65. 原発性免疫不全症候群
［臨床試験数：413，薬物数：581（DrugBank：97），標的遺伝子数：68，標的パスウェイ数：202］

Searched query = "Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase deficiency", "CD8 deficiency", "ZAP-70 deficiency", "MHC class I deficiency", "MHC class II deficiency", "Combined immunodeficiency", "Wiskott-Aldrich syndrome", "Telangiectasia ataxia", "Nijmegen breakage syndrome", "Bloom syndrome", "Immunodeficiency, centromere region instability, facial anomalies syndrome", "ICF syndrome", "PMS2 deficiency", "Radiosensitivity, immunodeficiency, dysmorphic features, and learning difficulties syndrome", "RIDDLE syndrome", "Schimke syndrome", "Netherton syndrome", "Thymic hypoplasia", "DiGeorge syndrome", "22q11.2 deletion syndrome", "Hyper-IgE syndrome", "Hepatic venoocclusive immunodeficiency", "Immunodeficiency with central hepatic vein atresia", "Dyskeratosis congenita", "X-linked agammaglobulinaemia", "Common variable immunodeficiency", "Hyper-IgM syndrome", "Isolated IgG subclass deficiency", "Selective IgA deficiency", "Specific antibody production deficiency", "Infant transient hypogammaglobulinemia", "Chédiak-Higashi syndrome", "Chediak-Higashi syndrome", "X-linked lymphoproliferative syndrome", "SAP deficiency", "SH2D1A/SLAM-associated protein deficiency", "XIAP deficiency", "X-linked inhibitor of apoptosis deficiency", "Autoimmune lymphoproliferative syndrome", "ALPS", "Familial hemophagocytic syndrome", "Perforin deficiency", "Munc13-4 deficiency", "Syntaxin 11 deficiency", "Munc18-2 deficiency", "Autoimmune polyendocrinopathy-candidiasis-ectodermal dystrophy", "APECED", "Immune dysregulation, polyendocrinopathy, enteropathy, X-linked syndrome", "IPEX syndrome", "CD25 deficiency", "ITCH deficiency", "Primary phagocytic dysfunction", "Severe congenital neutropenia", "Cyclic neutropenia", "Hermanskyi-Pudlak syndrome type 2", "Hermanskyi-Pudlak syndrome 2", "Griscelli syndrome type 2", "Griscelli syndrome 2", "p14 deficiency", "Warts, hypogammaglobulinemia, infections, myelokathexis syndrome", "WHIM syndrome", "Glycogen storage disease type Ib", "Leukocyte adhesion deficiency", "Shwachman-Diamond syndrome", "Chronic granulomatous disease", "Myeloperoxidase deficiency", "Mendelian susceptibility to mycobacterial disease", "MSMD", "Anhidrotic ectodermal dysplasia with immunodeficiency", "EDA-ID", "Interleukin-1 receptor-associated kinase-4 deficiency", "IRAK4 deficiency", "IMyD88 deficiency", "Chronic mucocutaneous candidiasis", "Epidermodysplasia verruciformis", "Herpes simplex encephalitis", "Caspase recruitment domain family member 9 deficiency", "CARD9 deficiency", "Trypanosomiasis", "Congenital complement deficiency", "C1q deficiency", "CC1r deficiency", "CC1s deficiency", "CC2 deficiency", "CC3 deficiency", "CC4 deficiency", "CC5 deficiency", "CC6 deficiency", "CC7 deficiency", "CC8 deficiency", "CC9 deficiency", "Factor D deficiency", "Properdin deficiency", "Factor I deficiency", "Factor H deficiency", "MASP1 deficiency", "3MC syndrome", "Mannose-binding protein-associated serine protease 2 deficiency", "MASP2 deficiency", "FCN3", "Hereditary angioedema type 1", "Hereditary angioedema type I", "C1 inhibitor deficiency type 1", "C1 inhibitor deficiency type I", "Hereditary angioedema type 2", "Hereditary angioedema type II", "C1 inhibitor deficiency type 2", "C1 inhibitor deficiency type II", "Hereditary angioedema type 3", "Hereditary angioedema type III", "C1 inhibitor deficiency type 3", "C1 inhibitor deficiency type III"  collapse"Primary immunodeficiency", "X-SCID", "Reticular dysgenesis", "Adenosine deaminase deficiency", "Omenn syndrome", "Purine nucleoside phosphorylase def ... show all
The queries were searched in Public_title, Scientific_title, and Condition. Export date: 03/15/2021. Trials are sorted by Date_enrollment from most recent to oldest in the table.

