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AUTUMN 2026 InTouch | 19 The TREAT-NMD Conference held in Lisbon from 10th – 12th February provided an invaluable opportunity to engage with the global neuromuscular disease community, and it was an honour to represent its members at this significant international forum. Bringing together scientists, clinicians, patients, advocates, regulators, geneticists, and trainees from across the world, the conference focused on major developments in clinical trials, the management of patient expectations, and evolving regulatory frameworks that support therapeutic innovation. Throughout the event, I worked to ensure that the needs of New Zealanders living with neuromuscular conditions were clearly articulated, highlighting the limited national resources, the scarcity of local clinical trials, and the importance of building strong relationships with key stakeholders to secure New Zealand’s place in an expanding therapeutic landscape. The following report summarises the conference across its primary themes. Primary conference takeaways • Misalignment exists between investor expectations and patient expectations, with differing thresholds for what is considered a successful outcome. • Fundraising challenges vary across diseases, particularly depending on whether a “realistic saviour”or curative pathway is perceived. RESEARCH NEWS TREAT-NMD Conference 2026 With support from the Muscular Dystrophy Association of New Zealand (MDANZ), Dr Angus Lindsay, Chair of Neuromuscular Research Foundation Trust, attended the TREAT-NMD Conference to represent the interests of its members. • There is a need to support industry in better managing and communicating patient expectations. • Investment in preclinical research is essential to ensure that only the most appropriate and promising drug candidates progress to clinical trials. • It is important to consider whether slowing or attenuating disease progression is an acceptable outcome compared with achieving a complete cure (e.g., discussions within PPMD). • For rare diseases, small patient populations can limit commercial interest from companies, often ending potential investment relationships. • Placebo data from both failed and successful trials can and should be leveraged to strengthen Natural History studies. • Sponsors must establish a clear communication plan with patients at the outset of a trial, regardless of the eventual outcome. • Newborn screening programs for Duchenne muscular dystrophy and spinal muscular atrophy are currently being implemented in Australia. • The use of video-based assessments to demonstrate treatment efficacy remains a point of debate, with differing views between patients, advocates, and clinicians. • A disconnect appears to exist between drug development progress and actual treatment availability and access. • Early decision-making regarding genetic testing and treatment pathways is critically important. • The American Society of Gene and Cell Therapy has launched an initiative to repurpose drugs that were previously discontinued by pharmaceutical companies. • A clinical trial must be clearly communicated as an experimental process. • Patients must be made aware that clinical trials can fail, and this understanding should be integrated into informed consent and ongoing communication. • Clinical trials should be framed as opportunities rather than guaranteed therapeutic solutions. Biopharmaceutical updates and clinical trials Entrada Therapeutics A clinical-stage biopharmaceutical company developing intracellular therapeutics using its proprietary Endosomal Escape Vehicle (EEV™) platform, enabling delivery of RNA- and protein-based medicines for Duchenne and Myotonic Dystrophy Type 1. Entrada’s drug trial portfolio includes multiple exon-skipping therapies targeting exons 44, 45, 50, and 51, with clinical data expected by end of Q1 2026.