Schaaf-Yang syndrome

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ORPHA:398069OMIM:615547Q87.1
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15Specialists8Treatment centers

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

Schaaf-Yang syndrome (SYS) is a rare genetic condition caused by changes (mutations) in the MAGEL2 gene, which is located on chromosome 15. This gene plays an important role in brain development and the function of the hypothalamus, a part of the brain that controls hunger, body temperature, hormones, and sleep. Schaaf-Yang syndrome shares some features with Prader-Willi syndrome but is a distinct condition with its own set of challenges. Babies with Schaaf-Yang syndrome often have low muscle tone (hypotonia) and difficulty feeding in the newborn period. As children grow, they may develop intellectual disability ranging from mild to severe, autism spectrum features, joint contractures (stiff or bent joints), and hormonal problems. Many children have distinctive facial features and small hands and feet. Unlike Prader-Willi syndrome, the extreme overeating (hyperphagia) seen in that condition is less common in Schaaf-Yang syndrome, though some individuals do develop increased appetite. There is currently no cure for Schaaf-Yang syndrome. Treatment focuses on managing individual symptoms through therapies such as physical therapy, occupational therapy, speech therapy, and hormone replacement when needed. A team of specialists typically works together to provide the best care. Research into this condition is ongoing, and understanding of the syndrome continues to grow as more patients are identified worldwide.

Also known as:

Key symptoms:

Low muscle tone (floppiness) in infancyFeeding difficulties in newbornsJoint contractures (stiff or bent fingers, wrists, or other joints)Intellectual disability (mild to severe)Autism spectrum behaviorsSpeech and language delaysSmall hands and feetHormonal problems (such as growth hormone deficiency)Sleep disturbancesDistinctive facial featuresDelayed motor milestones (sitting, walking)Temperature regulation problemsObesity or increased appetite in some individualsBehavioral challengesUnderdeveloped genitals in males (hypogonadism)

Clinical phenotype terms (50)— hover any for plain English
Clitoral hypoplasiaHP:0000060Hypoplastic labia minoraHP:0000064
Inheritance

Autosomal dominant

Passed on from just one parent; each child has about a 50% chance of inheriting it

Age of Onset

Neonatal

Begins at or shortly after birth (first 4 weeks)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

1 event
Sep 2010NOR-SYS: The Norwegian Stroke in the Young Study

Haukeland University Hospital — NA

TrialACTIVE NOT RECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Schaaf-Yang syndrome.

View clinical trials →

No actively recruiting trials found for Schaaf-Yang syndrome at this time.

New trials open frequently. Follow this disease to get notified.

Search ClinicalTrials.gov ↗Join the Schaaf-Yang syndrome community →

Specialists

15 foundView all specialists →
CS
Christian Patrick Schaaf
HOUSTON, TX
Specialist
2 Schaaf-Yang syndrome publications
JM
Johann Georg Maaß
Specialist
1 Schaaf-Yang syndrome publication
CS
Christian P Schaaf
HOUSTON, TX
Specialist
4 Schaaf-Yang syndrome publications
TS
Tim Schubert
TACOMA, WA
Specialist
2 Schaaf-Yang syndrome publications
MG
Mark Garrelfs
Specialist
1 Schaaf-Yang syndrome publication
GK
Gerthe F Kerkhof
Specialist
1 Schaaf-Yang syndrome publication
TS
Theresa V Strong
Specialist
2 Schaaf-Yang syndrome publications
DT
Demi Trueba-Timmermans
Specialist
1 Schaaf-Yang syndrome publication
RR
Raquel Rabionet
Specialist
2 Schaaf-Yang syndrome publications
RU
Roser Urreizti
Specialist
2 Schaaf-Yang syndrome publications
AJ
Alicia F Juriaans
Specialist
1 Schaaf-Yang syndrome publication
SB
Susanna Balcells
Specialist
2 Schaaf-Yang syndrome publications
MC
Mónica Centeno-Pla
Specialist
2 Schaaf-Yang syndrome publications
AP
Aina Prat-Planas
Specialist
2 Schaaf-Yang syndrome publications
DG
Daniel Grinberg
WEST NYACK, NY
Specialist
2 Schaaf-Yang syndrome publications

Treatment Centers

8 centers
🏥 NORD

Baylor College of Medicine Rare Disease Center

Baylor College of Medicine

📍 Houston, TX

🏥 NORD

Stanford Medicine Rare Disease Center

Stanford Medicine

📍 Stanford, CA

🔬 UDN

NIH Clinical Center Undiagnosed Diseases Program

National Institutes of Health

📍 Bethesda, MD

🔬 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

🏥 NORD

UCLA Rare Disease Day Program

UCLA Health

📍 Los Angeles, CA

Travel Grants

No travel grants are currently matched to Schaaf-Yang syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Schaaf-Yang syndrome

No recent news articles for Schaaf-Yang syndrome.

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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.

Questions for your doctor

Bring these to your next appointment

  • Q1.What specific MAGEL2 mutation does my child have, and what does it mean for their expected symptoms?,Should my child be tested for growth hormone deficiency, and would growth hormone therapy be beneficial?,What therapies (physical, occupational, speech) should we start, and how often?,Are there any clinical trials or research studies for Schaaf-Yang syndrome that we could participate in?,How should we monitor for hormonal problems, sleep issues, and other complications over time?,What is the risk of having another child with this condition, and should we pursue genetic counseling?,Are there any specific emergency situations we should be prepared for, and what should our action plan be?

Common questions about Schaaf-Yang syndrome

What is Schaaf-Yang syndrome?

Schaaf-Yang syndrome (SYS) is a rare genetic condition caused by changes (mutations) in the MAGEL2 gene, which is located on chromosome 15. This gene plays an important role in brain development and the function of the hypothalamus, a part of the brain that controls hunger, body temperature, hormones, and sleep. Schaaf-Yang syndrome shares some features with Prader-Willi syndrome but is a distinct condition with its own set of challenges. Babies with Schaaf-Yang syndrome often have low muscle tone (hypotonia) and difficulty feeding in the newborn period. As children grow, they may develop int

How is Schaaf-Yang syndrome inherited?

Schaaf-Yang syndrome follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.

At what age does Schaaf-Yang syndrome typically begin?

Typical onset of Schaaf-Yang syndrome is neonatal. Age of onset can vary across affected individuals.

Which specialists treat Schaaf-Yang syndrome?

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