Overview
Tuberous Sclerosis is treated with 1 medication in our database, including HYFTOR. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Nobelpharma. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Tuberous Sclerosis treatment below.
Also known as:
Autosomal dominant
Passed on from just one parent; each child has about a 50% chance of inheriting it
Variable
Can begin at different ages, from infancy through adulthood
FDA & Trial Timeline
3 eventsKatarzyna Kotulska — PHASE3
National Heart, Lung, and Blood Institute (NHLBI) — NA
Data sourced from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.
Treatments
2 availableCrestor
As an adjunct to diet to reduce LDL-C and slow the progression of atherosclerosis in adults
HYFTOR
Detailed copay and financial assistance information is not publicly available for this medication at this time. Please consult your pharmacist or the manufacturer's official patient support program fo
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Rare Disease Specialist
Treatment Centers
8 centersNational Institutes of Health Clinical Center
📍 Bethesda, Maryland
👤 Payal P Khincha, M.D.
👤 Christopher Grunseich, M.D.
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
1 resources2 travel grants are also available for Tuberous Sclerosis patients — see Travel Grants below ↓
Travel Grants
2 grantsAfinitor Everolimus Copay Assistance Program
Eligibility requires a valid prescription for an approved indication and varies by program, with copay cards for commercially insured patients and foundation assistance for those with limited income or no insurance. Applicants must be U.S. residents and typically 18 years or older to qualify for financial support.
Community
No community posts yet. Be the first to share your experience with Tuberous Sclerosis.
Start the conversation →Latest news about Tuberous Sclerosis
Disease timeline:
New trial: Gait in Rare Diseases
Phase NA trial recruiting.
New trial: Study of Skin Tumors in Tuberous Sclerosis
Phase NA trial recruiting.
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 Tuberous Sclerosis
What is Tuberous Sclerosis?
Tuberous Sclerosis is treated with 1 medication in our database, including HYFTOR. 1 of these has manufacturer assistance programs available to help reduce out-of-pocket costs. Medications are manufactured by Nobelpharma. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Tuberous Sclerosis treatment below.
How is Tuberous Sclerosis inherited?
Tuberous Sclerosis follows a autosomal dominant inheritance pattern. Genetic counseling can help families understand recurrence risk and testing options.
Are there clinical trials for Tuberous Sclerosis?
Yes — 3 recruiting clinical trials are currently listed for Tuberous Sclerosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.
Which specialists treat Tuberous Sclerosis?
5 specialists and care centers treating Tuberous Sclerosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.
What treatment and support options exist for Tuberous Sclerosis?
1 FDA-approved treatment are currently tracked on UniteRare for Tuberous Sclerosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.