WHIM syndrome

Last reviewed

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ORPHA:51636OMIM:193670D81.8
Who is this for?
Show terms as
1FDA treatments1Active trials3Specialists8Treatment centers1Financial resources

Where are you in your journey?

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

Also known as:

Clinical phenotype terms— hover any for plain English:

Abnormal neutrophil morphologyHP:0011992Bone marrow hypercellularityHP:0031020MyelokathexisHP:0031160Respiratory tract infectionHP:0011947PapillomaHP:0012740VerrucaeHP:0200043
Orphanet ↗OMIM ↗NORD ↗

FDA & Trial Timeline

2 events
Apr 2024

Xolremdi: FDA approved

to increase the number of circulating mature neutrophils and lymphocytes in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis)

FDAcompleted
Oct 2019Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome

X4 Pharmaceuticals — PHASE3

TrialACTIVE NOT RECRUITING

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

Treatments

1 available

Xolremdi

mavorixafor· X4 Pharmaceuticals, Inc.Orphan Drug

to increase the number of circulating mature neutrophils and lymphocytes in patients 12 years of age and older with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis)

Clinical Trials

1 recruitingView all trials with filters →
Phase 31 trial
Efficacy and Safety Study of Mavorixafor in Participants With Warts, Hypogammaglobulinemia, Infections, and Myelokathexis (WHIM) Syndrome
Phase 3
Active
PI: Chief Medical Officer (X4 Pharmaceuticals) · Sites: San Diego, California; Thousand Oaks, California +21 more · Age: 1299 yrs

Specialists

3 foundView all specialists →
DM
David H McDermott, M.D.
Specialist
PI on 3 active trials
PM
Pablo Tebas, MD
PHILADELPHIA, PA
Specialist
PI on 5 active trials
DM
David C Dale, MD
SEATTLE, WA
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

Financial Resources

1 resources
Xolremdi(mavorixafor)X4 Pharmaceuticals, Inc.

Travel Grants

No travel grants are currently matched to WHIM syndrome.

Search all travel grants →NORD Financial Assistance ↗

Community

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Latest news about WHIM syndrome

No recent news articles for WHIM syndrome.

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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 WHIM syndrome

Are there clinical trials for WHIM syndrome?

Yes — 1 recruiting clinical trial is currently listed for WHIM syndrome on UniteRare. See the clinical trials section on this page for phase, sponsor, and site details sourced from ClinicalTrials.gov.

Which specialists treat WHIM syndrome?

3 specialists and care centers treating WHIM 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 WHIM syndrome?

1 patient support program are currently tracked on UniteRare for WHIM syndrome. See the treatments and support programs sections for copay assistance, eligibility, and contact details.