A Trusted Bridge Between Industry and the LGMD Community
The LGMD CAB serves as a strategic partner for organisations developing therapies and conducting research in Limb Girdle Muscular Dystrophy. We provide structured, experience-based feedback that helps align scientific goals with the real-world needs of patients and families living with LGMD.
Our Approach
Every collaboration begins with listening. Through confidential discussions and structured consultations, we identify how research or development plans can better reflect patient priorities and practical realities.Our members bring both personal experience and professional backgrounds in advocacy, clinical research, and policy — creating a well-rounded advisory perspective.
We work according to principles of:
• Transparency – open dialogue with clear objectives and outcomes.
• Independence – unbiased advice that serves both community and scientific integrity.
• Confidentiality – protecting sensitive information while maintaining trust.
• Respect – valuing each stakeholder’s expertise and role.
What Collaboration Looks Like
Engagement with the LGMD CAB is flexible and tailored to each partner’s needs.
Common formats include:
• Advisory Meetings – one-time or recurring discussions on trial design, inclusion criteria, or protocol feasibility.
• Workshops – deeper exploration of patient experience, trial logistics, or communication materials.
• Document Reviews – written feedback on study protocols, informed consent forms, or patient materials.
• Long-Term Partnerships – ongoing collaboration throughout a research or development program.
The Process
1. Initial Contact – The partner outlines their project goals and objectives.
2. Scoping & Planning – We agree on topics, format, and confidentiality terms.
3. Consultation – The CAB meets, reviews, and provides collective feedback.
4. Summary Report – Partners receive structured, actionable insights.
5. Follow-Up – Continued dialogue or review as projects evolve.
The Value We Bring
• Improve trial design and participant retention.
• Anticipate practical challenges in implementation.
• Demonstrate authentic patient engagement to regulators and ethics boards.