FrancophonIA/ELRC3.0 huggingface dataset:
English: Adverse reactions were mostly mild to moderate in intensity and almost all occurred during the infusion or during the 2 hours following the infusion (infusion associated reactions, IARs).
Danish: Bivirkningerne var for det meste milde til moderate og forekom næsten alle under infusionen eller i de 2 timer umiddelbart efter infusionen (infusionsrelaterede reaktioner, IAR'er).
English: Do not invert, swirl or shake the vial.
Danish: Hætteglasset må ikke vendes, hvirvles eller rystes.
English: Other medicines and Myozyme
Danish: Brug af anden medicin sammen med Myozyme
English: Take care when driving or using any tools or machines shortly after infusion of Myozyme, since you may experience dizziness.
Danish: Kør ikke bil eller motorcykel og lad være med at cykle kort efter infusion af Myozyme, fordi De kan føle dig svimmel.
English: General disorders and administration site conditions very common
Danish: Almene symptomer og reaktioner på administrationsstedet meget almindelig
English: In a placebo-controlled study lasting 78 weeks, 90 patients with late-onset Pompe disease, aged 10 to 70 years, were treated with Myozyme or placebo randomized in a 2:1 ratio (see section 5.1).
Danish: I et placebokontrolleret randomiseret studie, der varede 78 uger, blev 90 patienter med sent debuterende Pompes sygdom i alderen 10-70 år behandlet med Myozyme eller placebo i forholdet 2:1 (se pkt.
English: Some IgE positive patients were successfully rechallenged with Myozyme using a slower infusion rate at lower initial doses and have continued to receive Myozyme under close clinical supervision.
Danish: Hos nogle IgE-positive patienter er reeksponering for Myozyme lykkedes ved anvendelse af langsommere infusionshastighed og lavere initialdoser, og patienterne kunne fortsætte behandlingen med Myozyme under nøje klinisk supervision.
English: Survival in the treated patients was compared to survival in a similar historical cohort of untreated subjects using a Cox proportional hazards regression analysis (See Table 3).
Danish: Overlevelsen hos de behandlede patienter blev sammenlignet med overlevelse hos en lignende historisk gruppe af ubehandlede forsøgspersoner ved hjælp af en Cox proportionel risiko regressions analyse (se Tabel 3).
English: It is recommended that the infusion begin at an initial rate of 1 mg/kg/h and be gradually increased by 2 mg/kg/h every 30 minutes if there are no signs of infusion associated reactions (IARs) until a maximum rate of 7 mg/kg/h is reached.
Danish: Det anbefales at infusionen indledes med en hastighed på 1 mg/kg/t og øges gradvist med 2 mg/kg/t for hver 30 minutter, hvis der ikke er nogen tegn på infusionsrelaterede reaktioner (IAR), indtil en maksimal hastighed på 7 mg/kg/t.
English: Each vial of Myozyme is for single use only.
Danish: Hvert enkelt hætteglas Myozyme kun til engangsbrug.
English: Ten patients with advanced late-onset Pompe disease (i.e. wheelchair-bound for 10/10 and ventilatordependent for 9/10) aged 9-54 years were treated in expanded access programs with alglucosidase alfa 20-40 mg/kg once every two weeks for various periods of time between 6 months and 2.5 years.
Danish: 10 patienter med fremskreden sent debuterende Pompes sygdom (dvs. 10/10 bundet til kørestol og 9/10 afhængig af ventilationsbehandling) i alderen 9-54 år blev behandlet i ”expanded access” behandlingsprogrammer med alglucosidase alfa 20-40 mg/kg én gang hver anden uge i perioder af forskellig varighed på mellem 6 måneder og 2,5 år.
English: Late-onset Pompe disease; other clinical trials and analyses Three independent, open-label, single arm, investigator-initiated studies with Myozyme were conducted:
Danish: Sent debuterende Pompes sygdom; andre kliniske undersøgelser og analyser Tre uafhængige investigator-initerede, åbne, enkeltarmede kliniske studier blev udført med Myozyme:
English: Because of the potential for severe infusion associated reactions, appropriate medical support measures, including cardiopulmonary resuscitation equipment, should be readily available when Myozyme is administered.
Danish: På grund af risikoen for svære infusionsrelaterede reaktioner, skal der være nødhjælpsbehandling til rådighed herunder genoplivningsudstyr, ved indgivelse af Myozyme.
English: After 52 weeks of treatment 14 of 18 patients (77.8%) had maintained or improved weight-for-age percentiles (above the 3rd percentile), 14 of 15 patients (93.3%) were above the 3rd percentile for length and 12 of 15 patients (80.0%) were above the 3rd percentile for head circumference.
