FrancophonIA/ELRC3.0 huggingface dataset:
English: A negative serology test for HIV is necessary to ensure acceptance of apheresis material for Zynteglo manufacturing.
Danish: Der skal foreligge en negativ serologisk test for hiv for at sikre accept af aferesemateriale til produktion af Zynteglo.
English: Zynteglo must not be administered to women who are breast-feeding.
Danish: Zynteglo må ikke gives til kvinder, der ammer.
English: France bluebird bio (France) SAS Tél: +33 (0)1 85 14 97 89 (0800 914 510)
Danish: France bluebird bio (France) SAS Tlf.: +33 (0) 1 85 14 97 89 (0800 914 510)
English: • Do not sample, alter, or irradiate the medicinal product.
Danish: • Der må ikke udtages prøver af lægemidlet, og det må ikke ændres eller bestråles.
English: • Confirm that Zynteglo is printed on the infusion bag(s).
Danish: • Bekræft, at Zynteglo er påtrykt infusionsposen(-erne).
English: Blood stem cells are collected To make Zynteglo and to serve as replacement cells if needed.
Danish: For at fremstille Zynteglo, og fungere som erstatningsceller efter behov.
English: Patients should be assessed for hepatic impairment to ensure HSC transplantation is appropriate (see section 4.4.).
Danish: Patienter skal vurderes for nedsat leverfunktion for at sikre, at transplantation med hæmatopoietiske stamceller (HSC) er relevant (se pkt.
English: 85521 Ottobrunn
Danish: 85521 Ottobrunn Tyskland
English: The median (min, max) time to last RBC transfusion was 0.95 (0.5, 1.1) months following Zynteglo infusion.
Danish: til sidste RBC-transfusionen var 0,95 (0,5; 1,1) måneder efter infusion med Zynteglo.
English: Each dose contains 391-1564 mg sodium.
Danish: Hjælpestof, som behandleren skal være opmærksom på Hver dosis indeholder 391-1564 mg natrium.
English: Start of Zynteglo treatment
Danish: Start af behandling med Zynteglo
English: Investigations
Danish: Studie
English: HSC transplantation must be appropriate for a patient with TDT to be treated with Zynteglo (see section 4.4).
Danish: Transplantation med hæmatopoietiske stamceller (HSC) skal være relevant for en patient med transfusionsafhængig β-thalassæmi (TDT), der skal behandles med Zynteglo (se pkt.
English: The minimum recommended dose of Zynteglo is 5.0 × 106 CD34+ cells/kg.
Danish: Den anbefalede minimale dosis af Zynteglo er 5,0 × 106 CD34+ celler/kg.
English: See section 2, Sodium content.
Danish: 2, natriumindhold.
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
Danish: For yderligere at bekræfte effekt og sikkerhed af Zynteglo hos patienter på 12 år eller mere med transfusionsafhængig β-thalassæmi (TDT), som ikke har en β0/β0-genotype, skal MAH'en indsende foreløbige og endelige data fra studie HGB-207
English: The minimum target number of CD34+ cells to be collected is 12 × 106 CD34+ cells/kg.
Danish: Antallet af CD34+ celler, der som et minimum skal indsamles, er 12 × 106 CD34+-celler/kg.
English: WA Hb during TI (g/dL)
Danish: Total Hb (g/dl)
English: The Netherlands e-mail: medinfo@bluebirdbio.com
Danish: Holland e-mail: medinfo@bluebirdbio.com
English: Marketing Authorisation Holder and Manufacturer bluebird bio (Netherlands) B.V. Stadsplateau 7
Danish: Indehaver af markedsføringstilladelsen og fremstiller bluebird bio (Holland) B.V.
English: Platelet counts should be monitored according to medical judgment until platelet engraftment and platelet recovery are achieved.
Danish: Trombocyttællinger skal monitoreres efter klinisk skøn, indtil der er opnået engraftment og stabilitet i antallet af trombocytter.
