Compact, hands-on CME on gene & cell therapies for rare diseases—a concise introduction for primary care providers. Gene and cell therapy is no longer medicine of the future—it has become a clinical reality. For general practitioners, foundational knowledge in this area is essential. This is where the new course series "Rare Diseases in Practice" comes in. Gene- and cell-based therapies are rapidly evolving from experimental concepts to clinical reality. Especially in the field of rare diseases, these approaches are opening up causal treatment options for the first time, where previously only symptomatic therapies were available. The live talk is a joint event by the Eva Luise and Horst Köhler Foundation, the National Strategy for Gene and Cell-Based Therapy, and the German Society for Gene Therapy e.V. ... Save the date: The live talk on October 13, 2025, will take place from 6:00 to 7:30 p.m. as an interactive live webinar on the STREAMED UP video platform. On… ... Neuronal ceroid lipofuscinoses (NCL) are the most common types of neurodegenerative diseases in childhood and adolescence. NCL specialist Dr. med. Angela Schulz from UKE Hamburg-Eppendorf provides an overview of the… ... On Rare Disease Day, February 28, 2025, our live and interactive training series for outpatient physicians will begin. Annette Grüters-Kieslich, Chair of the Executive Board of the Eva Luise and Horst Köhler Foundation, three distinguished experts presented and explained the current state of these therapies on February 24, 2026, in a live talk on the STREAMED UP platform: Prof. Dr. Hildegard Büning, Hannover Medical School: “Gene- and cell-based therapies – when concepts become clinical reality”

Original title: “Gene & Cell Therapy for Rare Diseases | CME Compact”

Although the first cases were described almost 200 years ago,7 there are still no proven disease-modifying therapy for any form of neuronal ceroid lipofuscinosis. Since 1977, there have been at least 5 completed prospective parallel group clinical trials and 19 case reports, series, or open-label studies addressing treatments for infantile, late-infantile, and juvenile neuronal ceroid lipofuscinosis (Table 1).

Original title: “Clinical Trials in Rare Disease: Challenges and Opportunities - PMC”