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Science Hub

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Date and time:

07.02.24 at 17:30-18:45 CET



Welcome and introduction - (17:30 – 17:35 CET)

Improvement and remaining gaps in Haemophilia B treatment - (17:35 – 17:50 CET)

Up to 8 years of safety and efficacy outcomes from children trials (PUPs and PTPs) - (17:50 – 18:10 CET)

Neurodevelopment in children on N9-GP prophylaxis - (18:10 – 18:35 CET)

Q&A and meeting closure - (18:35 – 18:45 CET)


Helle Holst (Denmark)

Senior Director, Medical & Science RED, LCM and Cell Therapies, Novo Nordisk

Helle Holst, MD, PhD, MSc, joined Novo Nordisk A/S in 2020 as the SR. Director Rare Endocrine Disorders, LCM & Cell Therapies M&S. She is a results-driven leader who prioritizes value creation for patients and has a strong focus on building high-performing teams. With over 14 years of experience in academia and the pharmaceutical industry, Helle is an experienced Leader in Clinical Development. As a board-certified Clinical Pharmacologist and with an MSC in Science in Paediatric Medicines Development and Evaluation, she has successfully led innovative clinical programs in rare endocrine and haemophilia disorders, cell therapy, and a spectrum of neurological disorders.


In addition to her technical expertise, Helle is committed to promoting diversity and equity in her leadership role. She actively seeks out diverse perspectives and ensures inclusive communication and collaboration within the team. Additionally, she prioritizes the professional development of individual team members, including those from underrepresented groups, to foster a culture of growth and learning.


Helle has contributed to RA/HA documents across therapeutic areas and has a strong knowledge of benefit-risk evaluation and proactive risk management strategies in the pharmaceutical industry. Her expertise extends to healthcare committees and professional societies, where she has served as Secretary of The Reimbursement Committee for the Danish Health Authorities and is a member of the CD-PH/PaedForm Group of Experts appointed by the European Pharmacopoeia Commission and deputy chair for EFPIA for the ICH discussion group for ATMP. Helle was previously the Vice President of the Nordic paediatric research network, NORDICPEDMED.


With a PhD in neuroinflammation and hypersensitivity, Helle has authored 36+ publications and has served as a reviewer for seven high-impact journals, demonstrating her extensive knowledge in the field of pharmacology and paediatric medicine.

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