FrancophonIA/ELRC3.0 huggingface dataset:
English: Tables 1, 2, and 3 are lists of adverse reactions attributed to mobilisation/apheresis, myeloablative conditioning, and Zynteglo, respectively, experienced by patients with TDT in clinical trials with Zynteglo.
Maltese: It-Tabelli 1, 2, u 3 huma listi ta' reazzjonijiet avversi attribwiti għal mobilizzazzjoni/aferesi, kondizzjonament majeloablattiv, u Zynteglo, rispettivament, esperjenzati minn pazjenti b'TDT fi provi kliniċi b'Zynteglo.
English: Store at ≤-140°C for up to a year.
Maltese: Aħżen f'temperatura ta' ≤-140°C sa sena.
English: Transfusion-dependent β-thalassaemia (TDT)
Maltese: Talassemija β dipendenti fuq it‑trasfużjoni (TDT, Transfusion‑dependent β‑thalassaemia)
English: This is equivalent to 20-78% of the recommended maximum daily dietary intake of sodium for an adult.
Maltese: Dan hu ekwivalenti għal 20-78% tat-teħid massimu ta' kuljum ta' sodium fid-dieta rakkomandat għal persuna adulta.
English: Rash, Hyperhidrosis
Maltese: Raxx, Iperidrożi
English: - Keep this leaflet.
Maltese: trasfużjonijiet ta' plejtlits.
English: {DD month YYYY}
Maltese: U MNEJN GĦANDU JINGĦATA
English: Reliable methods of contraception include intra-uterine device or a combination of oral contraceptive (also know as the pill) and condoms.
Maltese: Metodi affidabbli ta' kontraċettiv jinkludi apparat intraurinarju jew taħlita ta' kontraċettiv orali (magħruf ukoll bħala l-pillola) u kondoms.
English: Lot Number / COI ID
Maltese: Lott Numru /
English: The median (min, max) duration of follow-up was 40.48 (29.3, 58.6) months.
Maltese: It-tul medju (min, mass) tas-segwitu kien ta' 40.48 (29.3, 58.6) xhur.
English: The median reduction in liver iron content from baseline was 67.14%, ranging from an 83.3% reduction to a 269.2% increase (N=3, HGB-204; N=2, HGB-205).
Maltese: It-tnaqqis medjan fil-kontenut tal-ħadid fil-fwied mil-linja bażi kien 67.14%, li jvarja minn tnaqqis ta' 83.3% sa żieda ta' 269.2% (N=3, HGB-204; N=2, HGB-205).
English: In the clincal studies, patients received a median (min, max) RBC transfusion volume of 175.7 (139, 251) mL/kg/year and a median (min, max) number of 14.8 (10, 40) RBC transfusions per year.
Maltese: Fl-istudji kliniċi, il-pazjenti ngħataw volum medjan (min, mass) ta' trasfużjoni ta' RBC ta' 175.7 (139, 251) mL/kg/sena u numru medjan (min, mass) ta' 14.8 (10, 40) trasfużjonijiet RBC kull sena.
English: Zynteglo 1.2-20 × 106 cells/mL dispersion for infusion (autologous CD34+ cells encoding βA-T87Q-globin gene)
Maltese: Zynteglo 1.2-20 x 106 ċelluli/mL dispersjoni għall-infużjoni (ċelluli CD34+ awtologi li jikkodifikaw il-ġene βA-T87Q-globin)
English: Among these 4 patients, the median (min, max) weighted average Hb during TI was 12.42 (11.5, 12.6) g/dL (Table 5).
Maltese: Fost dawn l-4 pazjenti, l-Hb medju peżat medjan (min, mass) matul TI kien ta' 12.42 (11.5, 12.6) g/dL (Tabella 5).
English: The median (min, max) serum ferritin at baseline was 3778.7 (784, 22517) pmol/L and median (min, max) liver iron concentration was 6.75 (1.0, 41.0) mg/g (N=10, HGB-204; N=4, HGB-205; N=15, HGB-207; N=3, HGB-212).
Maltese: Il-ferritin medjan fis-serum (min, mass) fil-linja bażi kien 3778.7 (784, 22517) pmol/L u l-konċentrazzjoni medjana (min, mass) tal-ħadid fil-fwied kienet 6.75 (1.0, 41.0) mg/g (N=10, HGB-204; N=4, HGB-205; N=15, HGB-207; N=3, HGB-212).
