FrancophonIA/ELRC3.0 huggingface dataset:
English: The immunogenic response to nusinersen was determined in 346 patients with baseline and post-baseline plasma samples evaluated for anti-drug antibodies (ADA).
Danish: Det immunogene respons på nusinersen blev bestemt hos 346 patienter med plasmaprøver ved baseline og efter baseline, der blev evalueret for antistoffer mod lægemidlet (anti-drug antibodies, ADA).
English: Spinraza contains a small amount of sodium
Danish: Spinraza indeholder en lille mængde natrium
English: Presence of nusinersen was also demonstrated in neurons and other cell types in the spinal cord and brain, and peripheral tissues such as skeletal muscle, liver, and kidney.
Danish: Det blev også vist, at nusinersen var til stede i neuroner og andre celletyper i rygmarven og hjernen, og i det perifere væv, såsom skeletmuskulaturen, leveren og nyrerne.
English: Ultrasound (or other imaging techniques) may be considered to guide intrathecal administration of Spinraza, particularly in younger patients and in patients with scoliosis.
Danish: Ultralyd (eller andre teknikker til billeddannelse) kan overvejes til at guide en intratekal administration af Spinraza især hos yngre patienter og hos patienter med skoliose.
English: Among patients older than the WHO defined window for expected age of achievement (95th percentile), 17 of 22 (77%) had achieved walking alone.
Danish: Blandt de patienter, der var ældre end det WHO-definerede vindue for forventet alder for præstation (den 95. percentil), kunne 17 ud af 22 (77 %) gå alene.
English: The proportion of patients developing clinically manifested SMA was assessed amongst patients who reached the Day 700 visit at the interim analysis (n=16).
Danish: Andelen af patienter, der udviklede klinisk manifestation af SMA, blev vurderet blandt patienter, der var nået til besøget ved dag 700 ved interimanalysen (n=16).
English: Reproductive toxicology studies were conducted using subcutaneous administration of nusinersen in mice and rabbits.
Danish: Der er udført toksikologistudier af reproduktion med subkutan administration af nusinersen hos mus og kaniner.
English: At the interim analysis, all 25 (100%) patients had achieved the WHO motor milestone of sitting without support, 22 (88%) patients were walking with assistance.
Danish: Ved interimanalysen havde alle 25 (100 %) patienter opnået den motoriske WHO-milepæl at sidde uden støtte, og 22 (88 %) af patienterne kunne gå med hjælp.
English: All patients had the ability to suck and swallow at last assessment, with 22 (88%) infants achieving a maximal score on the HINE Section 1.
Danish: Alle patienter kunne sutte og synke ved den sidste vurdering, og 22 (88 %) af spædbørnene opnåede en maksimal score på HINE afsnit 1.
English: Spinraza treated Patients
Danish: Spinraza-behandlede patienter
English: 5 ml in a Type I glass vial with bromobutyl rubber stopper and an aluminium over-seal and plastic cap.
Danish: 5 ml i et type I glas-hætteglas med en brombutyl-gummiprop og en overforsegling af aluminium og et plasticlåg.
English: • Hypersensitivity (an allergic or allergic-like reaction that may include swelling of your face, lips or tongue, rash, or itching)
Danish: • Overfølsomhed (en allergisk eller allergilignende reaktion, som kan omfatte hævelse af ansigt, læber eller tunge, udslæt eller kløe)
English: Spinraza works by helping the body to produce more of the SMN protein that people with SMA are lacking.
Danish: Spinraza virker ved at hjælpe kroppen med at danne mere SMN-protein, som personer med SMA mangler.
English: Twelve out of 20 patients (60%) in the study met the primary endpoint with improvement in mean motor milestone achievement over time.
Danish: 12 ud af 20 patienter (60 %) i studiet opfyldte det primære endepunkt med en forbedring i gennemsnitlige resultater for motoriske milepæle over tid.
English: An interim analysis was conducted when patients had been on study for median of 27.1 months (15.135.5 months) and were of a median age at last visit of 26.0 months (14.0 -34.3 months).