English: Zynteglo in patients 12 years and older with transfusion-dependent β thalassaemia (TDT) who do not have a β0/β0 genotype, the MAH should conduct and submit the results of a study based on data from a product registry (REG501) and use data on patients treated with transfusions and/or HLA-matched allogenic HSCT treated patients from an established European registry as a comparator group.
Danish: Non-interventionelt studie af sikkerhed og effekt efter godkendelse: For yderligere at karakterisere og sætte den langvarige sikkerhed og effekt af Zynteglo hos patienter på 12 år eller mere med transfusionsafhængig β-thalassæmi (TDT), som ikke har en β0/β0-genotype, i sammenhæng, skal MAH'en udføre og indsende resultaterne af et studie baseret på data fra et produktregister (REG-501) og bruge data om patienter behandlet med transfusioner og/eller patienter behandlet med HLA-matchet allogen HSCT fra et etableret europæiske register som komparator.
English: The median (min, max) time to last RBC transfusion was 0.46 (0.2, 5.8) months following Zynteglo infusion.
Danish: til sidste RBC-transfusionen var 0,46 (0,2; 5,8) måneder efter infusion med Zynteglo.
English: Eighteen adults and adolescents with TDT with a non-β0/β0 genotype have been treated with Zynteglo in Phase 3 studies (N=15, HGB-207; N=3, HGB-212) and their median (min, max) duration of follow-up was 10.0 (1.3, 22.2) months.
Danish: Atten voksne og unge med TDT med en non-β0/β0 genotype er blevet behandlet med Zynteglo i fase 3-forsøg (N = 15, HGB-207; N=3, HGB-212), og den gennemsnitlige varighed af deres opfølgning var 10,0 (1,3; 22,2) måneder.
English: You will remain in the hospital for approximately 3-6 weeks
Danish: Efter infusion med Zynteglo Du skal blive på hospitalet i ca. 3-6 uger,
English: Inspect each infusion bag for any breaches of integrity before thawing and infusion.
Danish: Efterse hver infusionspose for eventuel lækage før optøning og infusion.
English: Contraindications to the mobilisation agents and the myeloablative conditioning agent must be considered.
Danish: Kontraindikationer mod midler til mobilisering og midlet til myeloablativ konditionering skal tages i betragtning.
English: Account for all infusion bags and confirm each infusion bag of Zynteglo is within the expiry date using the accompanying Lot Information Sheet.
Danish: Kvittér for alle infusionsposer, og kontrollér ved hjælp af det medfølgende batchinformationsark, at udløbsdatoen på hver infusionspose med Zynteglo ikke er overskredet.
English: In clinical studies doses up to 20 × 106 CD34+ cells/kg have been administered.
Danish: Doser på op til 20 × 106 CD34+ celler/kg er blevet administreret i kliniske studier.
English: Blood and lymphatic system disorders
Danish: Blod og lymfesystem trombocytopeni
English: • a low level of blood platelets, which may reduce the ability of blood to clot
Danish: • et lavt niveau af blodplader, hvilket kan forhindre blodet i at koagulere
English: Lymphopenia, Leukocytosis, Monocyte count decreased, Neutrophilia, Mean cell haemoglobin concentration increased
Danish: lymfopeni, leukocytose, reduceret antal monocytter, neutrofili, forøgelse af hæmoglobinkoncentration
English: o A negative serology test for HIV is necessary to ensure acceptance of apheresis material for Zynteglo manufacturing.
Danish: o Der skal foreligger en negativ serologisk test for hiv for at sikre accept af aferesemateriale til produktion af Zynteglo.
English: • dark patches on skin
Danish: • mørke skjolder på huden
English: Neutropenic sepsis, Systemic infection, Staphylococcal infection, Pneumonia, Lower respiratory tract infection, Urinary tract infection, Mucosal infection, Cellulitis, Vaginal infection, Rash pustular, Folliculitis, Gingivitis
Danish: Infektioner og parasitære sygdomme neutropen sepsis, systemisk infektion, stafylokokinfektion, pneumoni, infektion i de nedre luftveje, urinvejsinfektion, slimhindeinfektion, cellulitis, vaginal infektion, pustuløst udslæt, folliculitis, gingivitis
English: Mobilisation and apheresis
Danish: Mobilisering og aferese
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.
