Overview
Prader-Willi syndrome is treated with 1 medication in our database, including VYKAT XR. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by AstraZeneca. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Prader-Willi syndrome treatment below.
Also known as:
Clinical phenotype terms— hover any for plain English:
Variable
Can be inherited in different ways depending on the underlying gene
Neonatal
Begins at or shortly after birth (first 4 weeks)
FDA & Trial Timeline
10 eventsConSynance Therapeutics — PHASE2
Bright Minds Biosciences Pty Ltd — PHASE2
Soleno Therapeutics, Inc.
Harmony Biosciences Management, Inc. — PHASE3
University Hospital, Toulouse — NA
University Hospital, Toulouse
treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS)
Rhythm Pharmaceuticals, Inc. — PHASE2
Harmony Biosciences Management, Inc. — PHASE3
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableNorditropin
growth failure due to Prader-Willi Syndrome (PWS)
Vykat Xr
treatment of hyperphagia in adults and pediatric patients 4 years of age and older with Prader-Willi syndrome (PWS)
Rare Disease Specialist
Treatment Centers
8 centersErasmus Medical Center
📍 Rotterdam
👤 Robert Rissmann, Professor
Stanford Medicine Rare Disease Center ↗
Stanford Medicine
📍 Stanford, CA
🏥 NORDMayo Clinic Center for Individualized Medicine ↗
Mayo Clinic
📍 Rochester, MN
👤 Mayo Clinic Center for Individualized Medicine
🔬 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
🔬 UDNNIH Clinical Center Undiagnosed Diseases Program ↗
National Institutes of Health
📍 Bethesda, MD
🏥 NORDBaylor College of Medicine Rare Disease Center ↗
Baylor College of Medicine
📍 Houston, TX
Financial Resources
2 resourcesTravel Grants
No travel grants are currently matched to Prader-Willi syndrome.
Community
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1 articlesCaregiver 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 Prader-Willi syndrome
What is Prader-Willi syndrome?
Prader-Willi syndrome is treated with 1 medication in our database, including VYKAT XR. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by AstraZeneca. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Prader-Willi syndrome treatment below.
At what age does Prader-Willi syndrome typically begin?
Typical onset of Prader-Willi syndrome is neonatal. Age of onset can vary across affected individuals.
Are there clinical trials for Prader-Willi syndrome?
Yes — 19 recruiting clinical trials are currently listed for Prader-Willi syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Prader-Willi syndrome?
20 specialists and care centers treating Prader-Willi syndrome are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Prader-Willi syndrome?
1 FDA-approved treatment and 2 patient support programs are currently tracked on UniteRare for Prader-Willi syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.