Turner syndrome

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25Active trials34Specialists8Treatment centers

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UniteRare data is sourced from FDA.gov, ClinicalTrials.gov, Orphanet, OMIM, and NORD.
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Overview

Turner syndrome (also known as 45,X syndrome, Ullrich-Turner syndrome, or gonadal dysgenesis, 45,X) is a chromosomal condition that affects females, caused by complete or partial absence of one X chromosome. The most common karyotype is 45,X (monosomy X), though mosaic forms (e.g., 45,X/46,XX) and structural abnormalities of the X chromosome (such as isochromosome Xq, ring X, or deletions) also occur. Turner syndrome affects multiple body systems, including the reproductive, cardiovascular, skeletal, renal, and endocrine systems. Key clinical features include short stature, ovarian insufficiency leading to delayed puberty and infertility, congenital heart defects (particularly bicuspid aortic valve and coarctation of the aorta), lymphedema (especially in neonates), a broad or webbed neck, low posterior hairline, shield chest with widely spaced nipples, and renal anomalies such as horseshoe kidney. Girls and women with Turner syndrome are also at increased risk for autoimmune thyroid disease (Hashimoto thyroiditis), type 2 diabetes, hearing loss (both sensorineural and conductive), osteoporosis, and learning difficulties particularly involving visuospatial and mathematical skills, though intelligence is typically in the normal range. Cardiovascular complications, including aortic dissection and hypertension, are a leading cause of morbidity and mortality and require lifelong surveillance. Treatment is multidisciplinary and tailored to the individual's specific manifestations. Growth hormone therapy is standard for improving final adult height and is typically initiated in early childhood. Estrogen replacement therapy is used to induce puberty and maintain secondary sexual characteristics, followed by combined estrogen-progesterone therapy for uterine health. Regular cardiac imaging, thyroid function monitoring, hearing assessments, and bone density evaluations are essential components of ongoing care. Assisted reproductive technologies, including oocyte donation, may be considered for fertility, though pregnancy carries increased cardiovascular risks requiring careful evaluation. With appropriate medical management and surveillance, most individuals with Turner syndrome lead full and productive lives.

Also known as:

Clinical phenotype terms— hover any for plain English:

ThyroiditisHP:0100646GonadoblastomaHP:0000150Gastrointestinal angiodysplasiaHP:0000471Inflammation of the large intestineHP:0002037Biliary cirrhosisHP:0002613Aortic dissectionHP:0002647Gastrointestinal inflammationHP:0004386Arterial dissectionHP:0005294Aortic arch aneurysmHP:0005113Dermatoglyphic ridges abnormalHP:0005689Enlargement of the distal femoral epiphysisHP:0006438Irregular proximal tibial epiphysesHP:0006456
Inheritance

Sporadic

Usually appears on its own, not inherited from a parent

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗NORD ↗

FDA & Trial Timeline

10 events
Mar 2026Turner Syndrome: Genetic Considerations

Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)

TrialRECRUITING
Feb 2026A Study Comparing Different Treatment Approaches for the Initiation of Puberty in Girls With Turner Syndrome Using a TRIFECTA-DARED Approach for Rare Diseases

Universiti Kebangsaan Malaysia Medical Centre — PHASE2

TrialNOT YET RECRUITING
Dec 2025Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders

Ascendis Pharma A/S — PHASE3

TrialRECRUITING
Nov 2025Parental Project Amongst 93 Patients With Turner Syndrome

University Hospital, Strasbourg, France

TrialRECRUITING
Nov 2025A Long-Term Study of Bivamelagon in Participants With Hypothalamic Obesity (HO)

Rhythm Pharmaceuticals, Inc. — PHASE2

TrialENROLLING BY INVITATION
Sep 2025A Trial of Setmelanotide in Patients With Congenital Hypothalamic Obesity (Sub-study of NCT05774756)

Rhythm Pharmaceuticals, Inc. — PHASE3

TrialRECRUITING
Jul 2025Bleeding Patterns in Sequential and Continuous Progesterone Supplementation in Adolescents With Turner Syndrome

Children's Mercy Hospital Kansas City — PHASE4

TrialRECRUITING
Mar 2025Neuromuscular Complications of MEK Inhibitors: a French Case Series and a Systematic Review of the Literature

Central Hospital, Nancy, France

TrialNOT YET RECRUITING
Feb 2025Genetic and Epigenetic Background of Inner Ear Dysfunction in Turner Syndrome

Gødstrup Hospital

TrialRECRUITING
Jan 2025Characterization of Hepatopathy in Turner Syndrome: Analysis of Determinants

IRCCS Azienda Ospedaliero-Universitaria di Bologna

TrialRECRUITING

Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Treatments

No FDA-approved treatments are currently listed for Turner syndrome.

20 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.