Danish: HGB-204 og HGB-205 var ikke-blindede, enkeltarmede fase 1/2-studier af 24 måneders varighed, som omfattede 22 patienter med TDT, der blev behandlet med Zynteglo (N=18, HGB-204; N=4, HGB-205), hvoraf 14 havde en non-β0/β0 genotype (N=10, HGB-204; N=4, HGB-205) og 8 havde en β0/β0 genotype i HGB-204.
English: • low level of blood platelets, which may reduce the ability of blood to clot
Danish: • lavt niveau af blodplader, hvilket kan forhindre blodet i at koagulere
English: It is not known whether the ingredients of Zynteglo can pass into breast milk.
Danish: Det vides ikke, om indholdsstofferne i Zynteglo kan udskilles i modermælk.
English: (months) n median (min, max)
Danish: (måneder) n median (min, max)
English: After Zynteglo infusion patient iron chelation was managed at physician discretion.
Danish: Efter infusion med Zynteglo, blev jernkelering hos patienten administreret efter lægens skøn.
English: CLINICAL PARTICULARS 4.1 Therapeutic indication
Danish: KLINISKE OPLYSNINGER 4.1.
English: After Zynteglo administration
Danish: Efter administration af Zynteglo
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.
Danish: på 6,44 (1,1; 10,1) g/dl (N=14, HGB-204 og HGB-205), og i løbet af langtidsopfølgningen for LTF-303, blev der påvist stabil integration af βA-T87Q-globingenet i hæmatopoietiske stamceller (HSC'er) med lang levetid og stabil ekspression af βA-T87Q-globingenet i celler af erythroid oprindelse.
English: After Zynteglo infusion, transduced CD34+ HSCs engraft in the bone marrow and differentiate to produce RBCs containing biologically active βA-T87Q-globin (a modified β-globin protein) that will combine with α-globin to produce functional Hb containing βA-T87Q-globin (HbAT87Q).
Danish: Efter infusion med Zynteglo, vil de transducerede hæmatopoietiske CD34+ stamceller accepteres af knoglemarven, de vil differentiere og producere erythrocytter, der indeholder biologisk aktivt β A-T87Q-globin (et modificeret β-globinprotein), som vil kombineres med α-globin, så der produceres funktionelt hæmoglobin (Hb), der indeholder β A-T87Q-globin (HbENT87Q).
English: Citrate toxicity, Contusion, Procedural pain
Danish: Traumer, forgiftninger og behandlings-kom plikationer citrattoksicitet, kontusion, smerter i forbindelse med procedurer
English: You should not take hydroxyurea (a medicine for blood disorders) or any medicines for HIV infection from at least one month before you undergo mobilisation until at least 7 days after Zynteglo infusion (see also section 3, How Zynteglo is given).
Danish: Du må ikke tage hydroxyurea (en medicin mod blodsygdomme) eller medicin mod hiv-infektion fra mindst en måned, før du gennemgår mobilisering og indtil mindst 7 dage efter infusionen med Zynteglo (se også pkt.
English: • Periodic Safety Update Reports (PSURs)
Danish: C. ANDRE FORHOLD OG BETINGELSER FOR MARKEDSFØRINGSTILLADELSEN • Periodiske, opdaterede sikkerhedsindberetninger (PSUR'er)
English: Zynteglo is an autologous gene therapy medicinal product consisting of autologous cells that have been genetically modified ex vivo.
Danish: Det består af autologe celler, som er blevet genetisk modificerede ex vivo.
English: All patients with TDT with a non-β0/β0 genotype who received Zynteglo with at least 3 months of follow-up produced HbAT87Q (N=10, HGB-204; N=4, HGB-205; N=14, HGB-207; N=1, HGB-212).
Danish: Alle patienter med TDT, som har en non-β0/β 0 genotype, og som fik Zynteglo med mindst 3 måneders opfølgning, producerede HbAT87Q (N=10, HGB-204; N=4, HGB-205; N=14, HGB-207; N=1, HGB-212).
