Kedrion Biopharma brought new scientific insights, expert-led discussions, and rare disease evidence generation to the 34th International Society on Thrombosis and Haemostasis (ISTH) Congress in Paris, contributing to ongoing efforts to improve outcomes for patients.
“Scientific leadership is an essential part of how we deliver for patients,” said Bob Rossilli, Chief Commercial Officer, Kedrion Biopharma. “By investing in clinical research, collaborating with experts, and sharing new evidence at forums like ISTH, we’re helping advance the understanding of rare diseases while supporting better care for patients around the world.”
At a time when earlier diagnosis and more personalized care remain critical challenges across rare diseases, Kedrion is fostering scientific dialogue designed to improve disease recognition, clinical decision-making, and patient outcomes. Through scientific exchange, publication, and collaboration with the medical community, Kedrion continues to invest in generating evidence that helps address persistent gaps in diagnosis and disease management for patients living with rare conditions.
“At Kedrion, advancing care for patients with rare diseases begins with generating evidence that may help clinicians make more informed decisions,” said Manuela Scarpellini, Global Medical Therapeutic Area Lead for Hematology at Kedrion. “Through our clinical presentations and scientific exchange at ISTH, we are contributing to a deeper understanding of patient experiences, disease burden, and opportunities to improve outcomes in areas where significant unmet need remains.”
Medical posters will focus on the assessment and clinical management of two ultra-rare diseases:
- “A Sub-analysis of Historical Symptoms and Medical Interventions in Subjects with Type 1 Plasminogen Deficiency (PLGD-1) Prior to Plasminogen Replacement Therapy,” a study of PLGD-1 early stages, from symptom onset to diagnosis.
- “Gender Differences in Disease Burden and Quality of Life in Hereditary Factor X Deficiency: Findings from the Hereditary Factor X Deficiency in America Survey,” exploring how to assess disease burden and patient-level QOL based on direct or proxy assessment of hereditary Factor X deficiency (HFXD) patients stratified by gender.
“Assessing the time from symptom onset to diagnosis of PLGD-1 allows us to better understand the patient journey,” said Karen Thibaudeau, PhD, Global Medical Therapeutic Area Lead for Plasminogen Deficiency at Kedrion. “Patients with PLGD-1 often face a lengthy period between their first symptoms to a confirmed diagnosis. By better understanding where delays occur, we can generate evidence that supports earlier recognition, faster diagnosis, and appropriate care.”
Together, these presentations reflect Kedrion’s ongoing commitment to generating evidence to address significant unmet medical needs in Rare Diseases.
Kedrion also hosted a symposium focused on clinical decision-making in HFXD, with particular attention to women, girls, and pediatric patients. The discussions emphasized the need to improve recognition of HFXD in females, whose symptoms have historically been underrecognized or normalized across generations, contributing to delays in diagnosis and appropriate care.
“Because HFXD presents unique challenges in pediatric patients – due to developmental vulnerability, unique symptom patterns, and high risk of severe bleeding – tailored care is critical,” said Beatrice Nolan, MD, BCh, BAO, Hematologist at Children’s Health Ireland at Crumlin’s Department of Pediatric Hematology. “In children with severe HFXD, bleeding manifestations often occur early in life, with serious clinical manifestations such as umbilical cord bleeding and intracranial hemorrhage, frequently observed in neonates and infants. These findings highlight the importance of early diagnosis and appropriate management informed by a thorough understanding of the specific needs and clinical presentation of pediatric patients.”
Kedrion also convened educational sessions focused on often-overlooked challenges of plasminogen deficiency, exploring disease recognition, severe neonatal presentations, and reproductive and gynecologic manifestations that can significantly affect patient outcomes and quality of life.
“Earlier recognition of plasminogen deficiency can help reduce the burden of delayed diagnosis and improve patient management,” said Professor Per Morten Sandset, MD, Senior Consultant in hematology at the Oslo University Hospital and a professor in thrombosis research at the University of Oslo. “As a multisystem disease that can affect patients from early childhood through adulthood, plasminogen deficiency requires greater awareness among clinicians so patients can receive appropriate care as early as possible.”
Kedrion’s abstract presentations took place today during ISTH 2026 in Paris.