FrancophonIA/ELRC3.0 huggingface dataset:
English: Blood stem cells are collected To make Zynteglo and to serve as replacement cells if needed.
Slovenian: Za izdelavo zdravila Zynteglo in uporabo za nadomestne celice, če bodo potrebne
English: - The active substance of Zynteglo consists of your own blood stem cells that contain functional copies of the beta-globin gene that can be measured in your blood.
Slovenian: - Učinkovina zdravila Zynteglo je sestavljena iz vaših krvotvornih matičnih celic, ki vsebujejo funkcionalne kopije gena za beta globin, ki ga je mogoče v vaši krvi izmeriti.
English: Before infusion, it must be confirmed that the patient's identity matches the unique patient information on the Zynteglo infusion bag(s).
Slovenian: Pred infuzijo je treba potrditi, da se identiteta bolnika ujema z edinstvenimi informacijami o bolniku na infuzijski(h) vrečki(vrečkah).
English: Zynteglo is an autologous gene therapy medicinal product consisting of autologous cells that have been genetically modified ex vivo.
Slovenian: Zdravilo Zynteglo je zdravilo za avtologno gensko terapijo, sestavljeno iz avtolognih celic, ki so bile gensko spremenjene ex vivo.
English: The treating physician should confirm that HSC transplantation is appropriate for the patient before myeloablative conditioning is initiated (see section 4.4).
Slovenian: Lečeči zdravnik mora potrditi, da je presaditev HSC primerna za bolnika, preden se začne mieloablativni postopek (glejte poglavje 4.4).
English: It is not known whether the ingredients of Zynteglo can pass into breast milk.
Slovenian: Ni znano, ali lahko sestavine zdravila Zynteglo prehajajo v materino mleko.
English: No data from clinical studies are available regarding overdose of Zynteglo.
Slovenian: Podatki iz kliničnih študij glede prevelikega odmerjanja zdravila Zynteglo niso na voljo.
English: Five patients are evaluable for assessment of TI. Of these, 4/5 (80.0%, 95% CI 28.4-99.5%) achieved TI at last follow-up.
Slovenian: Štirje od petih (80,0 %, 95-odstotni interval zaupanja 28,4-99,5 %) so dosegli TI pri zadnjem spremljanju.
English: Patients not receiving prophylaxis for VOD appeared to be at an increased risk for developing VOD.
Slovenian: Kaže, da je pri bolnikih, ki niso prejeli profilakse za jetrno vensko-okluzivno bolezen, večje tveganje za razvoj jetrne vensko-okluzivne bolezni.
English: In the 3 patients who did not achieve TI, reductions of 100%, 86.9% and 26.8% in transfusion volume requirements and of 100%, 85.3% and 20.7% in transfusion frequency were observed between Month 6 through Month 24 visit when compared to their pre-study levels of RBC transfusions.
Slovenian: Pri 3 bolnikih, ki niso dosegli TI, so na pregledih od 6. do 24. meseca opazili zmanjšanje potrebne količine transfuzije za 100 %, 86,9 % in 26,8 % ter pogostnosti transfuzij za 100 %, 85,3 % in 20,7 % v primerjavi z njihovimi transfuzijami RBC pred študijo.
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.
Slovenian: Biopsije kostnega mozga, odvzete pred zdravljenjem, so bile skladne z diagnozo TDT, vključno z nizkim razmerjem mieloid/eritroid (N=15, HGB-207), kar kaže na eritroidno hiperplazijo.
English: HSC transplantation must be appropriate for a patient with TDT to be treated with Zynteglo (see section 4.4).
Slovenian: Presaditev celic HSC mora biti primerna za bolnika s TDT, ki bo zdravljen z zdravilom Zynteglo (glejte poglavje 4.4).
English: Other medicines and Zynteglo
Slovenian: Druga zdravila in zdravilo Zynteglo
English: Most side effects occur during or within a few days after treatment with Zynteglo but can occur later.
Slovenian: Večina neželenih učinkov se pojavi v nekaj dneh po zdravljenju z zdravilom Zynteglo, vendar se lahko pojavijo tudi pozneje.
English: MRI of the liver should be performed on all patients prior to myeloablative conditioning.
