Overview
FDA & Trial Timeline
10 eventsNational Human Genome Research Institute (NHGRI)
Assistance Publique Hopitaux De Marseille — NA
Baylor College of Medicine — NA
Bahçeşehir University — NA
The Hospital for Sick Children — NA
IRCCS Policlinico S. Donato
University Hospital, Rouen
University Hospital Freiburg — NA
Fondazione Policlinico Universitario Agostino Gemelli IRCCS — NA
IRCCS Azienda Ospedaliero-Universitaria di Bologna
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
No FDA-approved treatments are currently listed for Rare genetic disease.
9 clinical trialsare actively recruiting — trials can provide access to cutting-edge therapies.
View clinical trials →Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersBoston Children's Hospital
📍 Boston, Massachusetts
University of Pennsylvania
📍 Philadelphia, Pennsylvania
Stanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🔬 UDNUCLA UDN Clinical Site ↗
UCLA Health
📍 Los Angeles, CA
🔬 UDNBaylor College of Medicine UDN Clinical Site ↗
Baylor College of Medicine
📍 Houston, TX
🔬 UDNHarvard/MGH UDN Clinical Site ↗
Massachusetts General Hospital
📍 Boston, MA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
Travel Grants
No travel grants are currently matched to Rare genetic disease.
Community
No community posts yet. Be the first to share your experience with Rare genetic disease.
Start the conversation →Latest news about Rare genetic disease
Disease timeline:
New recruiting trial: Blood Spot and Urine Metabolomic Screening Applied to Rare Diseases
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: Expanding NGS Data with Optical Genome Mapping (OGM)
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: PREcision Diagnostics in Rare genetIC Diseases and Tumors - Long Read Sequencing
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: Universal Rare Gene Study: A Registry and Natural History Study of Retinal Dystrophies Associated With Rare Disease-Causing Genetic Variants
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: The Relationship Between Functional Independence and Family Well-being in Children With Rare Genetic Disorders
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: National Exhaustive Cohort of Hereditary Stomatocytoses and Other Channelopathies Affecting the Red Blood Cell
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: Identification of the Molecular and/or Pathophysiological Bases of Rare Diseases of Genetic Origin (or Rare Forms of Frequent Diseases Suspected of Being of Genetic Origin).
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: Genetic Aortic Diseases and Cardiovascular Complications. An Observational Survey of Risk Factors.
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: CAnadian CAncers With Rare Molecular Alterations (CARMA) - Basket Real-world Observational Study (BROS)
A new clinical trial is recruiting patients for Rare genetic disease
New recruiting trial: Genetic Causes of Discrepant Clinic in Monogenic Twins
A new clinical trial is recruiting patients for Rare genetic disease
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 Rare genetic disease
Are there clinical trials for Rare genetic disease?
Yes — 9 recruiting clinical trials are currently listed for Rare genetic disease on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Rare genetic disease?
25 specialists and care centers treating Rare genetic disease are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.