Danish: Der blev gennemført en interimanalyse, da patienterne havde deltaget i studiet i en medianperiode på 27,1 måneder (15,1-35,5 måneder) og havde en medianalder ved det sidste besøg på 26,0 måneder (14,0-34,3 måneder).
English: Of the 20 subjects enrolled, 11 (55%) were alive and free of permanent ventilation at the last visit.
Danish: Af de 20 inkluderede forsøgspersoner var 11 (55 %) i live og fri for permanent ventilering ved sidste besøg.
English: Intrathecal injection of nusinersen into the CSF allows nusinersen to be fully available for distribution from the CSF to the target central nervous system (CNS) tissues.
Danish: Intratekal injektion af nusinersen i CSF gør nusinersen fuldkommen tilgængelig for fordeling fra CSF til målvævet i centralnervesystemet (CNS).
English: This results in the loss of nerve cells in the spine, leading to weakness of the muscles in the shoulders, hips, thighs and upper back.
Danish: Dette resulterer i et tab af nerveceller i rygmarven og fører til muskelsvaghed i skuldre, hofter, lår og den øvre del af ryggen.
English: MedDRA preferred term Spinraza frequency category, n=84
Danish: Spinraza-hyppighedskategori, n = 84
English: Number of patients who died Hazard Ratio (95% CI) p-value6
Danish: Antal patienter, der døde Hazard Ratio (95 % CI) p-værdi6
English: Time to death or permanent ventilation (≥ 16 hours ventilation/day continuously for > 21 days in the absence of an acute reversible event or tracheostomy) was assessed as the primary endpoint.
Danish: Tidsperioden til død eller permanent ventilering (≥ 16 timers ventilering/dag kontinuerligt i > 21 dage ved fraværet af akutte reversible hændelser eller trakeostomi) blev vurderet som det primære endepunkt.
English: No impact on male or female fertility, or embryo-foetal development, or pre/post-natal development was observed.
Danish: Der blev ikke observeret nogen påvirkning af fertiliteten hos hanner eller hunner, embryoføtal udvikling eller præ/postnatal udvikling.
English: • Serious infection related to lumbar puncture (e.g. meningitis)
Danish: • Alvorlig infektion i forbindelse med lumbalpunktur (f.eks. meningitis)
English: April 2023.
Danish: studieresultater: April 2023
English: Some patients were implanted with a ventriculo-peritoneal shunt.
Danish: Nogle patienter fik implanteret en ventrikulo-peritoneal shunt.
English: • Meningitis not caused by an infection (inflammation of the membrane around the spinal cord and brain, which may present as neck stiffness, headache, fever, nausea and vomiting)
Danish: • Meningitis, der ikke er forårsaget af en infektion (inflammation af membranen omkring rygmarven og hjernen, der kan vise sig som nakkestivhed, hovedpine, feber, kvalme og opkastning).
English: The majority of patients (88%) have 3 copies of the SMN2 gene (8% have 2 copies, 2% have 4 copies, and 2% have an unknown copy number).
Danish: Hovedparten af patienterne (88 %) har 3 kopier af SMN2genet (8 % har 2 kopier, 2 % har 4 kopier og 2 % har et ukendt antal kopier).
English: This can include headaches, vomiting and back pain.
Danish: Dette kan omfatte hovedpine, opkastning og rygsmerter.
English: Each carton of Spinraza contains one vial.
Danish: Hver yderkarton med Spinraza indeholder ét hætteglas.
English: In repeat-dose toxicity studies (14-weeks and 53-weeks) of intrathecal administration to juvenile cynomolgus monkeys, nusinersen was well tolerated.
Danish: I toksicitetsstudier med gentagne doser (efter 14 uger og 53 uger) med intratekal administration til unge cynomolgus-aber var nusinersen veltolereret.
English: Number of patients who died or received permanent ventilation Hazard ratio (95% CI) p-value6
Danish: Antal patienter, der døde eller fik permanent ventilering Hazard ratio (95 % CI) p-værdi6
English: At the time of a Study CS11 interim analysis, 61 out of 65 patients (94%) were alive.
Danish: På tidspunktet for en interimanalyse i studie CS11 var 61 ud af 65 patienter (94 %) i live.
English: Reporting of side effects If you or your child get any side effects, talk to your doctor or nurse.