Slovenian: Magnetno resonančno slikanje (MRI) jeter je treba izvesti pri vseh bolnikih, ki so pred mieloablativnim postopkom.
English: (Second Infusion
Slovenian: (Druga infuzijska vrečka)
English: • bleeding or bruising
Slovenian: • krvavitve ali modrice
English: • The Guide for healthcare professionals shall contain the following key elements: o Warnings and precautions of the mobilisation agents and the myeloablative conditioning agent must be considered.
Slovenian: • Navodilo za zdravstvene delavce mora vsebovati naslednje ključne elemente: o Upoštevati je treba opozorila in previdnostne ukrepe za mobilizacijske učinkovine in učinkovino za mieloablativni postopek.
English: Mobilisation and apheresis
Slovenian: Mobilizacija in afereza
English: • pain in back, bone, skin, limbs, anus, or muscles
Slovenian: • bolečine v hrbtu, kosteh, na koži, v okončinah, anusu ali mišicah
English: Manufacturer apceth Biopharma GmbH Haidgraben 5
Slovenian: Izdelovalec apceth Biopharma GmbH Haidgraben 5
English: Zynteglo adds functional copies of a modified β-globin gene into the patients' HSCs through transduction of autologous CD34+ cells with BB305 LVV, thereby addressing the underlying genetic cause of the disease.
Slovenian: Zdravilo Zynteglo doda funkcionalne kopije spremenjenega gena za β-globin v bolnikove celice HSC s transdukcijo avtolognih celic CD34+ z BB305 LVV, s čimer se osredotoči na genski vzrok bolezni.
English: • dark patches on skin
Slovenian: • temne lise na koži
English: A back-up collection of CD34+ stem cells of at least 1.5 × 106 CD34+ cells/kg (if collected by apheresis) or >1.0 × 108 TNC/kg (if collected by bone marrow harvest) is required.
Slovenian: Potrebno je dodatno zbiranje matičnih celic CD34+, in sicer vsaj 1,5 × 106 celic CD34+ /kg (če jih zbiramo z aferezo) ali > 1,0 × 108 TNC/kg (če jih zbiramo z jemanjem kostnega mozga).
English: This being a conditional marketing authorisation and pursuant to Article 14(7) of Regulation (EC) No 726/2004, the MAH shall complete, within the stated timeframe, the following measures:
Slovenian: 726/2004, mora imetnik dovoljenja za promet z zdravilom v določenem časovnem okviru izvesti naslednje ukrepe:
English: • unusual hair loss or thinning
Slovenian: • neobičajno izpadanje ali tanjšanje las
English: Prior to launch of Zynteglo in each Member State the Marketing Authorisation Holder (MAH) must agree about the content and format of the educational and controlled distribution programme,
Slovenian: Pred prihodom zdravila Zynteglo na trg v vsaki državi članici se mora imetnik dovoljenja za promet z zdravilom s pristojnim nacionalnim organom dogovoriti o obliki in vsebini izobraževalnega programa in programa nadzorovane distribucije, vključno s sredstvi obveščanja, načini razdeljevanja in drugimi vidiki programa.
English: What Zynteglo looks like and contents of the pack
Slovenian: Izgled zdravila Zynteglo in vsebina pakiranja
English: Patients who experience neutrophil engraftment failure should receive rescue treatment with the back-up collection (see section 4.2).
Slovenian: Bolniki z neuspešno vsaditvijo nevtrofilcev morajo prejeti rešilno zdravljenje z dodatno zbranimi matičnimi celicami (glejte poglavje 4.2).
English: To prepare your bone marrow for treatment with Zynteglo.
Slovenian: Za pripravo kostnega mozga na zdravljenje z zdravilom Zynteglo
English: Expiry Date
Slovenian: Do datuma
English: The concentration is 1.2-20 × 106 CD34+ cells (blood stem cells) per millilitre.
Slovenian: Koncentracija znaša 1,2-20 × 106 celic CD34+ (krvotvornih matičnih celic) na mililiter.
English: • Do not sample, alter, or irradiate the medicinal product.
Slovenian: • Zdravila ne smete vzorčiti, spremeniti ali obsevati.
English: Patients should be assessed for hepatic impairment to ensure HSC transplantation is appropriate (see section 4.4.).
