In this free webinar, learn about some of the recent scientific advances in the field of pediatrics and rare disease drug development. The featured speakers will discuss the obstacles, from ethical considerations to long-term follow-up, faced in cell and gene therapy (CGT) trials for neonates and young children with rare diseases. Attendees will learn about the need to include the voice of the patients and families when planning and conducting these trials. The speakers will share a practical example on the education of a pediatric population participating in a CGT trial.
TORONTO, Feb. 3, 2025 /PRNewswire-PRWeb/ -- Ground-breaking scientific advances have been made in the field of cell and gene therapy (CGT) in the past few decades, which is evidenced by an increasing number of CGT-based clinical trials.
While there are more than 8000 known rare diseases, treatment options are limited and only available for a few of them. As about 80 percent of rare diseases are monogenic diseases and many of them manifest during childhood, CGT is a potentially curative treatment for children and their families living with a rare life-altering disease.
For rare diseases presenting early in childhood and for which no adult disease exists due to rapid progression, young children and neonates will be increasingly involved in early CGT trials and even in first-in-human trials. There is also an urgent need in treating these children early as recent trials have shown that early treatment can improve outcomes.
These highly complex trials hold a myriad of challenges ranging from product development (choice of vector, determination of dose and administration of product) to conduct of the trial (ethical considerations, safety monitoring of potential toxicities and treatment of adverse events) and the long-term follow-up required by regulators.
This webinar aims to highlight some of the recent scientific developments relevant to studies in young pediatric patients with rare diseases. The expert speakers will further discuss some of the operational challenges that they have encountered so far when conducting these studies.
They will also focus on the patient voice and caregiver by exploring their attitudes towards CGT and discuss incorporating these insights into the training of pediatric patients on CGT. The expert speakers will be joined by a rare disease patient and his mother giving their very own perspectives on CGT trials and available CGT treatments.
Register for this webinar to gain insights on improving outcomes through early treatment and addressing regulatory requirements for pediatric rare diseases.
Join Julie Maher (moderator), Senior Director, Rare Diseases, Advanced Therapies and Pediatrics Team (RAPT), ; Dr. Susanne Schmidt, MD, PhD, Senior Medical Director, Rare Diseases, Advanced Therapies and Pediatrics Team (RAPT), Fortrea; Darby Thomas, PhD, Scientific Director, Rare Diseases, Advanced Therapies and Pediatrics Team (RAPT), Fortrea; Dr. Chris Evans, MD, Founder & CEO, Little Journey; Michelle Burgess and Michael, for the on Monday, February 24, 2025, at 10am EST (4pm CET/EU-Central).
For more information, or to register for this event, visit .
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