Lymphangioleiomyomatosis

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ORPHA:538OMIM:606690D48.7
Who is this for?
Show terms as
1FDA treatments11Active trials41Specialists8Treatment centers

Where are you in your journey?

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

Lymphangioleiomyomatosis is treated with 1 medication in our database, including SIROLIMUS. Medications are manufactured by Pfizer. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Lymphangioleiomyomatosis treatment below.

Also known as:

Clinical phenotype terms— hover any for plain English:

EmphysemaHP:0002097PneumothoraxHP:0002107Multiple renal cystsHP:0005562
Inheritance

Variable

Can be inherited in different ways depending on the underlying gene

Age of Onset

Adult

Begins in adulthood (age 18 or older)

Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

10 events
Oct 2025The Lateral, Anterior, Medial (LAM) Femoral Cutaneous Block: A Case Series Harborview Medical Center, Seattle, WA

University of Washington

TrialRECRUITING
Oct 2025Evaluating the Long-term Safety and Tolerability of Imatinib in Patients With Lymphangioleiomyomatosis (LAM)

Columbia University — PHASE1

TrialRECRUITING
Oct 2025Impact of Medicalized Residential Care (LAM) on the Development of Care Plans for Vulnerable Patients in Palliative Care

University Hospital, Strasbourg, France

TrialRECRUITING
Aug 2025A Study of Exercise and Pharmacologic Intervention on Systemic Inflammation

Icahn School of Medicine at Mount Sinai — PHASE1

TrialRECRUITING
Jul 2025Switch to Dolutegravir Plus Lamivudine Dual-Therapy in Transgender Women Living With HIV on Virologically Suppressive Antiretroviral Therapy (TRANS-SWITCH)

UBATEC — PHASE4

TrialRECRUITING
Jul 2025Exploration of the Variability of Exposure to Antiretroviral Treatment in Hair With a View to Validating Its Value as a Diagnostic Tool for Partial and/or Total Non-compliance With Treatment

University Hospital, Toulouse

TrialRECRUITING
May 2025DOR/TDF/3TC COmpared With BIC/FTC/TAF in ART-Naïve People Living With HIV and Overweight or Obesity

José Antonio Mata Marín — PHASE3

TrialRECRUITING
Apr 2025Doravirine/Lamivudine (DOR/3TC) as a Maintenance ART in Comparison With Dolutegravir/Lamivudine (DTG/3TC) in PLWH Successfully

Centre de Recherches et d'Etude sur la Pathologie Tropicale et le Sida — PHASE2

TrialRECRUITING
Apr 2025Role of Extracellular Vesicles as Biomarkers of Pulmonary Involvement in Patients With Lymphangioleiomyomatosis and Tuberous Sclerosis Complex

University of Milan — NA

TrialRECRUITING
Apr 2025Safety, Tolerability and Effectiveness of DTG/3TC vs BIC/TAF/FTC in PWH Without Antiretroviral Experience

Instituto Mexicano del Seguro Social — PHASE4

TrialRECRUITING

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

Treatments

1 available

Sirolimus

Sirolimus· Pfizer

* Patient Copay Amount: Not Publicly Available * Maximum Annual Benefit Limit: $25,000 annually * Core Eligibility Restrictions: Patients must have commercial or private insurance. * RxBIN, PCN, and G

Clinical Trials

11 recruitingView all trials with filters →
Phase 31 trial
Multicenter Interventional Lymphangioleiomyomatosis (LAM) Early Disease Trial
Phase 3
Active
PI: Francis X. McCormack, M.D. (University of Cincinnati) · Sites: Palo Alto, California; Denver, Colorado +6 more · Age: 1899 yrs
Phase 12 trials
Evaluating the Long-term Safety and Tolerability of Imatinib in Patients With Lymphangioleiomyomatosis (LAM)
Phase 1
Actively Recruiting
PI: Jeanine D'Armiento, MD, PhD (Columbia University) · Sites: New York, New York; Charleston, South Carolina · Age: 1864 yrs
Feasibility of [11C]Acetate-PET in LAM and TSC
Phase 1
Active Prior treatment eligible
PI: Carmen P Priolo, MD PhD (Brigham and Women's Hospital) · Sites: Boston, Massachusetts · Age: 1899 yrs
N/A2 trials
The Genotype and Phenotype of Lymphangioleiomyomatosis
N/A
Actively Recruiting
PI: Yao-Wen Kuo (National Taiwan University Hospital) · Sites: Taipei · Age: 1899 yrs
Role of Extracellular Vesicles as Biomarkers of Pulmonary Involvement in Patients With Lymphangioleiomyomatosis and Tuberous Sclerosis Complex
N/A
Actively Recruiting
· Sites: Milan, Milano · Age: 1880 yrs
Other5 trials
A National Registry on Chinese Patients With Lymphangioleiomyomatosis
Actively Recruiting
· Sites: Beijing, Beijing Municipality
National Lymphangioleiomyomatosis Registry, France
Actively Recruiting
PI: Vincent Cottin, MD (Groupe d'Etudes et de Recherche sur les Maladies O) · Sites: Lyon · Age: 1899 yrs
Breath Analysis and Arterial Stiffness in Patients With Respiratory Diseases
Enrolling by Invitation
· Sites: Moscow · Age: 1899 yrs
TSC Biosample Repository and Natural History Database
Actively Recruiting
PI: Steve Roberds, PhD (TSC Alliance) · Sites: Birmingham, Alabama; Loma Linda, California +22 more
Safety and Durability of Sirolimus for Treatment of LAM
Actively Recruiting
PI: Francis X McCormack, MD (University of Cincinnati) · Sites: Stanford, California; Denver, Colorado +18 more · Age: 1899 yrs

