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    Accessing Emerging Treatments for Childhood Dementia


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    Event description

    With the limited number of treatment options available for childhood dementia disorders, pathways such as the special access scheme (SAS) are an important means to accessing emerging treatments. This webinar will cover a clinical case study that used the SAS to obtain an experimental treatment for a childhood dementia patient, and include perspectives from the involved clinician, pharmaceutical company, and patient’s family.

    Speakers: 

    Vicki Wong (Co-founder, Parasail) will present:

    “Antibody Enzyme Targeting for Degradation of Intracellular Aggregates: Special Access Treatment of VAL-1221 for Lafora Disease”

    Dr Alan McDougall (Head of Neurology, Liverpool Hospital, South Western Sydney Local Health District) will present:

    “Importation and Use of Medication VAL1221 for Lafora Body Disease - Challenges of Obtaining a New and Unregistered Treatment”

    Niki Markou (Parent and Advocate and Director Chelsea’s Hope Lafora Children Research Fund) will present:

    “The Family Perspective”


    About the Childhood Dementia Webinar Series: The Childhood Dementia webinar series aims to cover a range of topics to enhance engagement and foster collaborations amongst researchers, clinicians, and industry experts to accelerate research and development of treatments for childhood dementia.



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