Webinar Recording: Why CMT Biomarkers Can’t Wait

Webinar Recording: Why CMT Biomarkers Can’t Wait

Thank you to everyone who joined HNF’s live webinar, Why Biomarkers Can’t Wait, focused on the urgent need to develop meaningful, regulatory-ready biomarkers for Charcot–Marie–Tooth, (CMT).

HNF leadership and leading scientific experts shared why biomarkers are essential to faster, more successful clinical trials—and why patient participation is the foundation for making this progress possible.

What you’ll learn in the recording:

• What biomarkers are (in plain language) and why “you can’t fix what you can’t measure.” Biomarkers help researchers and regulators track disease progression and determine whether a therapy is truly working—especially in slowly progressive conditions like CMT.
• Why FDA-qualified biomarkers matter and how the FDA evaluates evidence for biomarker use in clinical trials (measurable, consistent, and clinically meaningful).
• Real-world measurement is the future: HNF shared updates on the BioSensics wearable study, which captures daily-life function over 14 days each year and is already correlating with clinic-based CMT scales. The long-term goal is to expand participation and pursue FDA qualification so wearable endpoints can support trials.
• Promising biomarker directions: Panelists discussed MRI muscle measures, neurofilament light chain (NfL), emerging candidates such as GDF15 and NCAM, and the growing potential of metabolomics—including how discoveries like the CMT-SORD story highlight the promise of finding treatable pathways.
• Why repeat participation matters: Larger and longitudinal datasets—blood + function + patient-reported outcomes over time—are what enable validation, subtype analyses, and stronger statistical confidence (especially for rarer subtypes).

Key takeaway: Biomarkers are the bridge between research and real treatments—and patients and families are the foundation of that bridge.

Ways to take action now

1) Donate blood at an HNF Roadshow:

You can donate blood for biomarker research at an HNF Roadshow near you. Click on the “Location” tab to view 2026 dates and sites:

2) Join Studies & Donate blood at the CMT Summit + Retreat:

Attending the Summit? You can also donate blood on-site to support HNF’s biobank and biomarker discovery efforts—helping accelerate progress toward FDA-qualified measures and future treatments.

3) Join or update GRIN:

If you’re already in the GRIN Registry, please log in and complete any annual updates—every data point strengthens the research