Slovenian: Bolnike je treba oceniti za okvaro jeter, da se potrdi primernost presaditve HSC (glejte poglavje 4.4).
English: Your name and date of birth, as well as coded information identifying you as the patient, are printed onto each infusion bag and each metal container.
Slovenian: Na vsako infuzijsko vrečko in vsak kovinski vsebnik so natisnjeni vaše ime in datum rojstva ter kodirane informacije, ki vas identificirajo kot bolnika.
English: Anti-retroviral and hydroxyurea use
Slovenian: Uporaba protiretrovirusnih zdravil in hidroksisečnine
English: • Administer each infusion bag of Zynteglo via intravenous infusion over a period of less than 30 minutes.
Slovenian: • Vsako infuzijsko vrečko zdravila Zynteglo je treba dati kot intravensko infuzijo, ki naj traja manj kot 30 minut.
English: Standard procedures for patient management after HSC transplantation should be followed after Zynteglo infusion.
Slovenian: Po infundiranju zdravila Zynteglo je treba upoštevati standardne postopke za obravnavo bolnikov po presaditvi HSC.
English: Transfusion-dependent β-thalassaemia (TDT)
Slovenian: Beta talasemija, ki zahteva transfuzijo (TDT)
English: The median (min, max) busulfan dose was 3.50 (2.5, 5.0) mg/kg/day (N=32).
Slovenian: odmerka busulfana je bila 3,50 (2,5; 5,0) mg/kg/dan (N=32).
English: In order to confirm the efficacy and safety of Zynteglo in patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0
Slovenian: Za potrditev učinkovitosti in varnosti zdravila Zynteglo pri bolnikih, starih 12 let in več, z β-talasemijo, odvisno od transfuzije (TDT), ki nimajo genotipa β0/β0,
English: Protocol submission:
Slovenian: Predložitev protokola: Julij
English: • Infuse Zynteglo as soon as possible and no more than 4 hours after thawing.
Slovenian: • Infundirajte zdravilo Zynteglo čim prej oziroma ne kasneje kot v 4 urah po odtajanju.
English: • a low level of blood platelets, which may reduce the ability of blood to clot
Slovenian: • nizka raven trombocitov v krvi, ki lahko zmanjša sposobnost strjevanja krvi
English: • This medicinal product contains genetically modified human blood cells.
Slovenian: • To zdravilo vsebuje gensko spremenjene človeške krvne celice.
English: Apheresis material from patients with a positive test for HIV will not be accepted for Zynteglo manufacturing.
Slovenian: Aferezni material bolnikov s pozitivnim testom za HIV ni sprejemljiv za izdelavo zdravila
English: Autologous CD34+ cells encoding βA-T87Q-globin gene This medicine is subject to additional monitoring.
Slovenian: Avtologne celice CD34+, ki kodirajo gen za βA-T87Q-globin Za to zdravilo se izvaja dodatno spremljanje varnosti.
English: In order to confirm the efficacy and safety of Zynteglo in patients 12 years and older with transfusion-dependent β-thalassaemia (TDT) who do not have a β0/β0 genotype, the MAH should submit interim and final data on Study HGB-207
Slovenian: Za potrditev učinkovitosti in varnosti zdravila Zynteglo pri bolnikih, starih 12 let in več, z β-talasemijo, odvisno od transfuzije (TDT), ki nimajo genotipa β0/β0, mora imetnik dovoljenja za promet predložiti vmesne in končne podatke za študijo HGB-207
English: Account for all infusion bags and confirm each infusion bag of Zynteglo is within the expiry date using the accompanying Lot Information Sheet.
Slovenian: Preštejte vse infuzijske vrečke in potrdite, da so vse infuzijske vrečke zdravila Zynteglo znotraj datuma izteka roka uporabnosti, ki je naveden v priloženem dokumentu za informacije o seriji.
English: 3521AZ Utrecht The Netherlands medinfo@bluebirdbio.com
Slovenian: 3521AZ Utrecht Nizozemska medinfo@bluebirdbio.com
English: • enlarged liver
Slovenian: • povečana jetra
English: Infuse as soon as possible and no more than 4 hours after thawing.
Slovenian: Infundirajte ga čim prej oziroma najkasneje v 4 urah po odtajanju.
