients can benefit from current and emerging therapies  You can also watch a video of the experts in conversation and download the accompanying transcript on our website: https://cor2ed.com/sleep-connect/programmes/narcolepsy-early-recognition-referral/?media=0 [https://cor2ed.com/sleep-connect/programmes/narcolepsy-early-recognition-referral/?media=0]  Or watch on YouTube: https://youtu.be/jHgORhjB_lw [https://youtu.be/jHgORhjB_lw]  Follow us on social media:  LinkedIn: https://www.linkedin.com/company/sleep-connect/about/ [https://www.linkedin.com/company/sleep-connect/about/]  X: https://x.com/SleepConnect_ [https://x.com/SleepConnect_]   Instagram: https://www.instagram.com/cor2edmeded [https://www.instagram.com/cor2edmeded]     This content is intended for healthcare professionals only.  This podcast is supported by an Independent Education Grant from Takeda     This podcast is developed by cor2ed.com  Published May 2026    Subscribe to this channel to stay up to date with new Independent Education programmes as they’re released.

Understanding EP-NEC: Through Diagnosis, Treatment, and Support

In this podcast, Medical Oncologist, Dr Aman Chauhan and Neuroendocrine Patient Advocate, Susan Meckler-Plummer, discuss extrapulmonary high-grade neuroendocrine carcinomas (EP-NECs), a rare, aggressive, and often misunderstood subset of neuroendocrine cancers.   They clarify the crucial differences between NETs and NECs, explain why accurate classification and rapid treatment are essential, and highlight current and emerging therapeutic approaches, including DLL3-targeted therapies.   Through both HCP and patient-centred perspectives, the conversation addresses real-world challenges, the importance of support networks, and the growing sense of hope for people affected by this difficult disease.  Key clinical takeaways:   * Unlike NETs, EP-NECs are poorly differentiated, high-grade, aggressive, and fast-growing cancers, with a need for more effective treatments * While there remains a medical need for effective treatment of EP-NECs, clinical studies with DLL3-targeted agents show promise  * Accessing a support network can provide emotional reinforcement and information for patients and their care partners    You can also watch a video of the experts in conversation and download the accompanying transcript on our website: https://cor2ed.com/net-connect/programmes/ep-nec-diagnosis-treatment-support/?media=0  Or watch on YouTube: https://youtu.be/nfng2s3jSw8  Follow us on social media:  LinkedIn: https://www.linkedin.com/company/cor2ed-net-connect/?viewAsMember=true [https://www.linkedin.com/company/cor2ed-net-connect/?viewAsMember=true]   X:https://x.com/net_connectinfo [https://x.com/net_connectinfo]  This podcast is supported by an Independent Education Grant from Boehringer Ingelheim. This podcast is developed by cor2ed.com  Published in November 2025    Subscribe to this channel to stay up to date with new Independent Education programmes as they’re released.

Exploring the role of targeted radiopharmaceutical treatment in NETs

How are advances in radioligand therapy shaping the treatment of neuroendocrine tumours, and what does recent trial evidence tell us? What are the key considerations for patient selection, sequencing, and safety?  In this podcast moderated by the Oncology Brothers, Drs Rohit and Rahul Gosain are joined by medical oncologist Dr Heloisa Soares and nuclear medicine physician Prof. Ken Herrmann to explore the evolving role of targeted radioligand therapy in NETs.  As the conversation unfolds, the experts discuss evidence from recent studies, including the COMPETE trial, share practical insights on multidisciplinary decision-making, and highlight future directions such as the potential for first-line treatment with radioligand therapy.  Key clinical takeaways:   * Pair the right patient with the right treatment using an understanding of the key clinical studies  * The role of PRRT as a very effective 2nd-line treatment was consolidated by the COMPETE study data   * The best outcomes are achieved through multi-disciplinary collaborations that include the patient     Listen to the audio version of this conversation on Spotify, Apple, or on the COR2ED website, where you can also download the accompanying transcript and infographic: https://cor2ed.com/net-connect/programmes/targeted-radiopharmaceutical-treatment-nets/?media=0 [https://cor2ed.com/net-connect/programmes/targeted-radiopharmaceutical-treatment-nets/?media=0]   Follow us on social media:  LinkedIn: https://www.linkedin.com/company/cor2ed-net-connect/?viewAsMember=true [https://www.linkedin.com/company/cor2ed-net-connect/?viewAsMember=true]   X: https://x.com/net_connectinfo [https://x.com/net_connectinfo]   This content is intended for healthcare professionals only.  This podcast is supported by an Independent Education Grant from ITM.   This podcast is developed by cor2ed.com    Published September 2025

