The Research Project "CLIMB-LMBRD2"

 

CLIMB-LMBRD2: Characterizing Longitudinal gait Impairments induced by LMBRD2 Mutations using Bioconnected sensors

 

Every child with LMBRD2 evolves differently. Some develop walking difficulties that worsen over time, others remain stable. Today, we cannot predict who will be affected or how quickly symptoms will progress.
 

The problem? Without knowing the natural history of the disease, testing treatments becomes impossible. How can we tell if a medication works if we don't know how the disease would have evolved without it?

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Our Solution: Medical-Grade Wearable Technology

Rather than relying solely on occasional hospital assessments (which capture only snapshots and don't reflect daily life), we're using Syde®, a regulatory-qualified wearable sensor platform developed by SYSNAV specifically for rare disease clinical trials.

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Why Syde® is revolutionary:

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• First digital endpoint qualified by the European Medicines Agency (EMA) for measuring treatment efficacy

• 97% stride detection accuracy with centimetric precision

• Proven in clinical trials for Duchenne muscular dystrophy

• >90% patient compliance across all age groups

• Designed in collaboration with patient associations

 

How it works:

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1. Two small wearable sensors (ankle and wrist) worn naturally during daily activities

2. Continuous, passive data collection as children go about their normal lives

3. Automatic nightly upload via docking station—no burden on families

4. Real-world measurements over 24 months, not artificial lab conditions

5. Privacy-first design: no GPS tracking, no microphone, no personal data

 

The goal: Create a precise map of each child's mobility progression. This data will become the essential reference for evaluating any future treatment—not just for participants, but for the entire LMBRD2 community worldwide.

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What We Will Discover:

• How quickly does mobility decline in each child?

• What are the early warning signs before major deterioration?

• Can we predict which children are at risk of developing spasticity?

• How do walking difficulties correlate with quality of life and cognitive abilities?

• What are the objective, regulatory-grade criteria to measure treatment effectiveness?

 

A French Study With Global Impact

This project tracks all French children registered with our association. Thanks to remote monitoring, no family is excluded due to geography.

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But the impact goes far beyond France. The natural history data we collect will establish the baseline reference for all LMBRD2 patients worldwide. When families in the US, Australia, or anywhere else ask "how will my child's spasticity progress?", this study will provide the first scientific answers. When researchers develop a treatment, these measurements will be the gold standard for testing it—regardless of where the trial takes place.

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We're building the roadmap for the global LMBRD2 community.

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Why This Is Strategic

Future therapies—whether antisense oligonucleotides, gene therapy, or other approaches—will need baseline data to prove they work. CLIMB-LMBRD2 creates that foundation with regulatory-grade precision.

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Without it, even if a treatment becomes available tomorrow, we won't have the validated tools to test it properly on our children. With it, we'll have EMA-qualified endpoints ready to use in clinical trials anywhere in the world.

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Partnership: Science Meets Expertise

SYSNAV Healthcare brings proven technology (ISO9001, ISO13485, ISO27001 certified) already validated in rare disease trials.

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Academic research team specialized in rare neurological diseases provides scientific expertise.

LMBRD2 families provide the essential real-world perspective that makes this study feasible and meaningful.

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Budget: ~€10,000

• Syde® sensor platform and data infrastructure

• 24-month continuous monitoring for all participants

• Expert data analysis and clinical validation

• Project coordination and regulatory compliance

• Scientific reporting and publication

 

Additional costs not covered: Family travel for initial sensor calibration and training sessions, supplementary hospital assessments. Our association covers these expenses through your donations to ensure no family is left out.

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