Lonafarnib clinical trials demonstrate uncoupling of the muscle-bone unit in Hutchinson-Gilford Progeria Syndrome
Authors' conclusion
These results demonstrate that the muscle-bone unit is uncoupled in children with HGPS.
Study context
- Population
- Youth with Hutchinson-Gilford Progeria Syndrome aged 2–18 yr
- Comparison
- Hutchinson-Gilford Progeria Syndrome (HGPS) before and after lonafarnib vs controls
Methodological detailspQCT imaging study, before-and-after treatment, comparison with controls
- pQCT imaging study
- Before-and-after treatment
- Comparison with controls
Primary result
As reported in the abstract
Radial pQCT muscle-bone unit
Untreated radii with highly abnormal shapes
70%
Forearm muscle area growth, controls vs HGPS
β = 1.4 cm²/yr vs 0.3 cm²/yr
Radial area growth, controls vs HGPS
β = 5.8 mm²/yr vs 0.5 mm²/yr
Further findings
| Outcome | P value |
|---|---|
| Muscle area normalized for BMI or radial lengthHGPS vs Controls · normalized for BMI; reduced in HGPS | P = 0.02 |
| Muscle area normalized for BMI or radial lengthHGPS vs Controls · normalized for radial length; reduced in HGPS | P ≤ 0.001 |
Fat area change with age in HGPS
- Fat area decreased with age
- β = −.2 cm²/yr, p < .001
Muscle area normalized for BMI or radial length
- Normalized outcomes similar to controls at younger ages but diverged as patients aged
Muscle-bone phenotype on lonafarnib · 24 mo
- Partial normalization of radial shape
- 25%
- Lonafarnib did not normalize the muscle-bone phenotype
Original publication
Lonafarnib clinical trials demonstrate uncoupling of the muscle-bone unit in Hutchinson-Gilford Progeria Syndrome. J Bone Miner Res. Published 4 December 2025. doi:10.
Composed by AI from the published abstract and checked against it. How Briefs are made →