English: At 48 months after infusion of Zynteglo for patients who achieved TI, the median reduction (min, max) in serum ferritin levels from baseline was 75.02% (39.2, 84.8) (N=3, HGB-204; N=2, HGB-205).
Slovenian: 48 mesecev po infundiranju zdravila Zynteglo bolnikom, ki so dosegli TI, je bila mediana zmanjšanja (min, maks) ravni feritina v serumu od izhodišča 75,02 % (39,2; 84,8) (N=3, HGB-204; N=2, HGB-205).
English: o Package leaflet o A patient/carer guide o A patient alert card
Slovenian: o Navodilo za uporabo o Vodnik za bolnika/skrbnika o Opozorilno kartico za bolnika
English: • small tear in tissue that lines the anus
Slovenian: • manjša raztrganina tkiva okrog anusa
English: The minimum recommended dose of Zynteglo is 5.0 × 106 CD34+ cells/kg.
Slovenian: Najmanjši priporočeni odmerek zdravila Zynteglo je 5,0 × 106 celic CD34+ /kg.
English: • After thaw, mix the medicinal product gently by massaging the infusion bag until all of the contents are uniform.
Slovenian: • Po odtajanju rahlo premešajte zdravilo tako, da infuzijsko vrečko stiskate, dokler vsebina ni enakomerno porazdeljena.
English: low sodium, low phosphate, decrease in protein, or decrease in albumin
Slovenian: nizko raven magnezija, nizko raven natrija, nizko raven fosfata, znižanje ravni beljakovin ali znižanje ravni albumina
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).
Slovenian: Za pridobitev matičnih celic CD34+ za izdelavo zdravila morajo bolniki opraviti mobilizacijo HSC, ki ji sledi afereza (glejte poglavje 5.1 za opis režima mobilizacije, ki je bil uporabljen v kliničnih študijah).
English: • The Guide to handling and method of administration for healthcare professionals shall contain the following key elements: o Instructions on receiving and storing of Zynteglo and how to check Zynteglo prior to administration o Instructions about the thawing of Zynteglo o Instructions on protective equipment and treatment of spills.
Slovenian: o Navodila o sprejemu in shranjevanju zdravila Zynteglo in kako je treba zdravilo Zynteglo pred dajanjem pregledati o Navodila o odtajanju zdravila Zynteglo o Navodila o zaščitni opremi in obravnavi razlitja.
English: The Netherlands e-mail: medinfo@bluebirdbio.com
Slovenian: Nizozemska e-pošta: medinfo@bluebirdbio.com
English: All patients with non-β0/β0 genotypes received anti-seizure prophylaxis with agents other than phenytoin prior to initiating busulfan.
Slovenian: Vsi bolniki z genotipi, ki ni genotip β0/β0, so bili pred začetkom odmerjanja busulfana deležni protiepileptične profilakse z učinkovinami, ki niso bile fenitoin.
English: Neutrophil engraftment occurred on median (min, max) Day 19.5 (13, 38) after medicinal product infusion.
Slovenian: prišlo po 19,5 (13, 38) dneh po infundiranju zdravila.
English: Pre-treatment conditioning
Slovenian: Postopek pred zdravljenjem
English: After thaw, mix the medicinal product gently by massaging the infusion bag until all of the contents are uniform.
Slovenian: Po odtajanju rahlo premešajte zdravilo tako, da infuzijsko vrečko stiskate, dokler vsebina ni enakomerno porazdeljena.
English: The Lot Information Sheet is included inside the lid of the cryoshipper used to transport Zynteglo.
Slovenian: Dokument z informacijami o seriji se nahaja v pokrovu kriogenskega transportnega tanka, ki se uporablja za transport zdravila Zynteglo.
English: The median (min, max) time to last RBC transfusion was 0.95 (0.5, 1.1) months following Zynteglo infusion.
Slovenian: do zadnje transfuzije RBC je bila 0,95 (0,5; 1,1) mesecev po infuziji zdravila Zynteglo.
English: Women of childbearing potential/Contraception in males and females There are insufficient exposure data to provide a precise recommendation on duration of contraception following treatment with Zynteglo.