Danish: Indberetning af bivirkninger Hvis du eller dit barn oplever bivirkninger, bør du tale med din læge eller sygeplejerske.
English: Median age at first dose was 22 days.
Danish: Medianalderen ved den første dosis var 22 dage.
English: The benefits and risks of continuing Spinraza whilst having a "ventriculo-peritoneal shunt" in place are not known at present.
Danish: Fordelene og risiciene ved at fortsætte behandling med Spinraza, mens der er implanteret en 'ventrikulo-peritoneal shunt', kendes ikke på nuværende tidspunkt.
English: Mean CSF trough concentrations of nusinersen accumulated approximately 1.4- to 3-fold after multiple loading and maintenance doses, and reached a steady state within approximately 24 months.
Danish: Gennemsnitlige minimale CSF-koncentrationer af nusinersen akkumulerede ca. 1,4 til 3 gange efter flere stød- og vedligeholdelsesdoser og nåede et steady-state i løbet af ca. 24 måneder.
English: There have been reports of communicating hydrocephalus not related to meningitis or bleeding in patients treated with nusinersen in the post-marketing setting.
Danish: Der har været rapporter om kommunikerende hydrocephalus, der ikke var forbundet med meningitis eller blødning, hos patienter behandlet med nusinersen efter markedsføring.
English: 1 out of 28 patients had 2 SMN2 gene copies, 21 had 3 copies, and 6 had 4 copies.
Danish: 1 ud af 28 patienter havde 2 SMN2-genkopier, 21 havde 3 kopier og 6 havde 4 kopier.
English: Nusinersen is an antisense oligonucleotide (ASO) which increases the proportion of exon 7 inclusion in survival motor neuron 2 (SMN2) messenger ribonucleic acid (mRNA) transcripts by binding to an intronic splice silencing site (ISS-N1) found in intron 7 of the SMN2 pre-messenger ribonucleic acid (pre-mRNA).
Danish: Nusinersen er et antisense-oligonukleotid (ASO), som øger andelen af exon 7-inklusion i survival motor neuron 2 (SMN2)-messenger-ribonukleinsyre (mRNA)-transkripter ved at binde til et intronsplejsnings-inaktiveringssted (intronic splice silencing site, ISS-N1), der findes i intron 7 af SMN2 præ-messenger ribonukleinsyre (præ-mRNA).
English: Taken together, there is sufficient evidence to support a casual association between with nusinersen administration and hypersensitivity; and the PI should be updated accordingly.
Danish: Samlet set er der tilstrækkelig evidens til at understøtte en kausal sammenhæng mellem administration af nusinersen og overfølsomhed, og produktinformationen skal opdateres i overensstemmelse dermed.
English: Pharmacotherapeutic group: Other drugs for disorders of the musculo-skeletal system, ATC code: M09AX07
Danish: Farmakoterapeutisk klassifikation: andre midler mod lidelser i muskler, led og knogler, ATC kode: M09AX07
English: In the efficacy set, 18 patients (25%) in the Spinraza group and 12 patients (32%) in the sham-control group required permanent ventilation.
Danish: I gruppen for effektevaluering havde 18 patienter (25 %) i Spinraza-gruppen og 12 patienter (32 %) i sham-kontrolgruppen behov for permanent ventilering.
English: The median age at screening was 3 years, and the median age of onset of clinical signs and symptoms of SMA was 11 months.
Danish: Medianalderen ved screening var 3 år, og medianalderen for debut af kliniske tegn og symptomer på SMA var 11 måneder.
English: Your doctor will tell you how long you or your child need to receive Spinraza.
Danish: Din læge vil fortælle dig, hvor længe du eller dit barn skal have Spinraza.
English: The analysis included 28 patients who received their first dose in study CS2, and then transferred to the extension phase, study CS12.
Danish: Analysen omfattede 28 patienter, der fik deres første dosis i studie CS2, og dernæst overgik til forlængelsesfasen,
English: There have been a small number of reports of patients developing hydrocephalus (a build-up of too much fluid around the brain) after Spinraza is given.
Danish: Der har været et lille antal rapporter om patienter, der har udviklet hydrocephalus (en ophobning af for meget væske omkring hjernen) efter behandling med Spinraza.
