Excellence in Pediatrics is a non-profit
global network dedicated to advancing
pediatric healthcare.
Rue des Vignerons 18, 1110 Morges 1 (VD), Switzerland
A webinar series dedicated to shining a light on alpha-mannosidosis, an ultra-rare genetic disorder caused by deficiency of the lysosomal enzyme alpha-mannosidase. The symptoms, severity, and progression of alpha-mannosidosis vary between individuals, which presents challenges for both diagnosis and management. The first best-practice recommendations for monitoring and care coordination of individuals with alpha-mannosidosis were recently published. Join our expert panel as they explore how these recommendations translate into clinical practice, offering insights from their own experiences in diagnosing alpha-mannosidosis and delivering personalized, multidisciplinary care. The webinars feature case study examples, panel discussions, and audience Q&A.
To consider approaches to monitoring treatment-related outcomes, including enzyme replacement therapy, hematopoietic stem cell transplant, and supportive care, and how timely access to treatment may improve patient outcomes.To demonstrate the need for a multidisciplinary approach to care and how this may be tailored to the individual. To discuss challenges and hopes for the future of care in alpha-mannosidosis.