Danish: Efter 52 ugers behandling havde 14 af 18 patienter (77,8 %) opnået vedligeholdelse eller forbedring i vægt/alder-percentilen (over 3. percentil), 14 af 15 patienter (93,3 %) var over den 3. percentil for højde, og 12 af 15 patienter (80,0 %) var over den 3. percentil for hovedomkreds.
English: Muscle spasms Muscle twitching Myalgia not known
Danish: Muskelspasmer Muskeltrækninger Myalgi ikke kendt
English: Nephrotic syndrome was observed in a few patients with Pompe disease treated with alglucosidase alfa and who had high IgG antibody titres (≥ 102,400) (see section 4.8).
Danish: Nefrotisk syndrom blev observeret hos nogle få patienter med Pompes sygdom, der blev behandlet med alglucosidase alfa, og som havde høje IgGantistoftitre (≥ 102.400) (se pkt.
English: Patients who experience hypersensitivity reactions may also be tested for IgE antibodies to alglucosidase alfa and other mediators of anaphylaxis.
Danish: Patienter, der får overfølsomhedsreaktioner, kan desuden testes for IgE-antistoffer mod alglucosidase alfa og andre mediatorer for anafylaksi.
English: Table 4: Change from baseline: efficacy outcomes in the placebo-controlled study
Danish: Ændring fra baseline: effektudfald i det placebokontrollerede studie Myozyme (N = 60)
English: The remaining 7 patients made no clinically significant motor gains or were unable to sustain the motor gains made and had very limited motor movement by the last study assessment (with individual patient treatment durations ranging from 52 to 142 weeks; mean follow-up period of 103 weeks).
Danish: De resterende 7 patienter opnåede ingen klinisk signifikant forbedring i den motoriske funktion, eller de kunne ikke opretholde forbedringen og havde meget begrænset motorisk bevægelse ved den sidste vurdering (med individuelle patientbehandlingsvarigheder fra 52 til 142 uger: gennemsnitlig opfølgningsperiode på 103 uger).
English: Of the 21 patients, 16 survived through the end of this study, with a median time from ERT initiation to last assessment of 44.6 months (range: 5.7 to 105.47); 5 patients died due to respiratory failure and disease progression, all of whom were ERTnaïve at the start of ERT+ITI treatment.
Danish: Af de 21 patienter overlevede 16 til og med studiets afslutning med en mediantid fra initiering af enzymerstatningsterapi til sidste evaluering på 44,6 måneder (interval: 5,7 til 105,47); 5 patienter døde på grund af respirationssvigt og sygdomsprogression, og de var alle enzymerstatningsterapi-naive ved initiering af enzymerstatningsterapi+ITI-behandling.
English: Patients with late-onset Pompe disease typically present with progressive myopathy, predominantly of the proximal muscles in the pelvic and shoulder girdles, and varying degrees of respiratory involvement, ultimately progressing to profound disability and/or the need for ventilatory support.
Danish: Patienter med sent debuterende Pompes sygdom præsenterer sig typisk med progredierende myopati, hovedsagelig af de proksimale muskler i bækkenområdet og skulderbælte og forskellige grader af respiratorisk involvering, som til sidst udvikler sig til udtalt invaliditet og/eller behov for ventilations support.
English: Tilt and roll each vial gently.
Danish: Vip og rul hvert hætteglas forsigtigt.
English: • sodium dihydrogen phosphate monohydrate (E339)
Danish: • Natriumdihydrogenphosphatmonohydrat (E339)
English: Infantile onset patients who develop high IgG antibody titres appear to be at higher risk for developing more frequent IARs.
Danish: Patienter med debut i spædbørnsalderen, som udvikler høje IgG-antistoftitre, har tilsyneladende større risiko for at udvikle hyppige infusionsrelaterede reaktioner.
English: Respiratory arrest Apnea
Danish: Åndedrætsstop Apnø
English: In clinical trials, 39 infantile-onset patients were treated with Myozyme for more than three years (168 weeks with a median of 121 weeks; see section 5.1).
Danish: I kliniske undersøgelser blev 39 patienter, der debuterede i spædbørnsalderen, behandlet med Myozyme i mere end tre år (168 uger med en median på 121 uger, se pkt.
English: The remaining 5 patients showed no significant change in motor development beyond Week 52 (with individual patient treatment durations ranging from 104 to 168 weeks; mean follow-up period of 140 weeks), including 4 patients with no significant motor skills in any of the positions evaluated and 1 patient with only functional sitting skills by the last study visit.