Drug = 16,5% Cutaquig; 36-482-Hyaluronoglucosaminidase PH20 (rHuPH20); A10BK03; ABATACEPT; ADA PBSC; ADA Umbilical Cord Blood Cells; ADA gene transfer; ALDESLEUKIN; ALEMTUZUMAB; AProArt; AUTOLOGOUS CD34+ CELLS TRANSDUCED EX-VIVO WITH THE PCCLCHIMGP91/VSVG LENTIVIRAL VECTOR; AUTOLOGOUS CD34+CELLS TRANSDUCED WITH THE W1.6_HWASP_WPRE (VSVG) LENTIVIRAL VECTOR; Abatacept; Acoziborole (SCYX-7158); Adagen; Adenosine; Administration of drug (Interferon-gamma 1-b) subcutaneously; Adverse Reactions of Gammagard subcutaneously at Week 12; Adverse Reactions of Gammagard subcutaneously at Week 24; Adverse Reactions of Gammagard subcutaneously at Week 36; Adverse Reactions of Gammagard subdermally at Week 12; Adverse Reactions of Gammagard subdermally at Week 24; Adverse Reactions of Gammagard subdermally at Week 36; Aldesleukin; Alefacept; Alemtuzumab; Alemtuzumab 0.2 mg; Alemtuzumab 0.3 mg; Alentuzumab (Campath); Allogeneic peripheral blood stem cell; Anti-CD45; Anti-Thymocyte Globulin (Rabbit); Anti-thymocyte globulin; Anti-thymocyte globulin (rabbit); Antithymocyte globulin; Autologous CD34+ HSCs transduced ex vivo with EFS lentiviral vector encoding for the human ADA gene; Autologous CD34+ cell transduced with G2SCID vector; Autologous CD34+ cells transduced with the Lentiviral vector containing the human Wiskott Aldrich Sy; Autologous CD34+ enriched cell fraction that contains CD34+ cells transduced with lentiviral vector that encodes for the human Wiskott Aldrich Syndrome (WAS) cDNA sequence; Autologous CD34+cells transduced with the w1.6_hWASP_WPRE (VSVg) lentiviral vector; Autologous ex vivo gene therapy products based on the EFS LV encoding for the human adenosine deaminase (ADA) gene (EFS-ADA LV); Azathioprine; B-Lymphocyte Stimulator (BLyS); BMS-188667; BPX-501 T cells; BT090; BT524; BT595; BT681; BUSILVEX - 6 MG/ML - CONCENTRATO PER SOLUZIONE PER INFUSIONE - USO ENDOVENOSO - FLACONCINO - 10 ML 8 FLACONCINI; BUSULFAN; Bacteriophage; Basiliximab; Benznidazole; Biological sampling; Bivigam; Busilvex; Busulfan; Busulfan IV; Busulfan test dose; Busulfan, Cyclophosphamide, ATG, GCSF; Busulfan, Fludarabine and ATG; Busulfan, Fludarabine, ATG, TLI; C1 ESTERASE INHIBITOR (HUMAN); C1 Esterase Inhibitor Human; C1 inhibitor; C1 inhibitor concentrate; C1-INH; C1-esterase inhibitor [recombinant] (C1-INH-R); CD3/CD19 neg allogeneic BMT; CD3/CD19 negative allogeneic hematopoietic stem cells; CD34 Stem Cell Selection Therapy; CD34+; CD34+ HSCs transduced with the lentivirus vector, VSV-G pseudotyped CL20-4i-EF1a-h?c-OPT; CD34+ cells transduced with ADA retrovir; CD34+CELLS; CD45RA-depleted DLI; CDZ173; CUTAQUIG; CUVITRU; Campath; Campath -1H; Campath 1H; Campath, Fludarabine, Cyclophosphamide; Chloride; Cryopreserved EFS-ADA LV transduced patient CD34+ cells; Cryopreserved G2SCID lentiviral vector transduced patient CD34+ cells; Cultured Thymus Tissue Implantation (CTTI); Cultured Thymus Tissue for Implantation (CTTI); Cuvitru; Cuvitru 200 mg/ml solution for subcutaneous injection; Cyclophosphamide; Cyclophosphamide (Cytoxan); Cyclophosphamide 30; Cyclophosphamide 40; Cyclophosphamide Dose Level 1; Cyclophosphamide Dose Level 2; Cyclophosphamide Dose Level 3; Cyclophosphamide Dose Level 4; Cyclophosphamide post transplant; Cyclosporine; Cyclosporins; DB289; DEXAMETHASONE SODIUM PHOSPHATE PH. EUR.; Daclizumab; Danazol; Data