Learn from the Experts, On Demand

Comprehensive overview of the current and emerging therapies for MPS and the role of multidisciplinary care.

Discussion on the importance of early MPS diagnosis, combining genetics and neuropsychology to improve outcomes.

Discover a detailed case-based discussion on the early detection of MPS, highlighting how variability in presentation can lead to treatment delays.

An in-depth walkthrough of the AM patient journey, from diagnosis to lifelong management, emphasizing the importance of early recognition.

A detailed overview of AM, focusing on the importance of early diagnosis, therapeutic strategies, and multidisciplinary management.

Clinical insights on diagnosing AM, highlighting outlines common clinical signs, phenotype variability and diagnostic challenges.

Discussion on early AM signs, highlighting diagnostic overlap with MPS & other lysosomal disorders.

Review on MLD progression, and emerging treatments, focusing on gene therapy and real-life case studies.

A comprehensive update on newborn screening for MLD, focusing on the importance of early identification and the implications for timely treatment.

Explore the challenges of recognizing early clinical manifestations of MLD and the importance of prompt referral and diagnosis.

Explore the nature of disease and the long-term care and management of individuals diagnosed with AM.

Hearing loss, developmental delay, and behavioral changes are key AM red flags—age-specific algorithms aim to shorten the years-long diagnostic odyssey.

How diagnostic delays, multidisciplinary care, surgical risks, and psychosocial strain shape everyday life for patients with MPS and their families.

Este webinar aborda el diagnóstico y manejo temprano de las mucopolisacaridosis (MPS), proporcionando herramientas clave para mejorar su detección y tratamiento

MPS I spectrum—from Hurler to Scheie—shown through cases of delayed diagnosis, urging pediatricians to spot symptom clusters early and pursue ERT sooner.

Explore how GAG accumulation drives disease, why HSCT and ERT have different strengths, and why newborn screening enables earlier, better outcomes.

Este webinar aborda el diagnóstico y manejo temprano de las mucopolisacaridosis (MPS), proporcionando estrategias clave para mejorar la identificación y tratami

Multiple unrelated symptoms across systems should raise MPS suspicion—key radiological clues, enzyme testing, and early ERT dramatically improve outcomes.

Microglial dysfunction drives MLD neurodegeneration. Explore HSCT and gene therapy strategies for faster CNS engraftment when given pre-symptomatically.

Clinical spectrum and progression, with focus on gene therapy outcomes showing early treatment dramatically improves motor and cognitive results.