English: Bag ID
Maltese: ID COI
English: Final results: December 2024
Maltese: Diċembru 2024
English: The median (min, max) age in the studies was 19.0 (12, 34) years, 56.3% were females, 59.4% were Asian, and 40.6% White/Caucasian.
Maltese: (12, 34) sena, 56.3% kienu nisa, 59.4% kienu Asjatiċi, u 40.6% Bojod/Kawkasi.
English: Marketing Authorisation Holder and Manufacturer bluebird bio (Netherlands) B.V. Stadsplateau 7
Maltese: Detentur tal-Awtorizzazzjoni għat-Tqegħid fis-Suq u l-Manifattur bluebird bio (Netherlands) B.V. Stadsplateau 7
English: Cholecystitis, Cholelithiasis, Hepatomegaly, Jaundice, Transaminases increased, Gamma-glutamyltransferase increased
Maltese: Koliċistite, Kolelitjażi, Epatomegalija, Suffejra, Żieda fittransaminases, Żieda fil-gamma glutamyltransferase
English: The Netherlands e-mail: medinfo@bluebirdbio.com
Maltese: L-Olanda e-mail: medinfo@bluebirdbio.com
English: The planned dose of G-CSF was 10 µg/kg/day in patients with a spleen, and 5 µg/kg/day in patients without a spleen, given on Days 1 through 5 of mobilisation in the morning.
Maltese: Id-doża ppjanata ta' G-CSF kienet ta' 10 µg/kg/jum f'pazjenti b'milsa, u 5 µg/kg/jum f'pazjenti mingħajr milsa, mogħtija f'Jiem 1 sa 5 ta' mobilizzazzjoni filgħodu.
English: For men, this may be sperm or testicular tissue.
Maltese: Għall-irġiel, dan jista' jkun sperma jew tessut testikulari.
English: • decrease in magnesium, calcium, potassium, phosphate, albumin, sodium in blood
Maltese: • tnaqqis fil-manjeżju, kalċju, potassju, fosfat, albumina, sodju fid-demm
English: Failure of neutrophil engraftment is a short-term but potentially severe risk, defined as failure to achieve 3 consecutive absolute neutrophil counts (ANC) ≥500 cells/µL obtained on different days by Day 43 after infusion of
Maltese: Nuqqas fiż-żieda fl-għadd tan-newtrofili huwa riskju għal żmien qasir iżda potenzjalment gravi, definit bħala nuqqas li jinkisbu 3 għadd ta' newtrofili assoluti konsekuttivi (ANC) ≥500 ċelluli/µL miksuba f'jiem differenti sa Jum 43 wara l-infużjoni ta' Zynteglo.
English: This medicine contains 391-1564 mg sodium (main component of cooking/table salt) in each dose.
Maltese: Din il-mediċina fiha 391-1564 mg sodium (komponent ewlieni ta' melħ tat-tisjir/tal-mejda) f'kull doża.
English: Strength cells/mL)
Maltese: (× 106 ċell uli/mL)
English: HbAT87Q remained generally stable through Month 24 with a median (min, max) of 6.44 (1.1, 10.1) g/dL (N=14, HGB-204 and HGB-205), and through long-term follow-up in LTF-303, demonstrating stable integration of the βA-T87Q-globin gene into long-term HSCs and stable expression of the βA-T87Q-globin gene in cells of the erythroid lineage.
Maltese: HbAT87Q baqgħu ġeneralment stabbli matul ix-Xahar 24 b'medjan (min, mass) ta' 6.44 (1.1, 10.1) g/dL (N=14, HGB-204 u HGB-205), u permezz ta' segwitu fit-tul f'LTF-303, li juru integrazzjoni stabbli tal-βA-T87Q ġene globin f'HSCs fit-tul u espressjoni stabbli tal-βA-T87Q ġene globin fiċ-ċelluli tan-nisel eritrojd.
English: ISA of post-transplantation BMCs demonstrated no preferred integration in the proximity of or within genes associated clinically (for gamma retroviral vectors) with either clonal dominance or leukaemia, and no evidence of clonal dominance was observed.