Specialists

Showing 25 of 41View all specialists →
FM
Francis X McCormack, MD
STURGEON BAY, WI
Specialist
PI on 3 active trials
CP
Carmen P Priolo, MD PhD
Boston, Massachusetts
Specialist

Rare Disease Specialist

PI on 1 active trial
DP
David M Ritter, MD, PhD
Cincinnati, Ohio
Specialist

Rare Disease Specialist

PI on 1 active trial
YK
Yao-Wen Kuo
Taipei
Specialist

Rare Disease Specialist

PI on 1 active trial25 Lymphangioleiomyomatosis publications
DH
Danjing Hu
Specialist
2 Lymphangioleiomyomatosis publications
SJ
Simon R Johnson
Specialist
4 Lymphangioleiomyomatosis publications
NG
Nishant Gupta
Specialist
5 Lymphangioleiomyomatosis publications
YT
Yan Tang
FREMONT, CA
Specialist
2 Lymphangioleiomyomatosis publications
MH
Marina K Holz
Specialist
2 Lymphangioleiomyomatosis publications
CM
Cormac McCarthy
CINCINNATI, OH
Specialist
2 Lymphangioleiomyomatosis publications
FM
Francis X McCormack
STURGEON BAY, WI
Specialist
2 Lymphangioleiomyomatosis publications
SH
Sergio Harari
Specialist
3 Lymphangioleiomyomatosis publications
CC
Chongsheng Cheng
Specialist
2 Lymphangioleiomyomatosis publications
YX
Yan Xu
Specialist
2 Lymphangioleiomyomatosis publications
YW
Yixin Wu
Specialist
2 Lymphangioleiomyomatosis publications
DM
Diane V Havlir, MD
SAN FRANCISCO, CA
Specialist
PI on 2 active trials
JM
Joel Moss, M.D.
Bethesda, Maryland
Specialist

Rare Disease Specialist

PI on 11 active trials
JM
Jean W Pape, MD
Specialist
PI on 2 active trials
FL
Francesca Luisi
Specialist
2 Lymphangioleiomyomatosis publications
DE
Davide Elia
Specialist
3 Lymphangioleiomyomatosis publications
RC
Roberto Cassandro
Specialist
2 Lymphangioleiomyomatosis publications
AC
Antonella Caminati
Specialist
2 Lymphangioleiomyomatosis publications
CM
Christine Katlama, MD
Specialist
PI on 3 active trials
GP
Graeme Meintjes, PhD
Specialist
PI on 2 active trials

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 Lymphangioleiomyomatosis.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about Lymphangioleiomyomatosis

Disease timeline:

New recruiting trial: Change in Body Weight and BMI in PWH with DOR/3TC/TDF Compared with INSTI

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: Doravirine/Lamivudine (DOR/3TC) as a Maintenance ART in Comparison With Dolutegravir/Lamivudine (DTG/3TC) in PLWH Successfully

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: Simplified Treatment of Anti-retrovirus in China (C-STAR)

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: Impact of Medicalized Residential Care (LAM) on the Development of Care Plans for Vulnerable Patients in Palliative Care

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: DOR/TDF/3TC Switch With M184V/I in People With Controlled HIV (Drive Off-Road)

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: A Study of Exercise and Pharmacologic Intervention on Systemic Inflammation

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: Dolutegravir Plus Lamivudine Simplified Therapy

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: DOR/TDF/3TC COmpared With BIC/FTC/TAF in ART-Naïve People Living With HIV and Overweight or Obesity

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: A Randomized Clinical Trial to Evaluate Solutions for the Management of Virologic Failure on TLD in Sub-Saharan Africa

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

New recruiting trial: Good-first: B/F/TAF As First-line ART

A new clinical trial is recruiting patients for Lymphangioleiomyomatosis

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 Lymphangioleiomyomatosis

What is Lymphangioleiomyomatosis?

Lymphangioleiomyomatosis is treated with 1 medication in our database, including SIROLIMUS. Medications are manufactured by Pfizer. Patients and caregivers can find copay cards, patient assistance programs, and travel grants for Lymphangioleiomyomatosis treatment below.

At what age does Lymphangioleiomyomatosis typically begin?

Typical onset of Lymphangioleiomyomatosis is adult. Age of onset can vary across affected individuals.

Are there clinical trials for Lymphangioleiomyomatosis?

Yes — 11 recruiting clinical trials are currently listed for Lymphangioleiomyomatosis on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat Lymphangioleiomyomatosis?

25 specialists and care centers treating Lymphangioleiomyomatosis are listed on UniteRare, sourced from ClinicalTrials.gov principal investigators, published research, and the NPPES NPI registry.

What treatment and support options exist for Lymphangioleiomyomatosis?

1 FDA-approved treatment are currently tracked on UniteRare for Lymphangioleiomyomatosis. See the treatments and support programs sections for copay assistance, eligibility, and contact details.