English: The safety and efficacy in patients with renal impairment has not been established and they should be closely observed.
Danish: Sikkerhed og virkning hos patienter med nedsat nyrefunktion er ikke klarlagt, og de skal overvåges nøje.
English: Of these patients, 6 (33%) in the Spinraza group and 0 (0%) in the sham-control group met the protocol-defined criteria for a motor-milestone responder.
Danish: Ud af disse patienter opfyldte 6 (33 %) i Spinraza-gruppen og 0 (0 %) i sham-kontrolgruppen de definerede kriterier i protokollen for et motorisk milepæl-respons.
English: Any unused product or waste material must be disposed of in accordance with local requirements.
Danish: Ikke anvendt lægemiddel samt affald heraf skal bortskaffes i henhold til lokale retningslinjer.
English: Improvements in motor function were observed among patients continuing Spinraza from Study CS3B, as well as those who initiated Spinraza in Study CS11 (Figure 3), with the greatest benefit observed in those with earlier treatment initiation.
Danish: Motoriske forbedringer blev observeret hos patienter, der fortsatte med Spinraza fra studie CS3B, samt dem, der startede med Spinraza i studie CS11 (figur 3), hvor den største fordel blev observeret hos dem, der havde startet behandlingen tidligere.
English: The benefits-and risks of nusinersen treatment in patients with a ventriculoperitoneal shunt are unknown at present and the maintenance of treatment needs to be carefully considered.
Danish: Fordelene og risiciene ved behandling med nusinersen hos patienter med en ventrikulo-peritoneal shunt er på nuværende tidspunktukendte, og en opretholdelse af behandlingen bør overvejes nøje.
English: The mean CHOP INTEND score at last assessment was 61.0 (46 - 64) amongst patients with 2 SMN2 copies and 62.6 (58 - 64) amongst those with 3 SMN2 copies.
Danish: Den gennemsnitlige CHOP INTEND-score ved den sidste vurdering var 61,0 (46-64) blandt patienter med 2 SMN2-kopier og 62,6 (58-64) blandt patienter med 3 SMN2-kopier.
English: The median age at first dose was 162 days.
Danish: Medianalderen ved første dosis var 162 dage.
English: Lumbar puncture procedure
Danish: Lumbalpunkturprocedure
English: Figure 3: Change in HINE Motor Milestones versus Study days for Study CS3B (treated and sham-control), CS3A, CS5 and CS11
Danish: Figur 3: Ændring i HINE motoriske milepæle over tid for studie CS3B (behandlede og sham-kontrol), CS3A, CS5 og CS11
English: Each vial is for single use.
Danish: Hvert hætteglas er til engangsbrug.
English: If a maintenance dose is delayed or missed, Spinraza should be administered as soon as possible and dosing continued every 4 months.
Danish: Hvis en vedligeholdelsesdosis er forsinket eller glemt, skal Spinraza administreres snarest muligt, og doseringen skal fortsætte hver 4. måned.
English: This injection, called a lumbar puncture, is done by inserting a needle into the space around the spinal cord.
Danish: Denne injektion, som kaldes en lumbalpunktur, foretages ved at indføre en kanyle i rummet rundt om rygmarven.
English: Do not use this medicine after the expiry date which is stated on the carton after “EXP”.
Danish: Brug ikke lægemidlet efter den udløbsdato, der står på yderkartonen efter EXP. Udløbsdatoen er den sidste dag i den nævnte måned.
English: An improvement in mean CHOP INTEND score over time was observed from baseline to day 1072 (mean change 21.30).
Danish: En forbedring i gennemsnitlig CHOP INTEND-score over tid blev observeret fra baseline til dag 1072 (gennemsnitlig ændring 21,30).
English: Some adverse events commonly associated with lumbar puncture (e.g. headache and back pain) could not be assessed in the infant population exposed to Spinraza due to the limited communication appropriate for that age group.
Danish: Visse bivirkninger, der er almindeligt forbundet med lumbalpunktur (f.eks. hovedpine og rygsmerter), kunne ikke vurderes i spædbørnspopulationen, der blev eksponeret for Spinraza, på grund af den begrænsede kommunikation, der er mulig med denne aldersgruppe.