Maltese: ISA ta' BMCs ta' wara t-trapjant ma wriet l-ebda integrazzjoni ppreferuta fil-viċinanza ta' jew ġo ġeni assoċjati klinikament (għall-vetturi gamma retrovirali) bi jew dominanza klonali jew lewkimja, u l-ebda evidenza ta' dominanza klonali ma kienet osservata.
English: The back-up collection may be needed for rescue treatment if there is: 1) compromise of Zynteglo after initiation of myeloablative conditioning and before Zynteglo infusion, 2) primary engraftment failure, or 3) loss of engraftment after infusion with Zynteglo (see section 4.4).
Maltese: Tista' tkun meħtieġa kollezzjoni ta' back-up għal kura ta' salvataġġ jekk ikun hemm: 1) kompromess ta' Zynteglo wara l-bidu ta' kondizzjonament majeloablattiv u qabel l-infużjoni ta' Zynteglo, 2) falliment primarju tat-trapjant, jew 3) telf tat-trapjant wara infużjoni b'Zynteglo (ara sezzjoni 4.4).
English: (First Infusion Bag)
Maltese: Borża tal-Infużjoni)
English: Patients with TDT treated with Zynteglo who achieved platelet engraftment had a median (min, max) platelet engraftment on Day 41.0 (19, 191) in clinical trials (N=39).
Maltese: Pazjenti b'TDT ikkurati b'Zynteglo li kisbu trapjant tal-plejtlits kellhom trapjant tal-plejtlits medjan (min, mass) f'Jum 41.0 (19, 191) fi provi kliniċi (N=39).
English: Only a few patients have been included in the clinical studies with genotypes characterised by low endogenous β-globin production phenotypically similar to patients with a β0/β0 genotype, such as patients homozygous for IVS-I-110 or IVS-I-5.
Maltese: Ftit pazjenti biss ġew inklużi fl-istudji kliniċi b'ġenotipi kkaratterizzati minn produzz endoġena baxxa talβ-globin fenotipikament simili għal pazjenti b'ġenotip β0/β0, bħal pazjenti li huma omożigotiċi għal IVS-I-110 jew IVS-I-5.
English: The minimum recommended dose of Zynteglo is 5.0 × 106 CD34+ cells/kg.
Maltese: Id-doża rakkomandata minima ta' Zynteglo hija 5.0 × 106 ċelluli CD34+/kg.
English: **Transfusion independence (TI): a weighted average Hb ≥9 g/dL without any RBC transfusions for a continuous period of ≥12 months at any time during the study after medicinal product infusion.
Maltese: **Indipendenza tat-trasfużjoni (TI): Hb medju peżat ≥9 g/dL mingħajr trasfużjonijiet ta' RBC għal perjodu kontinwu ta' ≥12-il xahar fi kwalunkwe ħin matul l-istudju wara l-infużjoni ta' prodott mediċinali.
English: Hb (g/dL) at 6 months*
Maltese: (g/dL) wara 6 xhur*
English: Febrile neutropenia, Neutropenia, Thrombocytopenia, Leukopenia, Anaemia
Maltese: Newtropenja, Tromboċitopenija, Lewkopenija, Anemija
English: The median (min, max) total Hb at Month 6 for patients who had not received a transfusion for the prior 60 days was 11.90 (8.4, 13.3) g/dL (N=11).
Maltese: L-Hb medjan (min, mass) totali f'Xahar 6 għal pazjenti li ma kinux irċivew trasfużjoni għas-60 jum preċedenti kien ta' 11.90 (8.4, 13.3) g/dL (N=11).
English: Pharmacotherapeutic group: Other haematological agents, ATC code: not yet assigned Mechanism of action
Maltese: Aġenti ematoloġiċi oħra, Kodiċi ATC: mhux assenjat
English: Before treatment with Zynteglo, you will be given medicines know as mobilisation medicine and chemotherapy medicine (see sections 3 and 4 for more information on these medicines, including possible side effects).
Maltese: Qabel il-kura b'Zynteglo, inti se tingħata mediċini magħrufa bħala mediċina ta' mobilizzazzjoni u mediċina ta' kimoterapija (ara sezzjonijiet 3 u 4 għal aktar informazzjoni dwar dawn il-mediċini, inkluż effetti sekondarji possibbli).