English: • Account for all infusion bags and confirm each infusion bag is within the expiry date using the accompanying Lot Information Sheet.
Danish: • Kvittér for alle infusionsposer, og bekræft ved hjælp af det medfølgende lot-informationsark, at der ikke er nogen af infusionsposerne, der har nået udløbsdatoen.
English: • loss of function of your ovaries
Danish: • funktionstab af dine æggestokke
English: Final results: December 2021
Danish: December 2021
English: • feeling sick (nausea)
Danish: • opkastningsfornemmelser (kvalme)
English: • swelling of the face
Danish: • hævelse i ansigtet
English: Patients had a Month 6 median (min, max) HbAT87Q of 4.90 (1.0, 9.6) g/dL in the Phase 1/2 studies (N=14, HGB-204 and HGB-205) and 9.49 (3.4, 10.6) g/dL in the ongoing Phase 3 study (N=11, HGB-207).
Danish: af HbAT87Q hos patienterne på 4,90 (1,0; 9,6) g/dl i fase 1/2-studie (N=14, HGB-204 og HGB-205) og 9,49 (3,4; 10,6) g/dl i de igangværende fase 3-studie (N=11, HGB-207).
English: Studies HGB-207 and HGB-212
Danish: Studie HGB-207 og HGB-212
English: • chest pain or discomfort
Danish: • ubehag i brystet eller smerter
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).
Danish: efter 6 måneder for patienter, som ikke havde modtaget en transfusion i de forudgående 60 dage, var 11,90 (8,4, 13,3) g/dl (N=11).
English: Warnings and precautions of the mobilisation agents and the myeloablative conditioning agent must be considered.
Danish: Advarsler og forsigtighedsregler i forbindelse med midler til mobilisering og midlet til myeloablativ konditionering skal tages i betragtning.
English: ≥100 × 109/L were 52 (20, 268) days and 63 (20, 1231) days, respectively.
Danish: Median (min, max) tid til trombocyttællinger uden støtte på ≥ 50 × 109/l og ≥ 100 × 109/l var hhv. 52 (20; 268) dage og 63 (20; 1231) dage (Se pkt.
English: MARKETING AUTHORISATION HOLDER AND NUMBER
Danish: INDEHAVER AF MARKEDSFØRINGSTILLADELSEN OG MARKEDSFØRINGSTILLADELSESNUMMER
English: Bag ID:
Danish: Pose-ID:
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).
Danish: Effekten var baseret på 32 voksne og unge patienter med TDT og en non-β0/β 0 genotype, der blev behandlet med Zynteglo (N=10, HGB-204; N=4, HGB-205; N=15, HGB-207; N=3, HGB-212) (se tabel 4).
English: Do not sample, alter, or irradiate the medicinal product.
Danish: Der må ikke udtages prøver af lægemidlet, og det må ikke ændres eller bestråles.
English: Interference with serology testing
Danish: Interferens med HIV-testning
English: What Zynteglo looks like and contents of the pack
Danish: Udseende af Zynteglo og pakningsstørrelser
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).
Danish: efter 6 måneder for patienter, som ikke havde modtaget en transfusion i de forudgående 60 dage, var 10,60 (7,6, 13,4) g/dl (N=11).
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).
Danish: Det er påkrævet, at patienterne skal gennemgå mobilisering med hæmatopoietiske stamceller efterfulgt af aferese for at opnå CD34+ stamceller til lægemiddelproduktion (se pkt.
English: All patients were administered Zynteglo with a median (min, max) dose of 7.80 × 106 (5.0, 19.4) CD34+ cells/kg as an intravenous infusion (N=32).
Danish: dosis på 7,80 × 106 (5,0; 19,4) CD34+- celler/kg som en intravenøs infusion (N=32).
English: Zynteglo administration
Danish: Administration af Zynteglo
English: • lip swelling
Danish: • unormal smagssans
English: (g/dL) at 24 months n median (min, max)
Danish: (g/dl) efter 24 måneder n median (min, max)
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.
