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Pompe disease: Expanding manifestations and evolving management
Watch leading experts discuss recent developments in Pompe disease, including refined understanding of phenotypes and multisystem clinical manifestations emerging in the era of enzyme replacement therapy (ERT). The impact on paediatric patients and their caregivers is also explored, alongside approaches to further optimize multidisciplinary management of Pompe disease.
- Discuss how successful ERT has led to the new phenotype of infantile-onset Pompe disease
- Describe the multisystem disorders of late-onset Pompe disease
- Explain the approaches that are being used to address the unmet treatment needs for Pompe disease
How do we personalize treatment of multiple sclerosis in the light of recent advances?
Watch leading experts involved in the care of patients with multiple sclerosis discuss how recent advances in understanding of MS pathophysiology may drive treatment choices, now and in the future.
- Recall the disease mechanisms contributing to the progression of MS
- Interpret the role of induction therapy in real-world clinical practice
- Recognize the importance of BTK as a potential future treatment target in MS
Presentation, diagnosis and management of SMA: Multidisciplinary perspectives
Watch leading experts discuss the diagnosis and management of spinal muscular atrophy (SMA), including causes of diagnostic delay, emerging treatment approaches and the crucial role of the multidisciplinary team in the management of this disease.
- Identify patients at risk of spinal muscular atrophy (SMA) through genetic screening and recognize early symptoms in the disease course
- Describe key trial and registry data (including antisense oligonucleotide and gene therapy) supporting improved treatment practice and outcomes in SMA patients
- Discuss roles within the multidisciplinary team (MDT) and impact of a standardized treatment approach across the MDT including utilization of guidelines and treatment innovations for SMA patients in clinical practic
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