FSHD Seminar – Community Information Session

5 March 2024

Presented by Dr. Cullen O’Gorman – Specialist Neurologist and Clinical Neurophysiologist, Brisbane Clinical Neuroscience Centre

This presentation provides an overview of FSHD clinical trials, including how they work, what participants can expect, and key considerations before joining. Clinical trials are scientific studies, not treatments, and are strictly monitored with ethical approval. Patients are encouraged to understand trial duration, procedures, risks, benefits, costs, and who is conducting the study before deciding to participate.

The talk also highlights current research targets, focusing on the DUX4 protein and muscle regeneration. Ongoing trials include therapies such as monoclonal antibodies, IL-6 antagonists, and agents that reduce DUX4 expression, while completed studies have explored creatine and Losmapimod. Trial readiness, including stable health, genetic confirmation, and the ability to commit time to visits and assessments, is emphasised.

Finally, the presentation covers practical guidance for participants, from managing health and exercise to coordinating care with multidisciplinary teams. It outlines what happens during the trial, including assessments and monitoring, as well as post-trial considerations such as withdrawal, ongoing access to therapy, and access to results. The goal is to help patients make informed decisions and support faster, more effective trial completion.

Watch the video here:

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