English: Name and address of the manufacturer(s) responsible for batch release apceth Biopharma GmbH Haidgraben 5
Maltese: Isem u indirizz tal-manifattur(i) responsabbli għall-ħruġ tal-lott apceth Biopharma GmbH Haidgraben 5
English: According to available data, the frequency, type, and severity of adverse reactions in adolescents 12-17 years of age are similar to adults with the exception of VOD and pyrexia that occurred more frequently in adolescents.
Maltese: Skont id-data disponibbli, il-frekwenza, it-tip, u s-severità tar-reazzjonijiet avversi fl-adolexxenti ta' bejn 12-17-il sena huma simili għall-adulti, bl-eċċezzjoni ta' VOD u deni li seħħew aktar frekwenti fl adolexxenti.
English: The nature of Zynteglo is such that conventional studies on pharmacokinetics, absorption, distribution, metabolism, and elimination are not applicable.
Maltese: In-natura ta' Zynteglo hija tali li studji konvenzjonali dwar il-farmakokinetiċi, l-assorbiment, id-distribuzzjoni, il-metaboliżmu, u l-eliminazzjoni ma japplikawx.
English: The safety of Zynteglo was evaluated in 42 patients with TDT.
Maltese: Is-sigurtà ta' Zynteglo kienet evalwata f'42 pazjent b'TDT.
English: Of the patients with a non-β0/β0 genotype, 11/14 (78.6%, 95% CI 49.2%-95.3%) achieved TI by Month 24 (Table 5).
Maltese: Mill-pazjenti b'ġenotip mhux β0/β0, 11/14 (78,6%, 95% CI 49.
English: Back pain, Musculoskeletal discomfort
Maltese: Uġigħ fid-dar, skumdità muskoluskeletali
English: Efficacy was based on 32 adult and adolescent patients with TDT and a non-β0/β0 genotype treated with Zynteglo (N=10, HGB-204; N=4, HGB-205; N=15, HGB-207; N=3, HGB-212) (see Table 4).
Maltese: Effikaċja klinika L-effikaċja kienet ibbażata fuq 32 pazjent adult u adolexxenti b'TDT u ġenotip mhux β0/β0 ikkurati b'Zynteglo (N=10, HGB-204; N=4, HGB-205; N=15, HGB-207; N=3, HGB-212) (ara Tabella 4).
English: In this study, there was no evidence of toxicity, genotoxicity or oncogenesis (tumorigenicity) related to BB305 LVV integration, and no toxicity related to production of βA-T87Q-globin.
Maltese: relatata mal-integrazzjoni ta' BB305 LVV, u l-ebda tossiċità relatata mal-produzzjoni ta' βA-T87Q-globin.
English: Bone marrow biopsies taken before treatment were consistent with a diagnosis of TDT, including a low myeloid/erythroid ratio (N=15, HGB-207), reflective of erythroid hyperplasia.
Maltese: Bijopsiji tal-mudullun li ttieħdu qabel il-kura kienu konsistenti ma' dijanjożi ta' TDT, inkluż proporzjon majelojde/eritrojdju baxx (N=15, HGB-207), li jirrifletti iperplasija tal-eritrojdju.
English: NA*** 4/5
Maltese: TI** n/N^ (%)
English: Zynteglo is indicated for the treatment of patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, for whom haematopoietic stem cell (HSC) transplantation is appropriate but a human leukocyte antigen (HLA)-matched related HSC donor is not available (see sections 4.4 and 5.1).
Maltese: Zynteglo huwa indikat għall-kura ta' pazjenti ta' 12-il sena u ikbar b'talassemija β dipendenti fuq it-trasfużjoni (TDT) li m'għandhomx ġenotip β0/β0, li għalihom huwa xieraq it-trapjant ta' ċelluli staminali ematopojetiċi (HSC) iżda donatur ta' HSC relatat mal-antiġen tal-lewkoċit uman (HLA) imqabbel ma jkunx disponibbli (ara sezzjonijiet 4.4 u 5.1).
English: Headache, Peripheral sensory neuropathy
Maltese: Disturbi fis-sistema nervuża Uġigħ ta' ras, newropatija sensorjali periferali
English: The median (min, max) time to last RBC transfusion was 0.46 (0.2, 5.8) months following Zynteglo infusion.