View clinical trials →

Clinical Trials

20 recruitingView all trials with filters →
Phase 33 trials
A Research Study to Compare Somapacitan Once a Week With Norditropin® Once a Day in Children Who Need Help to Grow
Phase 3
Active
PI: Clinical Transparency dept. 2834 (Novo Nordisk A/S) · Sites: Birmingham, Alabama; Los Angeles, California +197 more · Age: 210 yrs
A Research Study Looking at How Safe Somapacitan is and How Well it Works in Children Who Need Help to Grow - REAL 9
Phase 3
Active
PI: Clinical Transparency (dept. 2834) (Novo Nordisk A/S) · Sites: Birmingham, Alabama; Sacramento, California +15 more · Age: 1018 yrs
Trial Investigating the Efficacy and Safety of Weekly Lonapegsomatropin Compared to Daily Somatropin in Children and Adolescents With Short Stature or Growth Failure Due to Growth Hormone Sufficient Disorders
Phase 3
Actively Recruiting
PI: Medical Director, MD (Ascendis Pharma A/S) · Sites: Centennial, Colorado; Orlando, Florida +3 more · Age: 217 yrs
Phase 43 trials
Long Term Effects of Oral Versus Transdermal Estrogen Replacement Therapy in Turner Syndrome
Phase 4
Actively Recruiting
PI: Claus H Gravholt, Professor (Aarhus University Hospital) · Sites: Aarhus, Aarhus N · Age: 1850 yrs
Determining Dose Equivalence Between Oral and Transdermal Estrogen Treatment in Women With Turner Syndrome
Phase 4
Actively Recruiting
PI: Claus H Gravholt, Professor (Aarhus University Hospital) · Sites: Aarhus, Aarhus N · Age: 1850 yrs
Bleeding Patterns in Sequential and Continuous Progesterone Supplementation in Adolescents With Turner Syndrome
Phase 4
Actively Recruiting
PI: Tazim Dowlut-McElroy, M.D., M.S. (Children's Mercy Kansas City) · Sites: Kansas City, Missouri · Age: 1220 yrs
Phase 23 trials
Vosoritide for Short Stature in Turner Syndrome
Phase 2
Actively Recruiting
PI: Roopa Kanakatti Shankar, MBBS, MS (Children's National Research Institute) · Sites: Washington D.C., District of Columbia · Age: 311 yrs
A Basket Study of Vosoritide in Children With Turner Syndrome, Short Stature Homeobox-Containing Gene Deficiency, and Noonan Syndrome With Inadequate Growth During or After Human Growth Hormone Treatment
Phase 2
Actively Recruiting
PI: Medical Director, MD, PhD (BioMarin Pharmaceutical) · Sites: Orange, California; Aurora, Colorado +22 more · Age: 311 yrs
A Trial to Investigate Different Doses of Lonapegsomatropin Compared to Somatropin in Individuals With Turner Syndrome
Phase 2
Active
· Sites: Palo Alto, California; San Diego, California +17 more · Age: 110 yrs
N/A1 trial
Identification of Y Chromosome From Free Circulating DNA in Patients With Turner Syndrome
N/A
Actively Recruiting
· Sites: Lyon; Strasbourg · Age: 274 yrs
Other10 trials
Role of Cardiac AngioMR in Diagnosis of Cardiac and Vascular Anomalies in Adult Patients with Turner Syndrome
Actively Recruiting
· Sites: Modena · Age: 1899 yrs
Characterization of Hepatopathy in Turner Syndrome: Analysis of Determinants
Actively Recruiting
PI: Alessandra Gambineri, MD (IRCCS Azienda Ospedaliero-Universitaria di Bologna) · Sites: Bologna · Age: 1899 yrs
Inspiring New Science In Guiding Healthcare in Turner Syndrome Registry
Actively Recruiting
PI: Shanlee M Davis, MD, PhD (Children's Hospital Colorado) · Sites: Aurora, Colorado; Washington D.C., District of Columbia +8 more
Turner Syndrome: Genetic Considerations
Actively Recruiting
PI: Veronica Gomez-Lobo, M.D. (Eunice Kennedy Shriver National Institute of Child) · Sites: Bethesda, Maryland · Age: 0110 yrs
UTHealth Turner Syndrome Research Registry
Actively Recruiting
· Sites: Houston, Texas
Long-term Safety and Effectiveness of Growtropin®-II Treatment in Children With Short Stature
Actively Recruiting
· Sites: Seoul, Seodaemun-gu · Age: 299 yrs
Parental Project Amongst 93 Patients With Turner Syndrome
Actively Recruiting
· Sites: Strasbourg · Age: 1840 yrs
Long-term Safety and Effectiveness of Growth Hormone With GHD, TS, CRF, SGA , ISS and PWS in Children
Actively Recruiting
PI: HW Chae, M.D. (Kangnam severance hospital) · Sites: Seoul · Age: 299 yrs
Genetic and Epigenetic Background of Inner Ear Dysfunction in Turner Syndrome
Actively Recruiting
PI: Therese Ovesen, Prof (University Clinic of Flavour, Balance and Sleep) · Sites: Herning · Age: 1860 yrs
Lymphedema, Low-grade Inflammation and the Vasculature in Turner Syndrome
Actively Recruiting
· Sites: Aarhus N · Age: 18100 yrs

