Osteopetrosis
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Sign up freeTop drugs studied for this disease
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CLINICAL_TRIALS trial_result · 2026-01-07Clinical Trial Update: MT2013-31: Allo HCT for Metabolic Disorders and Severe Osteopetrosis [Phase 2]
Phase 2 trial update describing allogeneic hematopoietic cell transplantation for metabolic disorders and severe osteopetrosis, with the stu…
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CLINICAL_TRIALS trial_result · 2024-10-31Clinical Trial Update: Risk-Adapted Allogeneic Stem Cell Transplantation For Mixed Donor Chimerism In Patients With Non-Malignant Diseases [Phase 2, Phase 3]
Clinical trial update reports Phase 2 and Phase 3 results for risk-adapted allogeneic stem cell transplantation in non-malignant diseases wi…
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CLINICAL_TRIALS trial_result · 2024-07-26Clinical Trial Update: Outcomes of Patients With Osteopetrosis Weight-bearing Bone Fractures [NA]
This clinical trial update describes the outcomes of a completed study on weight-bearing bone fractures in patients with osteopetrosis at Ka…
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CLINICAL_TRIALS trial_result · 2023-10-11Clinical Trial Update: ACTIMMUNE in Intermediate Osteopetrosis [Phase 2]
An update on ACTIMMUNE (interferon gamma-1b) for intermediate osteopetrosis reports completion of a Phase 2 clinical trial with results post…
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CLINICAL_TRIALS trial_result · 2023-01-04Clinical Trial Update: Safety Study of Gene Modified Donor T-cells Following TCRαβ+ Depleted Stem Cell Transplant [Phase 1, Phase 2]
A phase 1/2 safety study evaluating gene-modified donor T-cells after TCRαβ+ depleted stem cell transplant was terminated; results were post…
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CLINICAL_TRIALS trial_result · 2022-07-12Clinical Trial Update: Study of Gene Modified Donor T-cells Following TCR Alpha Beta Positive Depleted Stem Cell Transplant [Phase 1, Phase 2]
Clinical trial update for a phase 1/2 study evaluating gene-modified donor T-cells after TCR alpha beta–negative depleted stem cell transpla…
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CLINICAL_TRIALS trial_result · 2019-07-31Clinical Trial Update: Allogeneic Transplantation For Severe Osteopetrosis [Phase 2, Phase 3]
A completed Phase 2/3 clinical trial evaluating allogeneic transplantation for severe osteopetrosis with results posted in 2019.
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CLINICAL_TRIALS trial_result · 2016-01-12Clinical Trial Update: rhPTH Therapy for Low Turnover Bone Fragility [Phase 1]
Phase 1 clinical trial update on recombinant human parathyroid hormone therapy for low turnover bone fragility in osteopetrosis; the study s…
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CLINICAL_TRIALS trial_result · 2015-03-25Clinical Trial Update: Phase III Randomized Study of Interferon Gamma in Children With Severe, Congenital Osteopetrosis [Phase 3]
Phase III randomized trial evaluating interferon gamma in children with severe congenital osteopetrosis has completed.
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CLINICAL_TRIALS trial_result · 2011-08-22Clinical Trial Update: Stem Cell Transplantation for Children Affected With Osteopetrosis [NA]
A pediatric stem cell transplantation trial for osteopetrosis was terminated; results were posted on August 22, 2011.
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CLINICAL_TRIALS trial_result · 2010-09-10Clinical Trial Update: Clinical Assessment of Patients With High Bone Mass Due to Mutation in Lrp5
Completed clinical assessment of patients with high bone mass caused by LRP5 mutation in osteopetrosis.
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CLINICAL_TRIALS trial_result · 2010-02-03Clinical Trial Update: Reduced Intensity AlloTransplant For Osteopetrosis [Phase 2]
Phase 2 trial update on reduced-intensity allogeneic transplantation for osteopetrosis has been terminated with posted results in 2010.
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CLINICAL_TRIALS trial_result · 2007-11-01Clinical Trial Update: Post Marketing Surveillance Study of Actimmune in Patients With Severe, Malignant Osteopetrosis
Post-marketing surveillance study of Actimmune (interferon gamma-1b) in patients with severe, malignant osteopetrosis has completed.