Natural history of hypochondroplasia: A retrospective, matched-cohort study using the clinical practice research Datalink (CPRD) aurum database.
Coordinated, multidisciplinary management of hypochondroplasia starting early improves quality of life and outcomes.
Hypochondroplasia is a rare genetic condition that causes disproportionate short stature. These results highlight the medical burden of hypochondroplasia and need for coordinated, multidisciplinary management starting early in life.
What the study was
- Study design
- Cohort study
- Population
- Study participants
- Sample size
- 549
- Category
- Prevention
- Maturity
- Validated
- Journal
- Bone
Why it surfaced
Matched topic(s): Rare diseases with high unmet need. Study design: Cohort study. Score 5/10.
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