Webinars

Learn from the Experts, On Demand

Explore our full library of expert-led webinars on rare pediatric diseases. Watch real clinical cases, diagnostic strategies, and multidisciplinary discussions at your own pace and build the confidence to recognise rare conditions sooner.

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Distinguishing CLN2 (Batten Disease): Navigating the differential diagnosis and referral pathways
Pasquale Striano; Marina Trivisano

Practical guidance on distinguishing CLN2 from similar conditions, recognizing early warning signs, and making timely referrals.

60 minutes
English
Batten (CLN2)
Progressive Neurodegeneration in Children: Are We Diagnosing Too Late?
Nicole Wolf; Serena Gasperini; Samuel Groschel

Practical insights from MLD and MPS III case studies to support earlier recognition and more confident clinical decision-making.

60 minutes
English
Leukodystrophy (MLD)
Recognizing the Early Signs of CLN2 (Batten Disease)
Paul Gissen

Explore the early symptom patterns of CLN2, follow a real clinical case, and gain confidence in timely referral decisions.

40 minutes
English
Batten (CLN2)
Metachromatic Leukodystrophy: Newborn Screening and Sibling Testing - Family and Expert Perspectives
Andreas Oberg; Erik Eklund; Ally Wallace

Explore the clinical and human impact of early diagnosis through family experiences and real-world implementation of newborn screening.

60 minutes
English
Leukodystrophy (MLD)
Understanding Immune System Dysfunction and Recurring Infections in Alpha-mannosidosis
Susanna Esposito; Giacomo Biasucci

Learn how immune system monitoring can improve Alpha-Mannosidosis diagnosis and care in this expert-led pediatric webinar.

60 minutes
English
Alpha-mannosidosis (AM)
Optimising Outcomes & Exploring Psychological Aspects of Alpha-mannosidosis
Julia Hennermann; Sarah Haupenthal

Explore enzyme replacement therapy and psychological support to optimise outcomes in Alpha-Mannosidosis patients.

60 minutes
English
Alpha-mannosidosis (AM)
Lessons from Germany and Austria's Successful MPS and Alpha-mannosidosis Screening Initiative
Christina Lampe; Florian Lagler

Discover how German and Austrian screening initiatives improved early diagnosis of MPS and Alpha-Mannosidosis.

60 minutes
English
Alpha-mannosidosis (AM)
Mucopolysaccharidosis Misdiagnosis: A Cross-Disciplinary Approach
Barbara K Burton; Klane White; David Molter; Elizabeth Braunlin

Follow multidisciplinary case discussions that highlight overlooked clues and support more accurate, timely referrals.

60 minutes
English
Mucopolysaccharidosis (MPS)
Mucopolysaccharidosis: Early Recognition and Management in Primary Care
Barbara K Burton; Joseph Muenzer; Kristin Mckay

Combine practical diagnostic guidance with patient stories to better recognize rare disease in everyday clinical practice.

60 minutes
English
Mucopolysaccharidosis (MPS)
Detección Temprana de la Leucodistrofia Metacromática
Angels Garcia Cazorla

Este webinar aborda la identificación y manejo temprano de la MLD, brindando herramientas para mejorar el diagnóstico y la atención de pacientes pediátricos.

30 minutes
Español
Leukodystrophy (MLD)
Rare Diseases – Current And Upcoming Treatments
Dipak Ram; Mireia Del Toro

From gene therapy to supportive care, discover the latest advances shaping treatment for children with MLD.

60 minutes
English
Leukodystrophy (MLD)
Understanding The Diagnostic Journey Of Metachromatic Leukodystrophy
Paul Gissen; Anna Ardissone

Explore real-world cases that reveal missed opportunities, practical diagnostic strategies, and the value of timely referral.

60 minutes
English
Leukodystrophy (MLD)
Spotting The Early Signs Of Metachromatic Leukodystrophy (MLD)
Francesca Fumagalli; Alejandra Darling

Early signs and diagnostic clues of MLD and the importance of early recognition and timely diagnosis to improve outcomes.

60 minutes
English
Leukodystrophy (MLD)
Shine A Light On Alpha-mannosidosis (AM): A Brighter Future
Christina Lampe; Karolina Stepien; Barbara K Burton

Εxplore the future of ΑΜ care, focusing on treatment follow-up, implementing a multidisciplinary approach, and preparing for lifelong patient needs.

60 minutes
English
Alpha-mannosidosis (AM)
Shine A Light On Alpha-mannosidosis. Illuminating The Patient Voice
Nathalie Guffon; Can Ficicioglu; Christina Lampe

Exploring early recognition, monitoring, and multidisciplinary care in alpha-mannosidosis to improve outcomes and quality of life for patients.

60 minutes
English
Alpha-mannosidosis (AM)
Shine A Light On Alpha-mannosidosis (AM): Providing Personalized Care
Nathalie Guffon; Martin Magner; Monica Lopez Rodriguez

Recognizing early signs, improving diagnosis, and applying personalised care strategies through expert guidance and multidisciplinary approaches.

60 minutes
English
Alpha-mannosidosis (AM)
Hearing Impairment As An Alpha-mannosidosis Sign In Children
Nathalie Guffon

Explore detailed insights into ENT manifestations, the diagnostic pathway, and distinguishing features of AM compared to other lysosomal storage disorders.

60 minutes
English
Alpha-mannosidosis (AM)
Understanding Alpha-mannosidosis Patients’ Needs
Monica Lopez Rodriguez; Sophie Thomas

Exploring the impact of alpha-mannosidosis on patients and families, with a focus on quality of life, caregiver support, and holistic care.

60 minutes
English
Alpha-mannosidosis (AM)
Cognitive Development As An Early Sign Of Alpha-mannosidosis
Martin Magner

Understanding alpha-mannosidosis, its clinical features, diagnostic challenges, and the importance of early recognition to improve patient outcomes.

30 minutes
English
Alpha-mannosidosis (AM)
The Multidisciplinary Team For Alpha-mannosidosis (AM)
Mercedes Gil Campos

Review of treatment options & multidisciplinary strategies for Alpha-mannosidosis, backed by consensus-based methodologies.

30 minutes
English
Alpha-mannosidosis (AM)
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