Danish: Den planlagte busulfan dosis var 3,2 mg/kg/dag hos patienter på ≥18 år, indgivet som en daglig intravenøs infusion af 3 timers varighed i 4 dage med et anbefalet mål-AUC0-24t på 3800-4500 µM*min. For patienter mellem 12-17 år var den planlagte busulfan dosis på 0,8 mg/kg og blev givet som en intravenøs infusion over 2 timer hver 6. time på i alt 16 doser med et anbefalet mål-AUC0-6t på 950-1125 µM*min. Produktresumeet for busulfan blev anvendt som reference for oplysninger om en passende metode til bestemmelse af vægtbaseret dosering til patienten.
English: Studies HGB-204 and HGB-205
Danish: Studie HGB-204 og HGB-205
English: Final results: December 2024
Danish: December 2024
English: Last Name First Name: Date of Birth: DIN:
Danish: Fødselsdato: DIN:
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).
Danish: Patienter må ikke tage anti-retroviral medicin eller hydroxyurea fra mindst en måned før mobilisering og indtil mindst 7 dage efter infusionen med Zynteglo (se pkt.
English: Baseline characteristics for non-β0/β0 patients with TDT ≥12 years of age treated with Zynteglo (Studies HGB-204, HGB-205, HGB-207, HGB-212, LTF-303)
Danish: Tabel 4 Baselinekarakteristika for non-β0/β0-patienter med TDT ≥ 12 år behandlet med Zynteglo (Studie HGB-204, HGB-205, HGB-207, HGB-212, LTF-303)
English: Full myeloablative conditioning must be administered before infusion of Zynteglo (see section 5.1 for a description of the myeloablative regimen used in clinical studies).
Danish: Fuldstændig myeloablativ konditionering skal administreres før infusion af Zynteglo (se pkt.
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).
Danish: af hæmoglobin (Hb) under TI på 12,42 (11,5, 12,6) g/dl (Tabel 5).
English: • itchy skin
Danish: • kløende hud
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).
Danish: Patienter må ikke tage anti-retrovirale lægemidler eller hydroxyurea i mindst en måned før mobilisering og indtil mindst 7 dage efter infusionen med Zynteglo (se pkt.
English: Zynteglo has not been studied in patients with renal impairment.
Danish: Zynteglo er ikke undersøgt hos patienter med nedsat nyrefunktion.
English: Zynteglo 1.2-20 × 106 cells/mL dispersion for infusion (autologous CD34+ cells encoding βA-T87Q-globin gene) For intravenous use.
Danish: Zynteglo 1,2-20 × 106 celler/ml infusionsvæske, dispersion (autologe CD34+ celler, der koder for βA-T87Q-globingenet) Til intravenøs anvendelse.
English: Other medicines and Zynteglo
Danish: Brug af anden medicin sammen med Zynteglo
English: • dry, itchy skin
Danish: • tør, kløende hud
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).
Danish: 48 måneder efter infusion af Zynteglo for patienter, som opnåede TI, var den gennemsnitlige ændring af ferritinniveauerne i serum i forhold til baseline -75,02 % (39.2, 84.8) og ændringen i leverens jernindhold var 67,14 %, strækkende fra en reduktion på 83,3 % til en stigning på 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.
Danish: transfusionsvolumen af erythrocytter på 175,7 (139; 251) ml/kg/år og et gennemsnit (min., maks.)
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).
Danish: varighed af TI på 12,0+, 18,2+ måneder (N=4).
English: Atrial fibrillation
Danish: Hjerte atrieflagren
English: • unusual hair loss or thinning
Danish: • usædvanligt hårtab
English: • agitation
Danish: • træthed
English: Back pain, Musculoskeletal discomfort
Danish: Knoglesmerter rygsmerter, muskuloskeletal stivhed
English: (g/dL) at 6 months n median (min, max)
Danish: (g/dl) efter 6 måneder n median (min, max)
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).
Danish: varighed af TI på 21,2+, 56,3+ måneder (N=11).