Maltese: Iż-żmien medjan (min, mass) għall-aħħar trasfużjoni ta' RBC kien ta' 0.46 (0.2, 5.8) xhur wara l-infużjoni ta' Zynteglo.
English: Zynteglo is a type of medicine called gene therapy.
Maltese: Zynteglo huwa tip ta' mediċina msejħa terapija tal-ġene.
English: 20 mL fluoro-ethylenepropylene infusion bag(s), each packed in a transparent pouch inside a metal cassette.
Maltese: Borża (boroż) tal-infużjoni fluoro-ethylenepropylene ta' 20 mL, kull waħda ppakkjata f'borża trasparenti ġo kasett tal-metall.
English: Data are available on the risk of infertility with myeloablative conditioning.
Maltese: Hemm dejta disponibbli dwar ir-riskju ta' infertilità b'kondizzjonament majeloablattiv.
English: Cryostor CS5 Sodium chloride
Maltese: Sodium chloride
English: INSTRUCTIONS ON USE
Maltese: Mobilizzazzjoni u aferesi
English: Once thawed store at room temperature (20°C-25°C) and use within 4 hours.
Maltese: Ladarba jinħall aħżen f' temperatura ambjentali (20°C-25°C) u uża fi żmien 4 sigħat.
English: All patients should be tested for HIV-1/2 and HTLV-1/2 prior to mobilisation and apheresis to ensure acceptance of the apheresis material for Zynteglo manufacturing (see section 4.2).
Maltese: Il-pazjenti kollha għandhom jiġu ttestjati għal HIV-1/2 u HTLV-1/2 qabel il-mobilizzazzjoni u laferesi sabiex tiġi żgurata l-aċċettazzjoni tal-materjal ta' aferesi għall-manifattura ta' Zynteglo (ara sezzjoni 4.2).
English: Neutrophil engraftment occurred on median (min, max) Day 19.5 (13, 38) after medicinal product infusion.
Maltese: It-trapjant tan-newtrofili seħħ bħala medjan (min, mass) f'Jum 19.5 (13, 38) wara l-infużjoni tal-prodott mediċinali.
English: All patients who have achieved TI have maintained TI with a min, max duration of TI of 12.0+, 18.2+ months (N=4).
Maltese: Il-pazjenti kollha li kisbu TI żammew TI b'tul ta' TI min, mass ta'12.0+, 18.2+ xhur (N=4).
English: The minimum recommended dose is the same for adults and adolescents 12 years of age and older.
Maltese: Id-doża minima rakkomandata hi l-istess għall-adulti u għall adolexxenti ta' 12-il sena jew iżjed.
English: The planned dose of plerixafor was 0.24 mg/kg/day, given on Days 4 and 5 of mobilisation in the evening.
Maltese: Id-doża ppjanata ta' plerixafor kienet ta' 0.24 mg/kg/jum, mogħtija fil-Jiem 4 u 5 tal-mobilizzazzjoni filgħaxija.
English: o Information on the possibility of false positivity of certain commercial HIV tests because of Zynteglo.
Maltese: o Informazzjoni dwar il-possibbiltà tal-pożittività falza ta' ċerti testijiet tal-HIV kummerċjali minħabba Zynteglo.
English: The median (min, max) time to last RBC transfusion was 0.95 (0.5, 1.1) months following Zynteglo infusion.
Maltese: Iż-żmien medjan (min, mass) għall-aħħar trasfużjoni ta' RBC kien ta' 0.95 (0.5, 1.1) xhur wara l-infużjoni ta' Zynteglo.
English: HSC transplantation with myeloablative conditioning is not appropriate for patients with TDT who have evidence of severely elevated iron in the heart i.e., patients with cardiac T2* <10 msec by magnetic resonance imaging (MRI).
Maltese: Trapjant ta' HSC b'kondizzjonament majeloablattiv mhuwiex adattat għal pazjenti b'TDT li għandhom evidenza ta' żieda ta' ħadid gravi fil-qalb jiġifieri, pazjenti b'T2* <10 msec kardijaċi permezz ta' immaġni b'reżonanza manjetika (MRI).