collection; Depletion in CD45RA graft donor; Device: Cell processing for TCRabeta+/CD19+ depletion; Device: Cell processing for TCRaß+/CD19+ depletion; Device: Chrono Super PID then Generic Syringe-Gammanorm; Device: CliniMACS; Device: CliniMACS® CD34 Reagent System cell sorter device; Device: CliniMacs; Device: Continuous Glucose Monitor; Device: Generic Syringe then Chrono Super PID-Gammanorm; Device: Isolex 300i Magnetic Cell Selector; Device: Miltenyi CliniMACS; Device: Miltenyi CliniMACS selection system; Device: unrelated PBSC with T cell depletion; Dexametasone Fosfato Sodico; Dexamethasone; Dexamethasone 21-dihydrogen phosphate; Dexamethasone sodium phosphate; Dextrose; Dextrose, 5% in Water; Diagnostic Test: Oral Glucose Tolerance Test; Diagnostic Test: RNA and neopterin detection; Diagnostic Test: blood drawing in healthy controls; Diagnostic Test: blood drawing in patients with WAS; Diagnostic Test: functional and antigenic C1 inhibitor; Donor peripheral blood stem cells.; EF1aS-ADA lentiviral vector gene modified autologous CD34+ cells; EF1aS-ADA lentiviral vector transduced patient CD34+ cells; EP2006; EP2006 (Filgrastim); EZN-2279; Efficacy of Gammagard subcutaneously at Week 12; Efficacy of Gammagard subcutaneously at Week 24; Efficacy of Gammagard subcutaneously at Week 36; Efficacy of Gammagard subdermally at Week 12; Efficacy of Gammagard subdermally at Week 24; Efficacy of Gammagard subdermally at Week 36; Eflornithine; Eflornithine plus Nifurtimox combination therapy; Elapegademase; Elapegademase-lvlr; Eltrombopag; Empagliflozin; Etoposide; Ex vivo culture and transduction of the patient's autologous CD34+ HSC with lentivirus vector VSV-G pseudotyped CL20- 4i-EF1a-hyc-OPT vector; FILGRASTIM; FLUDARABINA; FLUDARABINA TEVA - 25 MG/ML CONCENTRATO PER SOLUZIONE INIETTABILE O PER INFUSIONE 1 FLACONCINO DI VETRO DA 2 ML; FLUDARABINE; Fansidar (pyrimethamine and sulfadoxine); Fexinidazole; Fibrinogen; Fibrinogen (coagulation factorI); Fibrinogen Concentrate from Human Plasma; Filgrastim; Filgrastim Hexal; Filgrastim, Alemtuzumab; Flebogamma 5% DIF; Fludarabina; Fludarabina Accord; Fludarabine; Fludarabine Phosphate; Fludarabine monophosphate; Fludarabine phosphate; Fludarabine phosphate 30 mg; Fludarabine phosphate 40 mg; Fludarabine, Busulfan, Thymoglobulin; Fludarabine, Melphalan, Thiotepa; Fortecortin; Fortecortin Inject 40 mg Amp.; Fucose; G-CSF; G-CSF for Conditioning before HSCT.; G1XCG; G1XCGD transduced CD34+ cells; GAMMAGARD LIQUID; GAMUNEX-C; GTG003.08; GVHD Prophylaxis; Gadolinium; Gadolinium For abdomen; Gadolinium For lower back; Gammagard S/D (Solvent/Detergent); Gammaglobulin; Gammanorm; Gammanorm 165 mg/mL; Gammaplex; Gammaplex (5%); Gammaplex (Intravenous immunoglobulin); Gammaplex 10; Gamunex-C; Gcsf; Gene Therapy Method for CGD; Gene transfer; Gene-Transduced Autologous CD34+ Stem Cells; Genetic: CL20-i4-EF1a-h?c-OPT; Genetic: Infusion of autologous EFS-ADA LV CD34+ (OTL-101); Genetic: Infusion of autologous EFS-ADA LV CD34+ cells; Genetic: Infusion of autologous cryopreserved EFS-ADA LV CD34+ cells (OTL-101); Genetically modified autologous blood stem cells; Glucose; Glycolic acid; Glycosade; Gold; Granulocyte Colony Stimulating Factor; Granulocyte colony stimulating factor (G-CSF); HPC, A Infusion; HTLP; HUMAN FIBRINOGEN; HUMAN NORMAL IMMUNOGLOBULIN; HUMAN NORMAL