Short-stature conditions, including SPIGFD

Hear from Dr Andrew Dauber and Dr Marta Ramón Krauel in this podcast as they explore short stature conditions, including SPIGFD (severe primary IGF-1 deficiency) / growth hormone resistance, through real patient cases and clinical insights. While growth hormone deficiency is a well-known cause of short stature, this discussion covers patient cases where growth hormone levels are normal or even elevated, yet IGF-1 levels remain low—a hallmark of primary IGF-1 deficiency.  Gain valuable knowledge on identifying and managing this rare condition, with practical takeaways for clinical practice.   Key clinical takeaways:   * Growth hormone resistance can range from severe IGF-1 deficiency, like in classic Laron syndrome, to milder mutations in the same receptor. There can also be mutations in other parts of the signalling pathway  * Patients with SPIGFD have low IGF-1 levels resulting from growth hormone resistance or insensitivity, rather than deficiency, as they produce sufficient or even high levels of growth hormone without secondary causes of growth failure  * Laron syndrome, a classic form of SPIGFD, is characterised by severe short stature and distinct features such as spontaneous hypoglycemia, mid-face hypoplasia, frontal bossing, a high-pitched voice, and underdeveloped genitalia in boys  * Early and accurate diagnosis of SPIGFD is crucial for effective management, as growth hormone therapy is ineffective for these patients; recognising the distinction between severe and less severe IGF-1 deficiency, as well as primary versus secondary causes, ensures appropriate treatment to ensure optimal outcomes  * Recombinant IGF-1 therapy is indicated for patients with SPIGFD. Healthcare professionals must monitor for adverse events such as hypoglycemia and potential malignancies    Watch the experts in conversation in the full video or get the 5-minute highlights: https://cor2ed.com/pe-connect/programmes/short-stature-spigfd/ [https://cor2ed.com/pe-connect/programmes/short-stature-spigfd/]     Follow us on social media:  LinkedIn: https://www.linkedin.com/showcase/pe-connect/ [https://www.linkedin.com/showcase/pe-connect/] X: https://x.com/peconnectinfo [https://x.com/peconnectinfo]   This content is intended for healthcare professionals only.   The medical experts in this podcast are expressing their own views and not those of COR2ED, Supporters or their institution.  This podcast is supported by an Independent Education Grant from Ipsen  This podcast is developed by cor2ed.com  Published December 2024    Subscribe to this channel to stay up to date with new Independent Education programmes as they’re released.

Neuroendocrine tumours (NETs) – Shared decision-making. Part 2: Individualising treatment decisions

In this second episode of a two-part podcast series focusing on neuroendocrine tumours (NETs), gastroenterologist Dr Mohid S Khan and NET patient Sally Jenkins explore shared decision-making, between patient and physician.     The speakers discuss different treatment settings, including home vs hospital-based injections, as well as the importance of individualising treatment decisions, as there is often no standard therapy for patients with NETs. Finally, they look at some of the studies reporting patient preferences, and how the data can be used to inform patient decisions.    Prefer to watch and listen?  Watch the video and download the full transcript on the COR2ED website https://cor2ed.com/net-connect/programmes/net-shared-decision-making/?media=1 [https://cor2ed.com/net-connect/programmes/net-shared-decision-making/?media=1]  Watch the full video on YouTube https://youtu.be/2JYuPhc2jHg [https://youtu.be/2JYuPhc2jHg]  Watch the highlights video on YouTube – https://www.youtube.com/watch?v=pPMVLjPLv8k [https://www.youtube.com/watch?v=pPMVLjPLv8k]     Follow us on social media  LinkedIn: https://www.linkedin.com/company/cor2ed-net-connect [https://www.linkedin.com/company/cor2ed-net-connect]   X: https://x.com/net_connectinfo [https://x.com/net_connectinfo]     This content is intended for healthcare professionals only.  The medical experts in this podcast are expressing their own views and not those of COR2ED, Supporters or their institution.  This podcast is supported by an Independent Medical Education Grant from Ipsen  This podcast is developed by cor2ed.com  Published July 2024