Wiskott-aldrich Syndrome
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Sign up freeTop drugs studied for this disease
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EMA approval · 2026-03-05EMA Authorised: Waskyra (genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem and progenitor cells (HSPC) transduced ex vivo using a lentiviral vector encoding the human Wiskott-Aldrich Syndrome (WAS) gene) — Fondazione Telethon ETS
EMA authorised Waskyra, a gene therapy using autologous CD34+ cells modified with a lentiviral WAS gene, for Wiskott-Aldrich Syndrome.
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CLINICAL_TRIALS research · 2026-02-19Clinical Trial Update: Immune Disorder HSCT Protocol [Phase 2]
Phase 2 clinical trial update on a hematopoietic stem cell transplant protocol for several inherited immune deficiency disorders; the study …
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CLINICAL_TRIALS trial_result · 2026-01-15Clinical Trial Update: Allogeneic Hematopoietic Stem Cell Transplant for Patients With Primary Immune Deficiencies [NA]
This clinical trial investigates allogeneic hematopoietic stem cell transplantation as a treatment for primary immune deficiencies, includin…
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CLINICAL_TRIALS trial_result · 2025-12-15Clinical Trial Update: Sequential Cadaveric Lung and Bone Marrow Transplant for Immune Deficiency Diseases [Phase 1, Phase 2]
This clinical trial update describes an early-phase study evaluating sequential cadaveric lung transplantation followed by bone marrow trans…
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EMA approval · 2025-11-14First gene therapy to treat rare disease Wiskott-Aldrich syndrome
The EMA has approved the first gene therapy for Wiskott-Aldrich syndrome (Waskyra), which significantly reduces infections and bleeding epis…
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CLINICAL_TRIALS research · 2025-09-29Clinical Trial Update: A Clinical Study to Evaluate the Use of a Cryopreserved Formulation of OTL-103 in Subjects With Wiskott-Aldrich Syndrome [Phase 3]
Phase 3 clinical trial evaluating a cryopreserved formulation of OTL-103 for Wiskott-Aldrich Syndrome. The study is currently active but not…
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CLINICAL_TRIALS trial_result · 2025-08-14Clinical Trial Update: Pilot and Feasibility Study of Hematopoietic Stem Cell Gene Transfer for the Wiskott-Aldrich Syndrome [Phase 1, Phase 2]
Phase 1/2 pilot and feasibility study of hematopoietic stem cell gene transfer for Wiskott-Aldrich Syndrome has completed.
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CLINICAL_TRIALS trial_result · 2025-04-04Clinical Trial Update: Gene Therapy for Wiskott-Aldrich Syndrome [Phase 1, Phase 2]
Phase 1/2 gene therapy trial for Wiskott-Aldrich Syndrome completed with results posted on 2025-04-04.
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CLINICAL_TRIALS research · 2024-09-19Clinical Trial Update: Molecular and Clinical Studies of Primary Immunodeficiency Diseases
This clinical trial update describes molecular and clinical studies of primary immunodeficiency diseases, including Wiskott-Aldrich syndrome…
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CLINICAL_TRIALS trial_result · 2023-09-15Clinical Trial Update: Conditioning Regimen for Allogeneic Hematopoietic Stem-Cell Transplantation [Phase 1, Phase 2]
A Phase 1/2 clinical trial evaluating conditioning regimens for allogeneic hematopoietic stem-cell transplantation has completed recruitment…
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CLINICAL_TRIALS trial_result · 2021-06-03Clinical Trial Update: Gene Therapy for Wiskott-Aldrich Syndrome (WAS) [Phase 1, Phase 2]
Phase 1/2 clinical trial update on a gene therapy approach for Wiskott-Aldrich Syndrome has completed.
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CLINICAL_TRIALS trial_result · 2021-06-03Clinical Trial Update: Long Term Safety Follow up of Haematopoietic Stem Cell Gene Therapy for the Wiskott Aldrich Syndrome [Phase 1, Phase 2]
Long-term safety follow-up study of haematopoietic stem cell gene therapy for Wiskott-Aldrich Syndrome, currently active but not recruiting.