English: Mean plasma Cmax and AUC values increased approximately dose proportionally over the evaluated dose range.
Danish: Gennemsnitlige Cmax- og AUC-værdier blev ca. dosisproportionalt forøget over det evaluerede dosisinterval.
English: Submission of study results: Q2 2024.
Danish: Fremsendelse af studieresultater: 2. kvartal 2024
English: Upon completion of Study CS4 (CHERISH) 125 patients enrolled in Study CS11 (SHINE), where all patients received Spinraza.
Danish: Ved afslutningen af studie CS4 (CHERISH) blev 125 patienter inkluderet i studie CS11 (SHINE), hvor alle patienterne fik Spinraza.
English: Some of these patients had needed to have a device called a ventriculo-peritoneal shunt implanted to treat the hydrocephalus.
Danish: Hos nogle af disse patienter var det nødvendigt at implantere en anordning, der kaldes en ventrikulo-peritoneal shunt, til behandling af hydrocephalus.
English: These results are supported by an open-label Phase 2 study in symptomatic patients diagnosed with SMA (CS3A).
Danish: Disse resultater støttes af et åbent fase 2-studie hos symptomatiske patienter diagnosticeret med SMA (CS3A).
English: Pack size of one vial per carton.
Danish: Pakningsstørrelse med et hætteglas pr. karton.
English: Treatment should be administered by health care professionals experienced in performing lumbar punctures.
Danish: Behandlingen skal administreres af sundhedspersonale med erfaring i at udføre lumbalpunktur.
English: This will be done by a doctor experienced in doing lumbar punctures.
Danish: Dette udføres af en læge med erfaring i at lægge lumbalpunkturer.
English: Other products that are in the same group of medicines as Spinraza have been shown to affect the cells in the blood which help clotting.
Danish: Andre produkter, der tilhører den samme gruppe af lægemidler som Spinraza, har vist sig at påvirke de celler i blodet, der medvirker til størkning.
English: Communicating hydrocephalus, aseptic meningitis and hypersensitivity (e.g. angioedema, urticaria and rash) have also been reported.
Danish: Der er også blevet rapporteret kommunikerende hydrocephalus, aseptisk meningitis og overfølsomhed (f.eks. angioødem, urticaria og udslæt).
English: Before you or your child are given Spinraza your doctor may decide to do a urine test to check that your or your child's kidneys are working normally.
Danish: Før du eller dit barn får Spinraza kan din læge beslutte at lave en urinanalyse for at kontrollere, at dine eller dit barns nyrer fungerer normalt.
English: Two previously non-independent ambulatory patients (Type III) achieved independent walking, and one non-ambulatory patient (Type II) achieved independent walking.
Danish: To patienter, der tidligere kunne gå med støtte (type III) opnåede at kunne gå uden støtte, og én patient, der tidligere ikke kunne gå (type II) opnåede at kunne gå uden støtte.
English: Study CS11 is an open label extension study for SMA patients who previously participated in the other Spinraza clinical studies.
Danish: Studie CS11 er et åbent forlængelsesstudie for SMA-patienter, der tidligere har deltaget i andre kliniske studier med Spinraza.
English: Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.
Danish: Spinraza er indiceret til behandling af 5q spinal muskelatrofi.
English: Table 3: Primary and secondary endpoints at final analysis - Study CS41 Spinraza treated Patients
Danish: Tabel 3: Primære og sekundære endepunkter ved den endelige analyse - studie CS41 Spinraza-behandlede patienter
English: Figure 2: Mean change from baseline in HFMSE score over time at final analysis (ITT)Study CS4 1,2
Danish: Figur 2: Gennemsnitlig ændring fra baseline i HFMSE-score over tid ved den endelige analyse (ITT) - studie CS4 1,2
English: At baseline, patients had a mean Hammersmith Functional Motor Scale Expanded (HFMSE) score of 21.6, a mean revised upper limb module (RULM) of 19.1, all had achieved independent sitting, and no patients had achieved independent walking.