English: Bag)
Maltese: mass)
English: Petechiae, Ecchymosis, Pain of skin, Palpable purpura, Petechiae, Pruritus generalised, Purpura, Sweat gland disorder, Urticaria, Dry skin
Maltese: Tbenġil, Ekkimożi, Uġigħ filġilda, Purpura palpabbli, Tbenġil, Ħakk ġeneralizzat, Purpura,
English: HGB-205 HGB-204
Maltese: HGB-204 u HGB-205
English: This medicinal product contains 391-1564 mg sodium per dose equivalent to 20 to 78% of the WHO recommended maximum daily intake of 2 g sodium for an adult.
Maltese: Dan il-prodott mediċinali fih 391-1564 mg ta' sodium għal kull doża, ekwivalenti għal 20 sa 78% tat-teħid massimu ta' kuljum rakkomandat mill-WHO ta' 2 g ta' sodium għal persuna adulta.
English: low sodium, low phosphate, decrease in protein, or decrease in albumin
Maltese: sodium baxx, phosphate baxx, tnaqqis fil-proteina, jew tnaqqis fl-albumin
English: HGB-204 and HGB-205 were Phase 1/2 open-label, single-arm 24-month studies that included 22 patients with TDT treated with Zynteglo (N=18, HGB-204; N=4, HGB-205), of whom 14 had a non-β0/β0 genotype (N=10, HGB-204; N=4, HGB-205) and 8 had a β0/β0 genotype in HGB-204.
Maltese: HGB-204 u HGB-205 kienu studji ta' 24 xahar ta' Fażi 1/2 open-label, ta' fergħa waħda li kienu jinkludu 22 pazjent b'TDT ikkurati b'Zynteglo (N=18, HGB-204); N=4, HGB-205), li minnhom 14 kellhom ġenotip mhux β0/β0 (N=10, HGB-204; N=4, HGB-205) u 8 kellhom ġenotip β0/β0 f'HGB-204.
English: Patients are required to undergo HSC mobilisation followed by apheresis to obtain CD34+ stem cells for medicinal product manufacturing (see section 5.1 for a description of the mobilisation regimen used in clinical studies).
Maltese: CD34+ għall-manifattura ta' prodotti mediċinali (ara sezzjoni 5.1 għal deskrizzjoni tal-kors ta' mobilizzazzjoni użat fi studji kliniċi).
English: Thawing of each infusion bag takes approximately 2 to 4 minutes.
Maltese: It-tidwib ta' kull borża tal-infużjoni jieħu madwar 2 sa 4 minuti.
English: HGB-207 and HGB-212 are ongoing Phase 3 open-label, single-arm 24-month studies that are planned to include approximately 39 adults, adolescents, and children with TDT (N=23, HGB-207;
Maltese: HGB-207 u HGB-212 huma studji kontinwi ta' Fażi 3 open-label, ta' fergħa waħda, ta' 24 xahar, li huma ppjanati li jinkludu madwar 39 adult, adolexxenti u tfal b'TDT (N=23, HGB-207);
English: Phenytoin was not used for anti-seizure prophylaxis because of its well understood induction of glutathione-S-transferase and cytochrome P450 and resultant increased clearance of busulfan, and because of the widespread availability of effective anti-seizure medications that do not affect busulfan metabolism.
Maltese: Phenytoin ma ntużax għal profilassi kontra l-aċċessjonijiet minħabba l-induzzjoni magħrufa sew tiegħu ta' glutathione-S-transferase u cytochrome P450 u t-tneħħija miżjuda li tirriżulta ta' busulfan, u minħabba d-disponibbiltà mifruxa sew ta' mediċini effettivi kontra l‑aċċessjonijiet li ma jaffettwawx il-metaboliżmu ta' busulfan.
English: N=16, HGB-212), of whom 29 have a non-β0/β0 genotype (N=23, HGB-207; N=6, HGB-212) and 10 have a β0/β0 genotype in HGB-212.
Maltese: N=16, HGB-212), li minnhom 29 għandhom ġenotip mhux β0/β0 (N=23, HGB-207; N=6, HGB-212)
English: Infusion related reactions to Zynteglo were observed in 11.9% of patients and occurred on the day of Zynteglo infusion.
Maltese: Reazzjonijiet għal Zynteglo relatati mal-infużjoni ġew osservati fi 11.9% tal-pazjenti u seħħew fil-jum tal-infużjoni ta' Zynteglo.