IMMUNOGLOBULIN (IV); HUMAN NORMAL IMMUNOGLOBULIN FOR; HUMAN NORMAL IMMUNOGLOBULIN FOR INTRAVENOUS USE; HYALURONIDASE; HYQVIA; Haemocomplettan P; Haemocomplettan(R) P; Haemocomplettan(R) P 1g/2g; Haplo BM with T cell depletion; Haploidentical Hematopoietic Cell Transplantation; Hematopoetic Stem Cell Transplantation; Hematopoietic Stem Cell Transplant; Hizentra; Hizentra®; Horse -Anti-thymocyte; Human Fibrinogen Concentrate; Human Immunglobulin G (IgG); Human Immunoglobulin; Human Normal Immunoglobulin; Human Normal Immunoglobulin (Subcutaneous - Intramuscular Immunoglobulin); Human Normal Immunoglobulin G (IgG > 98% purity); Human Normal Immunoglobulin for Subcutaneous Administration; Human Normal Immunoglobulin for Subcutaneous Administration (IGSC); Human Normal Immunoglobulin for subcutaneous administration; Human Normal immunoglobulin; Human fibrinogen; Human immunoglobulin G; Human normal immunoglobulin; Human normal immunoglobulin (IVIg); Human normal immunoglobulin (subcutaneous); Human normal immunoglobulin for intravenous administration; Human normal immunoglobulin for intravenous use (IVIG); HyQvia; HyQvia 100 mg/ml solution for infusion for subcutaneous use; Hyaluronidase; Hyaluronoglucosaminidase; Hydroxyurea; Hyqvia; I10E; IBUPROFEN; IFN-gamma; IGIV, 10%; IGIV-C 10%; IGNG; IGSC 20%; IGSC 20% 150 mg/kg; IGSC 20% daily push versus 2 times per week pump; IGSC 20% daily push versus every 2 weeks pump; IGSC 20% daily push versus once a week pump; IGSC, 16%; IGSC, 20%; IMMUNOGLOBULIN G; INH; INTERLEUKIN-2; IV treatment with IGSC, 10%; IVIG; IVIG-PEG; IVIg; Ibuprofen; Ig VENA 50 g/l solution for infusion 100 ml vial + infusion set; IgG Next Generation; IgHy10; IgNextGen 16%; IgPro10; IgPro20; Immune Globulin Infusion (Human), 10%; Immune Globulin Intravenous; Immune Globulin Intravenous (Human); Immune Globulin Intravenous (Human) 5% Liquid, IVIG-SN™; Immune Globulin Intravenous (Human), 10%; Immune Globulin Intravenous (Human), 10% Solution; Immune Globulin Intravenous (Human), 10% TVR (Triple Virally Reduced) Solution; Immune Globulin Intravenous (IGIV); Immune Globulin Intravenous [Human], 10% Caprylate/Chromatography Purified; Immune Globulin Subcutaneos, 20%; Immune Globulin Subcutaneous (Human) (SCIG); Immune Globulin Subcutaneous (Human), 20%; Immune Globulin Subcutaneous (Human), 20% Caprylate/Chromatography Purified (IGSC 20%); Immune Globulin Subcutaneous (Human), 20% Solution; Immune Globulin Subcutaneous (Human), 20%, Caprylate/Chromatography Purified; Immune Globulin Subcutaneous, 20%; Immune Globulin Subcutaneous, 20% Solution (IGSC, 20%); Immune globulin subcutaneous (Human); Immune globulin subcutaneous (human); Immunglobulin (human); Immunoglobulin G; Immunoglobulin G (Ig NextGen 16%); Immunoglobulin intravenous (human); Immunoglobulins Intravenous (Human); Immunoglobulins, normal human; Immunological diagnosis tests; Immunosuppression Only Conditioning (IOC); Immunosuppression Only Conditioning - Closed with amendment L; Increlex; Infliximab; Interferon Gamma; Interferon Gamma-1B; Interferon gamma; Interferon gamma-1b; Interferon-gamma; Interleukin-2; Intratect; Intravenous immunoglobulin infusion; Intravenous infusion of transduced cells; Itraconazole; Jardiance; KIOVIG; KIOVIG 100 mg/ml solution for infusion; KIg10; KLH; KVD900; KVD900 100 mg Film Coated Tablet; Kedrion IVIG 