Wiskott-Aldrich syndrome Advocacy Hub | UniteRare

treatment of pediatric patients aged 6 months and older and adults with Wiskott-Aldrich Syndrome (WAS) who have a mutation in the WAS gene and for whom hematopoietic stem cell transplantation (HSCT) is appropriate and no suitable human leukocyte antigen (HLA)-matched related stem cell donor is available

FDAcompleted

Nov 2025Responsiveness of Hindi Version of Oral Health Impact Profile for Periodontitis (OHIP-P-HIN)

Postgraduate Institute of Dental Sciences Rohtak — NA

TrialRECRUITING

Nov 2025Treatment Outcome and Patient-reported Outcomes in Patient Undergoing Non-surgical Root Canal Treatment With Different Irrigation Protocols

Chulalongkorn University — NA

TrialACTIVE NOT RECRUITING

Oct 2025Long-term Evaluation of Sports Activity in Patients Whose Ankle Was Treated by Anatomical Reconstruction or Arthroscopic Repair Following Chronic Lateral Instability

GCS Ramsay Santé pour l'Enseignement et la Recherche — NA

TrialNOT YET RECRUITING

Sep 2025An Application That Can Provide Early Warning of Temperature Health Risks and Give Protective Suggestions or Personalized Suggestions Based on Patients' Own Secondary Prevention Risk Factors Was Applied to Intervene, and the Intervention Effect of APP on Stroke Recurrence Risk Was Analyzed

Xuanwu Hospital, Beijing — NA

TrialNOT YET RECRUITING

Jul 2025Outcomes of Children in Whom a Prenatal Exome Was Performed Based on Ultrasound Findings

University Hospital, Strasbourg, France

TrialRECRUITING

Jun 2025Relationship Between Remimazolam and Etomidate in Induction of General Anesthesia

Second Hospital of Shanxi Medical University — EARLY_PHASE1

TrialNOT YET RECRUITING

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

Financial Assistance

1 program

FINANCIAL LANDSCAPE SUMMARY

Total programs

Open now

Copay cards

Copay Assistance1

Waskyra

Fondazione Telethon ETS

OpenContact for details

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Approved Treatments

3 FDA-approved

Waskyra

(etuvetidigene autotemcel)Orphan drug

Fondazione Telethon ETS

Approved Dec 2025

Gamimune N

(Immune globulin intravenous, human)Orphan drugstandard

Bayer Corporation

Human Immunoglobulin G [EPC]

12.1 Mechanism of Action BIVIGAM is a replacement therapy in patients with primary humoral immunodeficiency (PI) (e.g. agammaglobulinaemia, hypogammag...

FDA label ↗

Doptelet

(avatrombopag)Orphan drugstandard

Sobi, Inc.

12.1 Mechanism of Action Avatrombopag is an orally bioavailable, small molecule TPO receptor agonist that stimulates proliferation and differentiation...

FDA label ↗

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