English: (mL/kg/year) median (min, max)
Maltese: Volumi tat-trasfużjoni qabel il-kura (mL/kg/sena) medjan (min, mass)
English: • dizziness, tiredness
Maltese: • uġigħ ieħor • aġitazzjoni
English: All patients who have achieved TI at any time have maintained TI at Month 30 with a min, max duration of TI of 21.2+, 56.3+ months (N=11).
Maltese: Il-pazjenti kollha li kisbu TI fi kwalunkwe ħin żammew it-TI f'Xahar 30 b'tul ta' TI min, mass ta' 21.2+, 56.3+ xhur (N=11).
English: • Confirm that Zynteglo is printed on the infusion bag(s).
Maltese: • Ikkonferma li Zynteglo huwa stampat fuq il-borża (boroż) tal-infużjoni.
English: • soreness of the rectum area
Maltese: • uġigħ fiż-żona tar-rektum
English: • tiredness
Maltese: • rimettar
English: βA-T87Q-globin expression is designed to correct the β/α-globin imbalance in erythroid cells of patients with TDT and has the potential to increase total Hb to normal levels and eliminate dependence on chronic RBC transfusions.
Maltese: L-espressjoni ta' βA-T87Q globin hija mfassla biex tikkoreġi l-iżbilanċ ta' β/α-globin fiċ-ċelluli eritrojdi ta' pazjenti b'TDT u għandha l-potenzjal li żżid l-Hb totali għal-livelli normali u telimina d-dipendenza fuq trasfużjonijiet kroniċi ta' RBC.
English: The planned dose of busulfan was 3.2 mg/kg/day for patients ≥18 years as a 3-hour IV infusion daily for 4 days with a recommended target AUC0-24h of 3800-4500 µM*min.
Maltese: Id-doża ppjanata ta' busulfan kienet ta' 3.2 mg/kg/jum għal pazjenti ≥18-il sena bħala infużjoni IV ta' 3 sigħat kuljum għal 4 ijiem b'AUC0-24siegħa fil-mira rakkomandatata' 3800-4500 µM*min.
English: Depending on the myeloablative conditioning agent administered, prophylaxis for seizures should be considered (see section 5.1 for a description of the prophylaxis regimen used in clinical studies.)
Maltese: Skont l-aġent tal-kondizzjonament majeloablattiv mogħti, għandha tiġi kkunsidrata profilassi għal aċċessjonijiet (ara sezzjoni 5.1 għal deskrizzjoni tal-kors ta' profilassi użat fi studji kliniċi).
English: Time
Maltese: Ara
English: A genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem cells (HSC) transduced with lentiviral vector (LVV) encoding the βA-T87Q-globin gene.
Maltese: Popolazzjoni arrikkita biċ-ċelluli CD34+ awtologi modifikati ġenetikament li fiha ċelluli staminali ematopojetiċi (HSC) trasdotti b'vettur lentivirali (LVV) li jikkodifika l-βA-T87Q-ġene globina.
English: The planned dose of busulfan was 0.8 mg/kg for patients 12-17 years of age as a 2-hour IV infusion every 6 hours for a total of 16 doses with a recommended target of AUC0-6h of 950-1125 µM*min.
Maltese: Id-doża ppjanata ta' busulfan kienet ta' 0.8 mg/kg għal pazjenti ta' bejn 12 u 17-il sena bħala infużjoni IV ta' sagħtejn kull 6 sigħat għal total ta ' 16-il doża b'AUC0-6sigħat fil-mira rakkomandata ta' ta' 950-1125 µM*min.
English: A genetically modified autologous CD34+ cell enriched population that contains haematopoietic stem cells transduced with lentiviral vector encoding the βA-T87Q-globin gene with a strength of 1.2-20 × 106
Maltese: Popolazzjoni ta' ċellula CD34+ awtologa ġenetikament modifikata li fiha ċelluli staminali ematopojetiċi trasdotti b'vettur lentivirali li jikkodifika l-βA-T87Qġene globina b'qawwa ta' 1.2-20 × 106 ċellula/mL.
English: Study HGB-204
Maltese: Studju HGB-204
English: The concentration is 1.2-20 × 106 CD34+ cells (blood stem cells) per millilitre.
Maltese: Il-konċentrazzjoni hija 1.2-20 × 106 ċelluli CD34+ (ċelluli staminali tad-demm) għal kull millilitru.