Key Takeaways: Neuromuscular Information and Research Day 2025

This year’s Neuromuscular Information and Research Day brought together experts, researchers, families with lived experience and community voices to share practical strategies for living well with a neuromuscular condition. From advances in clinical research and safe exercise tips to personal stories of resilience and advocacy, the day was filled with insights and connection.

Watch the full recording here for the complete experience or read on for the key highlights and takeaways from each session.

Australian Neuromuscular Muscle Disease Registry (ANMDR) Update

Session by Dr Eppie Yiu

What is the ANMDR?

• ANMDR is part of the TREAT-NMD Alliance, a global network of neuromuscular disorder registries. The Alliance collects, shares and updates data to help advance treatments, diagnosis and care for people living with a neuromuscular condition.
• Collects and maintains clinical, genetic and demographic data on children, adults and people who have passed away with genetically confirmed neuromuscular conditions.
• The data shows what’s happening now and also tracks changes over time, enabling ongoing monitoring.
• It requires active collaboration with patient groups, researchers and industry.

Benefits

• Using the same high-quality data everywhere makes research stronger and care better.
• The registry enables clinical trial sponsors and researchers to quickly identify eligible participants for studies, increasing the ability to conduct clinical trials in Australia. 
• Shows how many people in Australia are affected by neuromuscular conditions and where they are found.

Challenges

• Sustainability of funding, patient engagement, data quality and standardisation, and resource constraints.
• Registry is based in Victoria, so there is a need for representation in other states.
• The database is still being built out.

Key Takeaway

The ANMDR is building a high-quality data registry that attracts research and supports new treatments.

Lived Experience Panel on Getting the Support You Need

Session by Joe Iaquinto, Chris Carrero and Bridget Larsen

Joe’s Story

Joe’s openness about his needs shows the importance of communicating what works for you, as every experience with neuromuscular conditions is unique.

Bridget’s Perspective

As a mother, Bridget focuses on supporting rather than controlling her child, using creative family rituals like an annual ‘Razzie Awards’ to celebrate both highs and lows.

Plus! Bridget created a practical community resource to help others navigate the steps to getting support. It’s full of quick, experience-based tips that make the process feel more manageable and easier to follow.

Download the resource

Chris’s Perspective

Chris spoke about accepting the full scope of disability while pursuing independence and still doing what you love. He highlighted how MDNSW’s supports, including camps, meetups for dads and community activities, are safe, fun and empowering spaces.

Lived Experience Strategies

• Adjust your approach. Try something new!
• Find and talk to the right person to manage administration tasks
• Time your communication well
• Communicate openly and often

Support & Advocacy

• Professional support is important, don’t just rely on family
• Create safe spaces for peer support and connection
• Share information efficiently (e.g., 1-pager for specialists)
• Think ahead: What equipment is needed now and what will be needed in the future?
• Advocate with care teams to align with family needs

Challenges & Barriers

• Emotional strain, frustration and limited time/energy.
• Barriers from the providers side include arrogance, resource constraints and ignorance.

Key Takeaways

• Support networks matter. Patients, families and peers all play a role.
• Focus on what you can do, not just limitations.
• Recognise this as a shared journey, not one carried alone.
• Don’t be stoic. Please ask for help for yourself or your child.
• Proactive communication and vulnerability are powerful.

Keeping Muscle Strength vs Managing Fatigue

Session by Dr Veena Raykar and Tess Southcombe

Safe Exercise & Self-Management Tips

• Practice daily diaphragmatic or deep belly breathing. It improves breathing efficiency and reduces fatigue.
• Always include a warm-up and cool-down. Stretch after each session to prevent stiffness and injury.
• Regularly ask, “How hard does this feel right now?” Avoid pushing too far.
• Stay well hydrated throughout.

Fatigue Monitoring

• Use the BORG Scale (6 = no exertion; 20 = maximal effort) to stay in the safe zone.
• If your muscles are sore for more than a day, you’ve done too much.

Exercise Guidelines

• Aim for 10–20 minutes per session. Consistency matters more than intensity.
• Take rest days. Recovery is essential, not a setback.
• Exercises can be spread across the day if needed.
• Plan workouts when energy is highest (often midday).
• Focus on low-impact, energy-efficient activities.

Muscle Care

• Be mindful: muscle membranes are fragile and prone to inflammation.
• Use eccentric exercises (e.g., walking downhill, lowering weights) with caution.

Staying Motivated

• Set small, realistic goals.
• Use activity diaries or home exercise charts to track progress.
• Ask family or carers to help you stay on track.

Key Takeaways

• Prioritise breathing, pacing and recovery.
• Consistency over intensity for long-term benefits.
• Advocating for yourself, motivation and support are crucial for safe, sustainable exercise.

Clinical Research and Treatments Update

Session by Dr Dennis Yeow and Dr Aicee Calma

Evidence-Based Treatments & Research

• Why evidence matters: It’s important to use drugs and interventions supported by phase three and four clinical trials, where safety and effectiveness are established.
• Current reality: Most drugs for neuromuscular conditions are still in early-stage trials, not yet at phase three or four.
• Balance of risks and benefits: Patients and families need to understand both the potential benefits and the possible side effects.

Research Process

• Research takes time and can feel slow, but it’s designed to keep people safe. Ongoing research is promising – many studies are underway worldwide.

How to Engage

• Get involved with clinical studies if interested.
• Use reliable resources to stay updated on progress for specific diseases or drugs.
• If you’re wondering what clinical trials are happening for specific conditions around the world, this website can be useful: https://clinicaltrials.gov

Key Takeaways

• Evidence from late-stage trials is essential for safe treatments.
• Progress may be slow, but it is purposeful.
• Stay informed and consider participating in research.

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