10%; Kineret; L-fucose; LADICell; LDT; LENOGRASTIM; LFB-IgSC; Lemtrada; Leniolisib; Lenograstim; Lentiviral G1XCGD Gene Therapy; Lentiviral vector transduced CD34+ cells; MABTHERA - 2 FIALE 100 MG 10 ML; MCTLs; MESNA; MMF; MONITOR; MOZOBIL - 20 MG/ML - SOLUZIONE INIETTABILE - USO SOTTOCUTANEO - FLACONCINO (VETRO) - 24 MG/1.2 ML 1 FLACONCINO; MYELOSTIM; MYELOSTIM - 34 1 FLACONCINO LIOFILIZZATO 33.6 MIU + SIRINGA PRERIEMPITA SOLVENTE 1 ML; MYELOSTIM 34 milions UI/ml - powder and solvent for solution for injection/infusion; MYELOSTIM 34 milions UI/ml, powder and solvent for solution for injection or infusion; MabThera; Mavorixafor; Melarsoprol; Melarsoprol 1.8 mg/kg/d, 10d + eflornithine 400 mg/kg/d, 7d; Melarsoprol 1.8 mg/kg/d, 10d + nifurtimox 15/20 mg/kg/d, 10d; Melphalan; Mesna; Methotrexate; Methylprednisolone; Methylprednisolone or Prednisolone; Mozobil; Mozobil 20mg/mL vial (injectable solution for subcutaneous use); Mozobil 20mg/mL vial (injectable solution, subcutaneous use); Mozobil,; Mycophenolate; Mycophenolate Mofetil; Mycophenolate mofetil; Mycophenolate mofetil (MMF); Myeloablative Conditioning-Closed with amendment L; Myeloablative Preparative Regimen; Myelostim; NDV-3A; Nanogam 100 mg/ml; Nanogam® 50 mg/ml; Neopterin; NewGAM; NewGam; Newnorm; Nifurtimox; Nifurtimox 15/20 mg/kg/d 10d + eflornithine 400 mg/kg/d 7d; Nifurtimox-Eflronithine Combination Treatment (NECT); None; Normal human immunoglobulin G; Noxafil; Noxafil 40 mg/ml oral solution; OCTA-C1-INH; OCTAGAM; OCTAGAM 50 mg/ml oldatos infúzió; ORENCIA; ORENCIA® 125 mg solution for injection in pre-filled syringe; OTL-101; OTL-103; OTL-103 Dispersion for Infusion; OVASTAT; Octagam 10%; Octagam 5%; Octanorm; Octanorm 16.5%; Omalizumab; Omalizumab (Xolair); Orencia; Other hematological Agents; Other:; Other: 0.9% sodium chloride; Other: Blood sampling for Laboratory Developed Test (LDT) analysis; Other: Cultured Thymus Tissue Implantation and Parental Parathyroid Transplantation; Other: Cultured Thymus Tissue Implantation with Parathyroid Transplantation; Other: Donor Lymphocyte Infusion; Other: Food Diary; Other: Haematopoietic Stem Cell Transplantation (HSCT); Other: Laboratory Biomarker Analysis; Other: Medical History Questionnaires; Other: Modified Mixed Meal Tolerance Test; Other: Modified Oral Glucose Tolerance Test; Other: Palliative Care; Other: laboratory biomarker analysis; Ovastat 1000; Ovastat 1000 (Treosulfan injection); Ovastat 1000 mg, powder for solution for infusion; Ovastat 5000; Ovastat 5000 (Treosulfan injection); Ovastat 5000 mg, powder for solution for infusion; PANTOPRAZOLE; PEG; PEG-ADA ERT; PEG-interleukin-2; PHA-022121; PID; PLERIXAFOR; PNEUMO 23; POSACONAZOLE; PPS; PREVENAR; PROLEUKIN® S; PSRS11.EFS.IL2RG.pre* retroviral vector; PSRS11.EFS.IL2RG.pre* retroviral vector transduce; PSRS11.EFS.IL2RG.pre* retroviral vector transduced cells; PTH; PTH 1-34; Palifermin; Pantoprazole; Pantoprazolo 20 mg gastro-resistant tablets; Peg-Ada; Pentamidine; Peripheral blood stem cells; Phagocyte Oxidase Subunit Transduced CD34 Hematopoietic Stem Cells; Phosphate; Pioglitazone; Plerixafor; Plerixafor for Conditioning before HSCT.; PnCJ PPS; Polyclonal IgG; Posaconazole; Posaconazole (PSZ); Prednisolone; Prednisone; Privigen; Privigen®; Procedure: Allo BMT; Procedure: Allogeneic Bone Marrow Transplantation; Procedure: Allogeneic