Genetic hypopigmentation of the skin

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ORPHA:183469
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23Specialists8Treatment centers

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

Genetic hypopigmentation of the skin is a broad clinical category (Orphanet code 183469) that encompasses a group of inherited disorders characterized by reduced melanin pigmentation of the skin. These conditions result from genetic defects affecting melanocyte development, melanin biosynthesis, melanosome formation, or melanin transfer to keratinocytes. The degree and distribution of hypopigmentation can vary widely, ranging from localized patches of lighter skin to generalized reduction in skin, hair, and eye pigmentation. Conditions grouped under this umbrella include, but are not limited to, various forms of oculocutaneous albinism, piebaldism, and other genetic disorders featuring skin lightening as a prominent feature. The skin is the primary organ system affected, though depending on the specific underlying condition, the eyes and hair may also be involved. Key clinical features include patches or widespread areas of lighter-than-expected skin color, which may be present from birth or develop in early childhood. Some subtypes are associated with increased susceptibility to sunburn and elevated risk of skin cancers due to reduced photoprotection from melanin. Certain syndromic forms may also involve neurological, immunological, or hematological abnormalities. There is currently no curative treatment for genetic hypopigmentation of the skin. Management is primarily supportive and preventive, focusing on rigorous sun protection (broad-spectrum sunscreen, protective clothing, avoidance of excessive UV exposure), regular dermatological surveillance for skin cancers, and ophthalmological care when ocular involvement is present. Cosmetic camouflage products may be used to address psychosocial concerns. Genetic counseling is recommended for affected individuals and their families to clarify the specific diagnosis, inheritance pattern, and recurrence risk.

Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Variable

Can begin at different ages, from infancy through adulthood

Orphanet ↗NORD ↗

FDA & Trial Timeline

2 events
Oct 2025TRPM2 Gene Polymorphism, NLRP3 Inflammasome Expression in Vitiligo Patients

Aswan University

TrialACTIVE NOT RECRUITING
Oct 2024Functional Impairment in Albinism

Fondation Ophtalmologique Adolphe de Rothschild

TrialRECRUITING

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

Treatments

No FDA-approved treatments are currently listed for Genetic hypopigmentation of the skin.

View clinical trials →

No actively recruiting trials found for Genetic hypopigmentation of the skin at this time.

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

Search ClinicalTrials.gov ↗Join the Genetic hypopigmentation of the skin community →

Specialists

23 foundView all specialists →
LM
Lisa R. Young, MD
Specialist
PI on 1 active trial1 Genetic hypopigmentation of the skin publication
FD
Fanny MORICE-PICARD, Dr
Specialist
PI on 1 active trial
OP
Olivier Lortholary, MD, PhD
Specialist
PI on 5 active trials
WM
Wendy J Introne, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 4 active trials
BM
Brian P Brooks, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 8 active trials
NP
Nanja Van Geel, Pr
Specialist
PI on 1 active trial
JP
Joaquin Espinosa, PhD
Specialist
PI on 3 active trials
FP
Fu-Tong Liu, M.D., Ph.D.
LOS ANGELES, CA
Specialist
PI on 2 active trials
ZP
Zarqa Ali, MD, PhD
Specialist
PI on 1 active trial
GM
Guillemette FOUQUET, MD
Specialist
PI on 1 active trial1 Genetic hypopigmentation of the skin publication
LD
Laura Dalfonso
Specialist
PI on 1 active trial
SP
Smail HADJ-RABIA, MD, PhD
Specialist
PI on 1 active trial
AP
Antoni Gostynski, MD, PhD
Maastricht, Limburg
Specialist

Rare Disease Specialist

PI on 2 active trials
RP
Rachel Sommer, Priv-Doz.Dr.
Specialist
PI on 1 active trial
MF
Martine MAUGET FAYSSE
Specialist
PI on 3 active trials
KC
Kevin J O'Brien, C.R.N.P.
Specialist
PI on 1 active trial
DM
David R Adams, M.D.
Specialist
PI on 1 active trial
SP
Sameek Roychowdhury, MD, PhD
COLUMBUS, OH
Specialist
PI on 2 active trials
JM
Joshua D. Milner, MD
NEW YORK, NY
Specialist
PI on 1 active trial
LM
Ladan Mostaghimi, MD
Specialist
PI on 1 active trial1 Genetic hypopigmentation of the skin publication
TR
Tara Heagele, PhD, RN
Specialist
PI on 1 active trial

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 Genetic hypopigmentation of the skin.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Genetic hypopigmentation of the skin

Disease timeline:

New recruiting trial: Clinical and Basic Investigations Into Hermansky-Pudlak Syndrome

A new clinical trial is recruiting patients for Genetic hypopigmentation of the skin

New recruiting trial: Functional Impairment in Albinism

A new clinical trial is recruiting patients for Genetic hypopigmentation of the skin

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 Genetic hypopigmentation of the skin

What is Genetic hypopigmentation of the skin?

Genetic hypopigmentation of the skin is a broad clinical category (Orphanet code 183469) that encompasses a group of inherited disorders characterized by reduced melanin pigmentation of the skin. These conditions result from genetic defects affecting melanocyte development, melanin biosynthesis, melanosome formation, or melanin transfer to keratinocytes. The degree and distribution of hypopigmentation can vary widely, ranging from localized patches of lighter skin to generalized reduction in skin, hair, and eye pigmentation. Conditions grouped under this umbrella include, but are not limited t

Which specialists treat Genetic hypopigmentation of the skin?

23 specialists and care centers treating Genetic hypopigmentation of the skin are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.