Specialists

Showing 25 of 34View all specialists →
NM
Nelly Mauras, MD
JACKSONVILLE, FL
Specialist
PI on 6 active trials
RP
Régis Coutant, MD PhD
Rennes, Ile Et Vilaine
Specialist

Rare Disease Specialist

PI on 1 active trial
CM
Claus H Gravholt, MD
Specialist
PI on 2 active trials
SM
Stephanie B Seminara, MD
BOSTON, MA
Specialist
PI on 5 active trials
VM
Veronica Gomez-Lobo, M.D.
WASHINGTON, DC
Specialist
PI on 6 active trials
GC
Global Clinical Compliance
Specialist
PI on 12 active trials
XD
Xiaoping Luo, Doctor
ROCKVILLE, MD
Specialist
PI on 2 active trials
SP
Shanlee M Davis, MD, PhD
AURORA, CO
Specialist
PI on 3 active trials
CP
Claus H Gravholt, Professor
Specialist
PI on 2 active trials
RM
Richard Porreco, MD
DENVER, CO
Specialist
PI on 3 active trials
JC
J S Christíansen
Specialist
PI on 1 active trial
JC
Jens Sandahl Christiansen
Specialist
PI on 1 active trial
LG
Laura de Graaff
Specialist
PI on 1 active trial
JV
Janielle vd Velden
Specialist
PI on 1 active trial
CG
Claus Gravholt
Specialist
PI on 1 active trial
RK
Rebecca Knickmeyer
Specialist
PI on 1 active trial
KF
Kathrin Fleischer
PORTLAND, OR
Specialist
PI on 1 active trial
RK
Rebecca C Knickmeyer
Specialist
PI on 1 active trial
PD
Pauline Ducatel
Specialist
PI on 1 active trial
TO
Therese Ovesen
Specialist
PI on 1 active trial
AG
Alessandra Gambineri
Specialist
PI on 1 active trial
MJ
Muhammad Irfan Abdul Jalal
SYRACUSE, NY
Specialist
PI on 1 active trial
LG
Laura CG de Graaff
Specialist
PI on 1 active trial

Treatment Centers

8 centers
⚗️ Trial Site

CHU Angers

📍 Angers

👤 Julien Rossignol, Dr

⚗️ Trial Site

CHU Nantes

📍 Nantes

👤 Sebastien HASCOËT

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

UCLA UDN Clinical Site

UCLA Health

📍 Los Angeles, CA

🔬 UDN

Baylor College of Medicine UDN Clinical Site

Baylor College of Medicine

📍 Houston, TX

🔬 UDN

Harvard/MGH UDN Clinical Site

Massachusetts General Hospital

📍 Boston, MA

🏥 NORD

Mayo Clinic Center for Individualized Medicine

Mayo Clinic

📍 Rochester, MN

👤 Mayo Clinic Center for Individualized Medicine

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

Travel Grants

No travel grants are currently matched to Turner syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Turner syndrome

1 articles
NewsCLINICALTRIALS.GOVFeb 5, 2026
New recruiting trial: Genetic Aortic Diseases and Cardiovascular Complications. An Observational Survey of Risk Factors.
The aim of this observational study is to define the behavioural and cardiovascular risk factor profiles of patients with genetic aortopathies. The main questio
See all news about Turner syndrome

Caregiver Resources

NORD Caregiver Resources

Support, advocacy, and financial assistance for caregivers of rare disease patients.

Mental Health Support

Rare disease caregiving can be isolating. Connect with counseling and peer support.

Family & Caregiver Grants

Financial assistance programs specifically for caregivers of rare disease patients.

Social Security Disability

Learn how rare disease patients may qualify for SSDI/SSI benefits.

Common questions about Turner syndrome

What is Turner syndrome?

Turner syndrome (also known as 45,X syndrome, Ullrich-Turner syndrome, or gonadal dysgenesis, 45,X) is a chromosomal condition that affects females, caused by complete or partial absence of one X chromosome. The most common karyotype is 45,X (monosomy X), though mosaic forms (e.g., 45,X/46,XX) and structural abnormalities of the X chromosome (such as isochromosome Xq, ring X, or deletions) also occur. Turner syndrome affects multiple body systems, including the reproductive, cardiovascular, skeletal, renal, and endocrine systems. Key clinical features include short stature, ovarian insufficien

How is Turner syndrome inherited?

Turner syndrome follows a sporadic inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Turner syndrome typically begin?

Typical onset of Turner syndrome is neonatal. Age of onset can vary across affected individuals.

Are there clinical trials for Turner syndrome?

Yes — 20 recruiting clinical trials are currently listed for Turner syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Turner syndrome?

25 specialists and care centers treating Turner syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.