Danish: De resterende 5 patienter udviste ingen signifikant ændring i den motoriske funktion efter uge 52 (med individuelle patientbehandlingsvarigheder fra 104 til 168 uger: gennemsnitlig opfølgningsperiode på 140 uger), herunder 4 patienter uden signifikant motorisk funktion i nogen af de positioner, der blev vurderet, og 1 patient, der kun kunne sidde ved det sidste besøg i studiet.
English: Package leaflet: Information for the user Myozyme 50 mg powder for concentrate for solution for infusion
Danish: Indlægsseddel: Information til brugeren Myozyme 50 mg pulver til koncentrat til infusionsvæske, opløsning
English: Also remove an equal volume of sodium chloride 9 mg/ml (0.9%) solution for injection, that will be replaced with reconstituted Myozyme.
Danish: Fjern også en lige så stor mængde 9 mg/ml (0,9 %) natriumchloridopløsning til injektion, som vil blive erstattet med rekonstitueret Myozyme.
English: Immune system disorders common
Danish: Immunsystemet almindelig
English: Confidence Interval p-value
Danish: Ratio mellem behandlingseffekt og risiko konfidensinterval p-værdi
English: 2 Reactions reported in 60 late-onset patients in a placebo-controlled clinical trial.
Danish: 2 Bivirkninger, der er indberettet fra 60 patienter med sen debut i et placebokontrolleret klinisk forsøg.
English: Some patients have been successfully rechallenged and continued to receive alglucosidase alfa under close clinical supervision.
Danish: Hos visse patienter er reeksponering og fortsat behandling med alglucosidase alfa under nøje klinisk supervision lykkedes.
English: There are no clinical data on the effects of alglucosidase alfa on fertility.
Danish: Der foreligger ingen kliniske data om påvirkningen af alglucosidase alfa på fertilitet.
English: • Inflammation of membrane that covers eyeball and eyelid
Danish: • Betændelse i membranen der dækker øjeæblet og øjenlåget
English: There was no evidence that IgG antibodies to alglucosidase alfa affected pharmacokinetics.
Danish: Der var ingen tegn på, at IgG-antistoffer over for alglucosidase alfa påvirkede farmakokinetikken.
English: Respiratory, thoracic and mediastinal disorders very common
Danish: Luftveje, thorax og mediastinum meget almindelig
English: A non-typical, more slowly progressive form of infantile-onset Pompe disease has been described which is characterised by a less severe cardiomyopathy and consequently a more prolonged survival.
Danish: En atypisk, mere langsomt progredierende form af Pompes sygdom med debut i spædbørnsalderen er beskrevet og kendetegnes ved mindre svær kardiomyopati og dermed længerevarende overlevelse.
English: Symptoms of life-threatening allergic reactions include, but are not limited to, low blood pressure, very fast heart rate, difficulty breathing, vomiting, facial swelling, hives or rash.
Danish: Symptomer på livstruende, allergiske reaktioner inkluderer, men er ikke begrænset til, lavt blodtryk, meget hurtig hjertefrekvens (puls), besværet åndedræt, opkastning, opsvulmet ansigt, nældefeber eller udslæt.
English: Patients received 20 mg/kg Myozyme once every two weeks for 26 weeks.
Danish: Patienterne fik 20 mg/kg Myozyme én gang hver anden uge i 26 uger.
English: Reconstitute each 50 mg vial of Myozyme with 10.3 ml water for injections.
Danish: Hvert 50 mg hætteglas Myozyme rekonstitueres med 10,3 ml vand til injektionsvæske.
English: • Feeling hot
Danish: • Varm fornemmelse
English: • Severe ulcerative lesions of the skin
Danish: • Alvorlige sår i huden
English: • One study in the Netherlands enrolled 69 late-onset patients with a median follow-up of 23
Danish: • Et studie i Holland inkluderede 69 sent debuterende patienter med en gennemsnitlig opfølgning på 23 måneder.
English: The recommended dose regimen of alglucosidase alfa is 20 mg/kg of body weight administered once every 2 weeks.
Danish: Den anbefalede doseringsplan for alglucosidase alfa er 20 mg/kg kropsvægt indgivet en gang hver anden uge.
English: Your doctor should consider the risks and benefits of re-administering Myozyme.
Danish: Deres læge bør vurdere fordele og ulemper ved at starte behandling med Myozyme igen.
English: Additional adverse reactions4
Danish: Yderligere bivirkninger4
English: The recommended dose regimen of Myozyme is 20 mg/kg of body weight administered once every 2 weeks as an intravenous infusion.
