Functional neutrophil defect
MONDO:0015978Also known as: neutrophil disease, neutrophilopathy
13 clinical trials for this condition and its sub-types, 0 tagged with Functional neutrophil defect itself.
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Sub-types of Functional neutrophil defect
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Leukocyte adhesion deficiency 3 trials · 7 incl. sub-types
3 sub-types
- Leukocyte adhesion deficiency 1 7 trials
- Leukocyte adhesion deficiency type II 2 trials
- Leukocyte adhesion deficiency 3 0 trials
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Papillon-Lefevre disease 5 trials
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Myeloperoxidase deficiency 1 trial
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Neutrophil immunodeficiency syndrome 0 trials
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Specific granule deficiency 0 trials
2 sub-types
- Specific granule deficiency 1 0 trials
- Specific granule deficiency 2 0 trials
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New hope for ALS patients: experimental drug CNM-Au8 now available through expanded access
Disease control Expanded access (paused)This program provides early access to an experimental drug called CNM-Au8 for people with amyotrophic lateral sclerosis (ALS), a progressive nerve disease. Participants must be 18 or older and have a confirmed ALS diagnosis. The goal is to offer treatment to those who cannot join…
Sponsor: Clene Nanomedicine • Aim: Disease control
Last updated Jun 27, 2026 12:04 UTC
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Gene therapy for rare immune disease shows promise in Long-Term study
Disease control OngoingThis study follows 9 people with a rare immune disorder called LAD-I who received a one-time gene therapy that adds a working copy of the ITGB2 gene to their own blood stem cells. Researchers want to see if the treatment safely controls the disease over many years and helps patie…
Sponsor: Rocket Pharmaceuticals Inc. • Aim: Disease control
Last updated Jun 27, 2026 08:05 UTC
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Desperate patient gets experimental stem cells for rare nerve disease
Disease control Expanded access (ended)This study provides an experimental stem cell treatment (HB-adMSCs) to an 83-year-old man with Primary Lateral Sclerosis, a rare nerve disease that causes progressive muscle weakness. The patient's own banked stem cells are used, aiming to slow the disease. This is a single-patie…
Sponsor: Hope Biosciences Research Foundation • Aim: Disease control
Last updated Jun 27, 2026 07:52 UTC