Danish: Ved baseline havde patienterne en gennemsnitlig HFMSE-score (Hammersmith Functional Motor Scale Expanded) på 21,6, en gennemsnitlig RULM-score (Revised Upper Limb Module) på 19,1, alle havde opnået at sidde uden støtte, og ingen af patienterne havde opnået at gå uden støtte.
English: A comparison of motor milestone achievement among the patients with symptomatic infantile-onset SMA and pre-symptomatic SMA is shown in Figure 3.
Danish: En sammenligning af opnåede motoriske milepæle blandt patienter med symptomatisk SMA med debut i spædbarnsalderen og præsymptomatisk SMA vises på figur 3.
English: SMA is a clinical spectrum of disease with disease severity linked to fewer numbers of SMN2 gene copies and younger age of symptom onset.
Danish: SMA er et klinisk sygdomsspektrum, hvor sygdommens sværhedsgrad er koblet til lavere antal af SMN2-genkopier og en yngre alder ved symptomdebut.
English: At the interim analysis, all 25 patients (2 SMN2 gene copies, n=15; 3 SMN2 gene copies, n=10) were alive without permanent ventilation..
Danish: Ved interimanalysen var alle 25 patienter (2 SMN2-genkopier, n=15, 3 SMN2genkopier, n=10) i live uden permanent ventilering.
English: The protocol-defined criteria for clinically manifested SMA included age-adjusted weight below the fifth WHO percentile, a decrease of 2 or more major weight growth curve percentiles, the placement of a percutaneous gastric tube, and/or the inability to achieve expected age-appropriate WHO milestones (sitting without support, standing with assistance, hands-and-knees crawling, walking with assistance, standing alone and walking alone).
Danish: Protokoldefinerede kriterier for klinisk manifestation af SMA omfattede aldersjusteret vægt under det femte WHO-percentil, en reduktion på 2 eller flere større vægt/vækstkurvepercentiler, anlæggelse af en perkutan gastrisk slange og/eller manglende evne til at opnå forventede alderssvarende WHO-milepæle (sidde uden støtte, stå med hjælp, kravle på hænder og knæ, gå med hjælp, stå alene og gå alene).
English: An additional clinical study, CS7 (EMBRACE) was opened for patients not eligible for participation in Study CS3B or Study CS4 due to screening age or SMN2 copy number.
Danish: Der blev givet adgang til et yderligere klinisk studie, CS7 (EMBRACE), for patienter, der ikke var egnede til at deltage i studie CS3B eller CS4 som følge af deres alder på screeningstidspunktet eller antallet af SMN2-kopier.
English: Survival Event-free survival2
Danish: Overlevelse Hændelsesfri overlevelse2
English: Thrombocytopenia and coagulation abnormalities, including acute severe thrombocytopenia, have been observed after administration of other subcutaneously or intravenously administered antisense oligonucleotides.
Danish: Koagulationsanormaliteter og trombocytopeni, herunder akut svær trombocytopeni, er observeret efter administration af andre subkutant eller intravenøst administrerede antisense-oligonukleotider.
English: If no refrigeration is available, Spinraza may be stored in its original carton, protected from light at or below 30°C for up to 14 days.
Danish: Hvis der ikke er et tilgængeligt køleskab, kan Spinraza opbevares i den originale yderkarton beskyttet mod lys ved eller under 30 °C i op til 14 dage.
English: Spinraza is administered as an intrathecal bolus injection over 1 to 3 minutes, using a spinal anaesthesia needle.
Danish: Spinraza administreres som en intratekal bolusinjektion i løbet af 1 til 3 minutter med en kanyle til spinalanæstesi.
English: Baseline disease characteristics were generally similar with the exception of an imbalance in the proportion of patients who had ever achieved the ability to stand without support (13% of patients in the Spinraza group and 29% in sham-control) or walk with support (24% of patients in the Spinraza group and 33% in sham-control).
Danish: Sygdomskarakteristika ved baseline var generelt sammenlignelige, med undtagelse af en ubalance i andelen af patienter, som havde opnået evnen til at stå op uden støtte (13 % af patienterne i Spinraza-gruppen og 29 % i shamkontrolgruppen) eller gå med støtte (24 % af patienterne i Spinraza-gruppen og 33 % i shamkontrolgruppen).