HSC; Procedure: Allogeneic HSCT; Procedure: Blood Draw; Procedure: Blood Sample; Procedure: CT Scan; Procedure: Hematopoietic stem cell transplantation; Procedure: Nonmyeloablative Allogeneic Hematopoietic Stem Cell Transplantation; Procedure: Peripheral Blood Stem Cell Transplantation; Procedure: Stem Cell Infusion; Procedure: Stem Cell Transplantation; Procedure: Stem cell infusion; Procedure: Stem cell transplant; Procedure: Total Body Irradiation; Procedure: Total lymphoid irradiation; Procedure: allogeneic bone marrow transplantation; Procedure: allogeneic hematopoietic stem cell transplantation; Procedure: cyclophosphamide; Procedure: in vitro-treated peripheral blood stem cell transplantation; Procedure: peripheral blood stem cell transplantation; Procedure: umbilical cord blood transplantation; Proleukin; Promacta; Prometic's Immune Globulin Intravenous 10%; Purified Vero Cell Vaccine); Pyrimethamine; R-hIL-18BP; RAG1 LV CD34+ cells; RI-002; RITUXIMAB; RP-L201; RTX; Rabbit; Rabbit anti-thymocyte globulin; Radiation: Total Body Irradiation; Radiation: Total Body Irradiation (TBI); Radiation: Total Lymphoid Irradiation (TLI); Radiation: Total body 200cGy; Radiation: Total body Irradiation; Radiation: Total-Body Irradiation; Radiation: radiation therapy; Radiation: total-body irradiation; Ranitidine; Recombinant Human Hyaluronidase (rHuPH20); Recombinant human hyaluronidase; Recombinant human hyaluronidase (rHuPH20)+ immune globulin intravenous (IGIV); Recombinant human hyaluronidase + immune globulin intravenous; Reduced Intensity Conditioning; Reduced Intensity Conditioning (RIC); Reduced Intensity Preparative Regimen; Reduced Toxicity Ablative Regimen; Retroviral SF71-gp91phox transduced CD34+ cells; Retrovirus-mediated gene transfer; RhuCD40L; Rifaximin; Rimiducid; Rituximab; Rituximab (RTX) and Azathioprine (AZA); Rivogenlecleucel; Romiplostim; SC treatment with IGSC, 10% with rHuPH20 followed by IV/IGSC, 10% only (safety); SC treatment with IGSC, 10% with rHuPH20 followed by SC/IGSC, 10% only (safety); SCID screening; SCYX-7158; STA-5326; STRIMVELIS; SUBCUVIA; Sirolimus; Sodium chloride; Somatic gene-therapy by X-CGD; Stelara (ustekinumab); Stem Cell Transplant; Stem Cell Transplantation; Strimvelis; Subgam; Sulfadoxine; Suspension of autologous CD34+cells transduced with the G1XCGD viral vector; T-Cell Depleted & CD34+Select/w/StemCell Enriched Product; TADEKINIG ALFA; TBX-1400; TCR alfa beta T cell depletion; TREOSULFAN; TYF-IL-2Rg gene-modified autologous stem cells; Tabelecleucel; Tablets Fexinidazole; Tacrolimus; Tacrolimus (Tacro); Tadekinig alfa; Thalidomide; Therapeutic allogeneic lymphocytes; There is no recommended INN; Thiotepa; Thiotepa--escalated dose; Thiotepa--single daily dose; Thymoglobulin; Thymus Tissue for Transplantation; Thymus/Parathyroid Transplantation; Traditional treatment of CGD and TB; Transduced Lymphocytes; Transduced patient CD34+ cells; Transplant Conditioning with Mobilization Only; Transplant Conditioning with Mobilization and Alemtuzumab; Transplant preparative regimen of alemtuzumab, fludarabine, thiotepa, and melphalan; Treosulfan; Unrelated BM with T cell depletion; Unrelated cord blood; Ustekinumab; VM106; Valacyclovir; Valproic Acid; Valproic acid; Verorab® (PVRV; Vigantol; Vitamin D3; Vivaglobin; Water; X4P-001; Xifaxan; Zarzio; Zoledronate; [NA]; [na]