English: Zynteglo 1.2-20 × 106 cells/mL dispersion for infusion (autologous CD34+ cells encoding βA-T87Q-globin gene)
Danish: Zynteglo 1,2-20 × 106 celler/ml infusionsvæske, dispersion (autologe CD34+ celler, der koder for βA-T87Q-globingenet)
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).
Danish: Inden behandling med Zynteglo vil du få lægemidler til mobilisering og kemoterapi (se pkt.
English: • pain in larynx (voice box)
Danish: • smerter i strubehovedet (stemmelæberne)
English: Hypocalcaemia, Hypokalaemia, Metabolic acidosis, Fluid overload, Fluid retention, Hypomagnesaemia, Hyponatraemia, Hypophosphataemia
Danish: Almindelig (≥ 1 % - < 10 %) hypokaliæmi, metabolisk acidose, overhydrering væskeretention, hypomagnesiæmi, hyponatriæmi, hypofosfatæmi
English: Hypokalaemia, Hypomagnesaemia
Danish: Metabolisme og ernæring hypocalcæmi hypokaliæmi, hypomagnesiæmi,
English: bluebird bio (Netherlands) B.V. Stadsplateau 7
Danish: bluebird bio (Holland) B.V.
English: Name (Last, First):
Danish: Efternavn: Fornavn:
English: After Zynteglo infusion, avoid use of these iron chelators for 6 months.
Danish: Det skal undgås at anvende disse jernkelatorer i 6 måneder efter infusionen med Zynteglo.
English: Keep infusion bag(s) in the metal cassette(s).
Danish: Opbevar infusionsposer(ne) i metalkassette(r).
English: SPECIFIC OBLIGATION TO COMPLETE POSTAUTHORISATION MEASURES FOR THE CONDITIONAL MARKETING AUTHORISATION
Danish: E. SÆRLIG FORPLIGTELSE TIL AT AFSLUTTE FORANSTALTNINGER EFTER UDSTEDELSE AF MARKEDSFØRINGSTILLADELSE TIL LÆGEMIDLER GODKENDT UNDER BETINGEDE OMSTÆNDIGHEDER
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
Danish: En genetisk modificeret autolog CD34+-beriget cellepopulation, som indeholder hæmatopoietiske stamceller (HSC), som er transduceret med en lentiviral vektor (LVV), der koder for βA-T87Q-globingenet med en styrke på 1,2-20 × 106 celle/ml.
English: Study HGB-207
Danish: Studie HGB-207
English: • soreness of the rectum area
Danish: • ømhed i endetarmsområdet
English: Confirm that Zynteglo is printed on the infusion bag(s).
Danish: Kontrollér, at Zynteglo er påtrykt på infusionsposen(-erne).
English: Headache, Peripheral sensory neuropathy
Danish: Nervesystemet hovedpine, perifer sensorisk neuropati svimmelhed, ondt i hovedet, paræstesi
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).
Danish: Patienter med TDT, som blev behandlet med Zynteglo, og som opnåede engraftment med trombocytter, opnåede en gennemsnitlig engraftment (min., maks.)
English: genotype, the MAH should submit interim and final data from patients with a severe non-β0/β0 genotype such as IVS-I-110 included in Study HGB-212.
Danish: For at bekræfte effekt og sikkerhed af Zynteglo hos patienter på 12 år eller mere med transfusionsafhængig β-thalassæmi (TDT), som ikke har en β0/β0-genotype, skal MAH'en indsende foreløbige og endelige data fra patienter med en svær non-β0/β0 genotype såsom IVS-I-110 inkluderet i studie HGB-212.
English: Total Hb remained stable at Month 24 with a median (min, max) of 10.60 (8.8, 13.7) g/dL (N=12) and at Month 36 with a median (min, max) of 11.30 (7.8, 13.5) g/dL (N=11).
Danish: på 10,60 (8,8; 13,7) g/dl (N=12) og efter 36 måneder med en median (min., maks.)
English: Study HGB-205
Danish: Studie HGB-205