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Disease

Wiskott-Aldrich Syndrome

Late-stage therapeutic developmentEmerging research
MeSHWiskott-Aldrich Syndrome
1
Publications
14
Clinical trials
2013
Latest publication
Latest activity
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Recent clinical, regulatory, research and industry developments relating to this disease.

What's happening now

An analyst briefing on current research, clinical, regulatory and industry activity surrounding this disease.

Executive briefingUpdating summary…Momentum: Low
Key developments
  • 1 clinical trial with recent milestones.
Major developments
Major clinical milestoneWorth watching
Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies
Primary completion2025-12-30
Clinical Milestones1View
Recently completed 1
Activity timeline1

Clinical trials

13 sponsors · 0 new · 0 completed in the last 12 months (net +0)

The current development programme across all trial phases.

Clinical programme
14
All trials
2
Active
2
Late-stage
6
Completed
Recently completed

Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome

Phase 1/2 · Completed · David Williams

Reinstituting Natural Killer Cell Cytotoxicity and Cytoskeletal Dynamics in Wiskott-Aldrich Syndrome With IL-2 Therapy

Phase 1 · Completed · Soma Jyonouchi

A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents

Phase 4 · Completed · Bio Products Laboratory

Phase I/II Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome

Phase 1/2 · Completed · The Korean Society of Pediatric Hematology Oncology

Haploidentical Hematopoietic Stem Cell Transplantation for Pediatric Patients With Wiskott-Aldrich Syndrome: A Pilot Study

Phase 1 · Completed · St. Jude Children's Research Hospital

IGIV-C 10% Rapid Infusion Trial in Primary Immune Deficient Patients

Phase 3 · Completed · Grifols Therapeutics LLC

Research activity

1 papers

Key research shaping understanding of this disease, combining the latest publications with the most influential evidence.

Most influential

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

Science (New York, N.Y.) · 2013 · 799 cites
Recent publications

Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome.

Science (New York, N.Y.) · 2013 · 799 cites
Major themes1
  • Hematopoietic Stem Cell Transplantation1
Leading journals1
  • Science (New York, N.Y.)1
Leading researchers8
  • Aiuti A1
  • Assanelli A1
  • Banerjee PP1
  • Baricordi C1
  • Benati C1
  • Biasco L1
  • Biffi A1
  • Bosticardo M1
Affiliations (unnormalised)1
  • San Raffaele Telethon Institute for Gene Therapy1

Reference

Authoritative identity, definition & identifiers.

Defined in MeSH

A rare, X-linked immunodeficiency syndrome characterized by ECZEMA; LYMPHOPENIA; and, recurrent pyogenic infection. It is seen exclusively in young boys. Typically, IMMUNOGLOBULIN M levels are low and IMMUNOGLOBULIN A and IMMUNOGLOBULIN E levels are elevated. Lymphoreticular malignancies are common.

Identifiers
MeSHWiskott-Aldrich Syndrome
References & data sources
  • Disease identity & definition — NLM Medical Subject Headings (MeSH), public domain
  • Clinical trials — ClinicalTrials.gov (U.S. National Library of Medicine)
  • Research activity — Europe PMC (EMBL-EBI) + OpenAlex-derived paper links
  • Related entities are derived from literature co-mention (studied together) — associative, not causal.