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Disease

Adrenal Hyperplasia, Congenital

Clinical development underwayEmerging research
1
Publications
2
Clinical trials
2022
Latest publication
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

No activity recorded in this window. Try a wider timeframe.

Therapeutic landscape

Therapies with a regulatory footing for this condition, alongside the wider set of agents co-studied with it in the literature.

Approved & established therapies

Approval — Treatment of congenital adrenal hyperplasia (CAH) in adolescents aged 12 years and over a… (2021)

Clinical trials

2 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
2
All trials
0
Active
0
Late-stage
2
Completed
Recently completed

Dexamethasone Treatment of Congenital Adrenal Hyperplasia

Phase 2 · Completed · Boston Children's Hospital

A Novel Therapeutic Modality for Congenital Adrenal Hyperplasia

N/A · Completed · Baylor College of Medicine

Regulatory timeline

Drug regulatory events matched to this condition by indication — EMA.

First approvals
2021emaApprovalHydrocortisone· Treatment of congenital adrenal hyperplasia (CAH) in adolescents aged 12 years and over and adults. source ↗

European Medicines Agency (CC BY 4.0). Events are matched to this condition by drug indication text — approvals/updates for drugs indicated for it, not disease-specific acts.

Research activity

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Major themes1
  • Adrenal Hyperplasia, Congenital1
Leading journals1
  • Endocrine reviews1
Leading researchers8
  • Ahmed SF1
  • Arlt W1
  • Auchus RJ1
  • Claahsen-van der Grinten HL1
  • Falhammar H1
  • Flück CE1
  • Guasti L1
  • Huebner A1
Affiliations (unnormalised)6
  • Bern University Hospital1
  • Center for Rare Endocrine Diseases of Growth and Development1
  • Center for Reproductive Sciences1
  • Centre for Endocrinology1
  • Cohen Children's Medical Center of NY1
  • Diponegoro University1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A group of inherited disorders of the ADRENAL GLANDS, caused by enzyme defects in the synthesis of cortisol (HYDROCORTISONE) and/or ALDOSTERONE leading to accumulation of precursors for ANDROGENS. Depending on the hormone imbalance, congenital adrenal hyperplasia can be classified as salt-wasting, hypertensive, virilizing, or feminizing. Defects in STEROID 21-HYDROXYLASE; STEROID 11-BETA-HYDROXYLASE; STEROID 17-ALPHA-HYDROXYLASE; 3-beta-hydroxysteroid dehydrogenase (3-HYDROXYSTEROID DEHYDROGENASES); TESTOSTERONE 5-ALPHA-REDUCTASE; or steroidogenic acute regulatory protein; among others, underlie these disorders.

References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.