Slovenian: Ni dovolj podatkov o izpostavljenosti, da bi zagotovili natančno priporočilo glede trajanja kontracepcije po zdravljenju z zdravilom Zynteglo.
English: Hypokalaemia, Hypomagnesaemia
Slovenian: hiponatriemija, hipofosfatemija
English: Medicinal product subject to special and restricted medical prescription (see Annex I: Summary of Product Characteristics, section 4.2).
Slovenian: POGOJI ALI OMEJITVE GLEDE OSKRBE IN UPORABE Predpisovanje in izdaja zdravila je le na poseben recept in le s posebnim režimom (glejte Prilogo I: Povzetek glavnih značilnosti zdravila, poglavje 4.2).
English: The number
Slovenian: Številka serije/
English: The median (min, max) duration of follow-up was 40.48 (29.3, 58.6) months.
Slovenian: trajanje spremljanja je bilo 40,48 (29,3; 58,6) mesecev.
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).
Slovenian: skupnega Hb v 6. mesecu pri bolnikih, ki niso prejeli transfuzije v predhodnih 60 dneh, je znašala 11,90 (8,4; 13,3) g/dl (N=11).
English: (mL/kg/year) median (min, max)
Slovenian: (ml/kg/leto) mediana (min, maks)
English: • dizziness, tiredness
Slovenian: • omotica, utrujenost
English: The median (min, max) total Hb at Month 6 for patients who had not received a transfusion for the prior 60 days was 10.60 (7.6, 13.4) g/dL (N=11).
Slovenian: skupnega Hb v 6. mesecu pri bolnikih, ki niso prejeli transfuzije v predhodnih 60 dneh, je znašala 10,60 (7,6; 13,4) g/dL (N=11).
English: In clinical studies doses up to 20 × 106 CD34+ cells/kg have been administered.
Slovenian: V kliničnih študijah so uporabili odmerke do 20 × 106 CD34+ celic/kg.
English: Patients should not take anti-retroviral medicinal products or hydroxyurea from at least one month prior to mobilisation until at least 7 days after Zynteglo infusion (see section 4.4).
Slovenian: Bolniki ne smejo jemati protiretrovirusnih zdravil ali hidroksisečnine vsaj en mesec pred mobilizacijo ter do vsaj 7 dni po infuziji zdravila Zynteglo (glejte poglavje 4.4).
English: The median (min, max) time to last RBC transfusion was 0.46 (0.2, 5.8) months following Zynteglo infusion.
Slovenian: do zadnje transfuzije RBC je bila 0,46 (0,2; 5,8) mesecev po infuziji zdravila Zynteglo.
English: Zynteglo 1.2-20 × 106 cells/mL dispersion for infusion.
Slovenian: Zynteglo 1,2-20 × 106 celic/ml disperzija za infundiranje.
English: Insertion site analysis (ISA) of pre-transplantation transduced mouse BMCs and human CD34+ HSCs showed no enrichment for insertion in or near cancer-related genes.
Slovenian: Analiza mesta vstavitve (ISA) transduciranega CKM miši in humanih celic CD34+ HSC pred transplantacijo ni pokazala obogatitve za vstavljanje v gene,
English: Pre-medication for infusion reactions was managed at physician discretion.
Slovenian: Reakcije, povezane z infundiranjem zdravila Zynteglo Premediciranje za reakcije, povezane z infundiranjem, so obvladovali glede na presojo zdravnika.
English: Patients should not take anti-retroviral medications or hydroxyurea from at least one month prior to mobilisation until at least 7 days after Zynteglo infusion (see section 4.5).
Slovenian: Bolniki ne smejo jemati protiretrovirusnih zdravil ali hidroksisečnine vsaj en mesec pred mobilizacijo ter do vsaj 7 dni po infuziji zdravila Zynteglo (glejte poglavje 4.5).
English: Platelet counts should be monitored according to medical judgment until platelet engraftment and platelet recovery are achieved.
Slovenian: Po medicinski presoji je treba spremljati število trombocitov, dokler nista doseženi vsaditev in obnova trombocitov.
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.
Slovenian: 6,44 (1,1; 10,1) g/dL (N=14, HGB-204 in HGB-205) in v času dolgoročnega spremljanja v LTF-303, pri čemer se je kazala stabilna integracija gena za βA-T87Q-globin v dolgoročnih celicah HSC in stabilno izražanje gena za βA-T87Q-globin v celicah eritroidne linije.
