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Disease

Rickets

Late-stage therapeutic developmentEmerging research
MeSHRickets
2
Publications
12
Clinical trials
1
Related treatments
2
Associated genes
1
Related proteins
2024
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.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial expected to report results, the earliest in Q4 2026.
Upcoming Milestones
Clinical Milestones2View
Results expected 1
Other updates 1
Activity timeline2

Research-associated treatments

Drugs and agents co-studied with this disease across the research literature — associative, not necessarily established treatments. Number shows shared papers.

Clinical trials

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

The current development programme across all trial phases.

Clinical programme
12
All trials
2
Active
5
Late-stage
6
Completed
Late-stage studies

The ENERGY 2 Study: An Open-Label Phase 3 Study to Evaluate the Efficacy and Safety of INZ-701 in Infants With Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 (ENPP1) Deficiency

Phase 3 · Active, not recruiting · Inozyme Pharma

Effectiveness of Vitamin C Supplementation in Infants and Children Suffering of Nutritional Rickets

Phase 2/3 · Completed · Cairo University

A Phase 3 Open-Label Trial to Assess the Efficacy and Safety of KRN23 in Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia and a Postmarketing Study of KRN23 Switched From the Phase 3 Trial

Phase 3 · Completed · Kyowa Kirin Co., Ltd.

An Open Label Trial to Assess the Safety and Efficacy of Burosumab (KRN23), an Investigational Antibody to FGF23, in a Single Pediatric Patient With Epidermal Nevus Syndrome(ENS) and Associated Hypophosphatemic Rickets

Phase 3 · Completed · Redwood Dermatology Sciences

Therapeutic Use of Oral Sodium Phosphate (Z-521) in Primary Hypophosphatemic Rickets

Phase 3 · Completed · Zeria Pharmaceutical
Recently completed

Effectiveness of Vitamin C Supplementation in Infants and Children Suffering of Nutritional Rickets

Phase 2/3 · Completed · Cairo University

A Phase 3 Open-Label Trial to Assess the Efficacy and Safety of KRN23 in Pediatric Patients With X-linked Hypophosphatemic Rickets/Osteomalacia and a Postmarketing Study of KRN23 Switched From the Phase 3 Trial

Phase 3 · Completed · Kyowa Kirin Co., Ltd.

An Open Label Trial to Assess the Safety and Efficacy of Burosumab (KRN23), an Investigational Antibody to FGF23, in a Single Pediatric Patient With Epidermal Nevus Syndrome(ENS) and Associated Hypophosphatemic Rickets

Phase 3 · Completed · Redwood Dermatology Sciences

Therapeutic Use of Oral Sodium Phosphate (Z-521) in Primary Hypophosphatemic Rickets

Phase 3 · Completed · Zeria Pharmaceutical

Proposition Pour un Traitement Par Hormone de Croissance Des Enfants Atteints de Rachitisme Hypophosphatemique Familial

Phase 1/2 · Completed · Bicetre Hospital

Effect of Calcimimetic (Cinacalcet) on Phosphate-Induced Hyperparathyroidism in Children With Hypophosphatemic Rickets

Phase 1 · Completed · Children's Mercy Hospital Kansas City

Research activity

2 papers

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

Publications over time
20062024
Major themes2
  • Endocrinology1
  • Rickets1
Leading journals2
  • Frontiers in endocrinology1
  • The Journal of clinical investigation1
Leading researchers8
  • Aversa T1
  • Baroncelli GI1
  • Baronio F1
  • Cassio A1
  • Chiarito M1
  • Comberiati P1
  • Cosci O di Coscio M1
  • De Sanctis L1
Affiliations (unnormalised)6
  • Bambino Gesù Children Hospital1
  • Children Hospital1
  • Department of Clinical and Experimental Medicine1
  • Hospital of Bolzano (SABES-ASDAA)1
  • IRCCS Azienda Ospedaliero-Universitaria di Bologna1
  • Laboratory of Pediatric Endocrinology1

Associated genes

2 matches

Genes associated with this disease in the canonical knowledge graph (Open Targets evidence). Number shows the association score.

CYP2R10.38CYP27B10.38

Disease biology

1 match

Proteins whose encoding gene is associated with this disease, reached through the canonical gene→disease graph. Number shows the gene's association score.

25-hydroxyvitamin D-1 alpha hydroxylase, mitochondrialvia CYP27B10.38

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

Disorders caused by interruption of BONE MINERALIZATION manifesting as OSTEOMALACIA in adults and characteristic deformities in infancy and childhood due to disturbances in normal BONE FORMATION. The mineralization process may be interrupted by disruption of VITAMIN D; PHOSPHORUS; or CALCIUM homeostasis, resulting from dietary deficiencies, or acquired, or inherited metabolic, or hormonal disturbances.

Identifiers
MeSHRickets
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.