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CLINICAL_TRIALS trial_result · 2020-05-06Clinical Trial Update: Efficacy and Safety of Romiplostim Versus Eltrombopag in the Treatment of Thrombocytopenia in Patients With Wiskott-Aldrich Syndrome [Phase 2]
Phase 2 trial comparing romiplostim to eltrombopag for thrombocytopenia in Wiskott-Aldrich Syndrome; efficacy and safety data not disclosed …
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CLINICAL_TRIALS trial_result · 2019-03-18Clinical Trial Update: Thrombocytopenia and Bleeding in Wiskott-Aldrich Syndrome (WAS) Patients [Phase 2]
Phase 2 clinical trial update addressing thrombocytopenia and bleeding in patients with Wiskott-Aldrich Syndrome; study terminated with post…
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CLINICAL_TRIALS trial_result · 2018-12-12Clinical Trial Update: A Trial of Plerixafor/G-CSF as Additional Agents for Conditioning Before TCR Alpha/Beta Depleted HSCT in WAS Patients [Phase 2]
Phase 2 trial evaluating plerixafor/G-CSF as conditioning agents before TCR alpha/beta depleted HSCT in patients with Wiskott-Aldrich Syndro…
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CLINICAL_TRIALS trial_result · 2018-05-22Clinical Trial Update: Gene Therapy for WAS [Phase 1, Phase 2]
Genethon's Phase 1/2 gene therapy trial for Wiskott-Aldrich Syndrome has completed.
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CLINICAL_TRIALS trial_result · 2017-08-16Clinical Trial Update: Interleukin-2 Treatment for Wiskott-Aldrich Syndrome [Phase 1]
A Phase 1 clinical trial evaluating interleukin-2 therapy for Wiskott-Aldrich syndrome and X-linked thrombocytopenia has completed.
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CLINICAL_TRIALS trial_result · 2017-06-27Clinical Trial Update: Post-transplant Cyclophosphamide in Wiskott-Aldrich Syndrome
This entry reports an update on a clinical trial evaluating post-transplant cyclophosphamide for patients with Wiskott-Aldrich Syndrome.
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CLINICAL_TRIALS trial_result · 2017-04-26Clinical Trial Update: Haploidentical Hematopoietic Stem Cell Transplantation Patients With Wiskott-Aldrich Syndrome [Phase 1]
Phase 1 clinical trial evaluating haploidentical hematopoietic stem cell transplantation in patients with Wiskott-Aldrich syndrome.
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CLINICAL_TRIALS trial_result · 2016-06-23Clinical Trial Update: Unrelated Hematopoietic Stem Cell Transplantation(HSCT) for Genetic Diseases of Blood Cells [Phase 1, Phase 2]
A completed Phase 1/2 clinical trial evaluated unrelated hematopoietic stem cell transplantation for several genetic blood cell disorders, i…
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CLINICAL_TRIALS trial_result · 2016-06-17Clinical Trial Update: Related Hematopoietic Stem Cell Transplantation (HSCT) for Genetic Diseases of Blood Cells [Phase 2]
Phase 2 study evaluating related hematopoietic stem cell transplantation for several genetic blood disorders; the trial was terminated and r…
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CLINICAL_TRIALS trial_result · 2015-03-09Clinical Trial Update: Alefacept and Allogeneic Hematopoietic Stem Cell Transplantation [NA]
Clinical trial update indicating termination of an Alefacept plus allogeneic hematopoietic stem cell transplantation study, with results pos…
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CLINICAL_TRIALS trial_result · 2014-12-23Clinical Trial Update: A Study to Find Out How Safe and Effective Gammaplex® is in Young People With Primary Immunodeficiency [Phase 4]
Phase 4 study evaluating the safety and effectiveness of Gammaplex in youths with primary immunodeficiency disorders, including Common Varia…
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CLINICAL_TRIALS trial_result · 2014-07-14Clinical Trial Update: Study of Reduced Toxicity Myeloablative Conditioning Regimen for Wiskott-Aldrich Syndrome (WAS) [Phase 1, Phase 2]
A completed Phase 1/2 clinical trial evaluating a reduced-toxicity myeloablative conditioning regimen for Wiskott-Aldrich Syndrome.
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CLINICAL_TRIALS trial_result · 2013-07-02Clinical Trial Update: Allogeneic SCT Of Pts With SCID And Other Primary Immunodeficiency Disorders [Phase 1, Phase 2]
This clinical trial update reports on allogeneic stem cell transplantation in patients with severe combined immunodeficiency and other prima…