English: The effect of administration of Zynteglo to mothers on their breast-fed children has not been studied.
Slovenian: Učinek na dojene otroke pri dajanju zdravila Zynteglo doječim materam ni bil preučen.
English: Investigations
Slovenian: Študija
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.)
Slovenian: Glede na učinkovino, uporabljeno za mieloablativni postopek, je treba razmisliti o profilaksi epileptičnih napadov (glejte poglavje 5.1 za opis režima profilakse, ki je bil uporabljen v kliničnih študijah).
English: Start of Zynteglo treatment
Slovenian: Začetek zdravljenja z zdravilom Zynteglo
English: • Physician educational material • Patient information pack
Slovenian: • izobraževalno gradivo za zdravnika
English: • bone or muscle pain
Slovenian: • bolečine v kosteh ali mišicah
English: Store in the vapour phase of liquid nitrogen at ≤-140°C until ready for thaw and administration.
Slovenian: Shranjujte v parni fazi tekočega dušika pri ≤ -140 °C do priprave za odtajanje in odmerjanje.
English: • feeling sick (nausea), being sick (vomiting)
Slovenian: • občutek slabosti (navzea), slabost (bruhanje)
English: Table 4
Slovenian: 4. četrtletje
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).
Slovenian: obtežena povprečna vrednost Hb med TI znašala 12,42 (11,5; 12,6) g/dl (tabela 5).
English: o The need to explain and to ensure that patients understand: - potential risks of treatment with Zynteglo - signs of leukaemia/lymphoma and what action to take - content of patient's guide - the need to carry the patient alert card and show it to every healthcare professional - enrolment in the drug product Registry o Scope of the Registry and how to enrol patients
Slovenian: - znake levkemije/limfoma in kako naj ukrepa - vsebino vodnika za bolnika - potrebo, da ima opozorilno kartico za bolnika vedno s seboj in jo pokaže vsem zdravstvenim delavcem
English: Dispersion for infusion
Slovenian: Disperzija za infundiranje
English: • have abnormal bruising or bleeding (such as bruising without injury, blood in your urine, stool, vomit, or cough up blood).
Slovenian: • imate nenormalne modrice ali krvavitve (kot so modrice, ne da bi se poškodovali, kri v urinu,
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.
Slovenian: Načrtovani odmerek busulfana je bil 3,2 mg/kg/dan za bolnike ≥ 18 let v obliki 3-urne intravenske infuzije dnevno v trajanju 4 dni s priporočeno ciljno AUC0-24 h v vrednosti 3800-4500 µM*min. Načrtovani odmerek busulfana je bil 0,8 mg/kg za bolnike, stare 12-17 let, v obliki 2-urne intravenske infuzije vsakih 6 ur do skupno 16 odmerkov s priporočeno ciljno AUC0-6 h 950-1125 µM*min. Za informacije o ustrezni metodi za določitev odmerka na podlagi telesne mase bolnika je bil uporabljen SmPC za busulfan.
English: • stomach pain, upset stomach, constipation, diarrhoea
Slovenian: • bolečine v trebuhu, razdražen želodec, zaprtost, driska
English: Zynteglo 1.2-20 × 106 cells/mL dispersion for infusion (autologous CD34+ cells encoding βA-T87Q-globin gene)
Slovenian: Zynteglo 1,2-20 × 106 celic/ml disperzija za infundiranje (avtologne celice CD34+, ki kodirajo gen za βA-T87Q-globin)
English: • skin abrasion/scrape
Slovenian: • kožne odrgnine/praske
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.
Slovenian: HGB-204 in HGB-205 sta bila nezaslepljeni 24-mesečni študiji faze I/II z eno skupino bolnikov, ki sta vključevali 22 bolnikov s TDT, ki so se zdravili z zdravilom Zynteglo (N=18, HGB-204; N=4, HGB-205), od katerih je bilo 14 bolnikov z genotipom, ki ni genotip β0/β0 (N=10, HGB-204; N=4, HGB-205), in 8 bolnikov z genotipom β0/β0 v študiji HGB-204.