Danish: Den anbefalede doseringsplan for Myozyme er 20 mg/kg kropsvægt indgivet en gang hver anden uge som intravenøs infusion.
English: Name and address of the manufacturers of the biological active substance Genzyme Corporation 45, 51, 76, 74 and 80 New York Avenue, Framingham, MA 01701, United States
Danish: Navn og adresse på fremstillerne af det biologisk aktive stof Genzyme Corporation 45, 51, 74, 76 and 80 New York Avenue, Framingham, MA 01701, USA Genzyme Flanders bvba, Cipalstraat 8, 2440 Geel, Belgien Navn og adresse på den fremstiller, der er ansvarlig for batchfrigivelse Genzyme Ireland Limited, IDA Industrial Park, Old Kilmeaden Road, Waterford, Irland
English: Immunomodulation
Danish: Immunmodulation
English: Infantile-onset Pompe disease; clinical trial in patients aged 6 months or less The safety and efficacy of Myozyme was assessed in a pivotal, randomised, open-label, historicallycontrolled clinical trial of 18 non-ventilated infantile-onset patients aged 6 months or less at the onset of treatment.
Danish: Pompes sygdom med debut i spædbørnsalderen; klinisk forsøg med patientalder op til 6 måneder Sikkerhed og effekt for Myozyme er vurderet i en randomiseret, open-label, historisk kontrolleret klinisk pivotalundersøgelse med 18 ikke-ventilerede patienter med sygdomsdebut i spædbørnsalderen og i alderen 6 måneder eller mindre ved behandlingens start.
English: • disodium phosphate heptahydrate (E339)
Danish: • Dinatriumphosphatheptahydrat (E339)
English: Myozyme is indicated in adults and paediatric patients of all ages.
Danish: Myozyme er indiceret til voksne og børn i alle aldre.
English: Renal and urinary disorders not known
Danish: Nyrer og urinveje ikke kendt
English: Based on these results the 20 mg/kg qow dose is recommended.
Danish: Baseret på disse resultater anbefales en dosis på 20 mg/kg hver anden uge.
English: Myozyme has to be reconstituted with water for injections, then diluted with sodium chloride 9 mg/ml (0.9%) solution for injection and then administered by intravenous infusion.
Danish: Myozyme skal rekonstitueres med vand til injektionsvæske, derefter fortyndes med 9 mg/ml (0,9 %) natriumkloridopløsning til injektion, og derefter indgives ved intravenøs infusion.
English: Myozyme is a powder for concentrate for solution for infusion in a vial (50 mg/vial).
Danish: Myozyme er et pulver til koncentrat til infusion, opløsning (i et hætteglas (50 mg/hætteglas).
English: Myozyme 50 mg powder for concentrate for solution for infusion Alglucosidase alfa
Danish: Myozyme 50 mg pulver til koncentrat til infusionsvæske, opløsning Alglucosidase alfa
English: *One patient who did not have data post baseline was excluded from the analyses.
Danish: Wilcoxon-Mann-Whitney-test p-værdi *Én patient, som ikke havde nogen data efter baseline, blev ekskluderet fra analyserne.
English: Because it is a recombinant human protein, alglucosidase alfa is an unlikely candidate for cytochrome P450 mediated drug-drug interactions.
Danish: Da alglucosidase alfa er et rekombinant, humant protein, er det usandsynligt, at det vil medføre CYP-medierede lægemiddelinteraktioner.
English: IARs are defined as any related adverse event occurring during the infusion or during the hours following infusion.
Danish: Infusionsrelaterede reaktioner er defineret som enhver bivirkning, der indtræffer under infusionen eller i løbet af timerne efter infusionen.
English: Feeling hot not known
Danish: Varmefølelse ikke kendt
English: An additional 4 patients made motor development gains during the study and were sitting independently by the last study assessment (with individual patient treatment durations ranging from 78 to 130 weeks; mean follow-up period of 110 weeks), although they did not have functional use of the legs.
Danish: Yderligere 4 patienter opnåede bedre motorisk funktion under studiet og kunne sidde selv ved den sidste vurdering (med individuelle patientbehandlingsvarigheder fra 78 til 130 uger: gennemsnitlig opfølgningsperiode på 110 uger), selvom de ikke kunne bruge benene.
English: After 52 weeks of treatment, LVM decreased from baseline in all 14 patients with available data and was within normal limits in 3 of 14 patients.
Danish: Efter 52 ugers behandling faldt LVM fra baseline hos alle 14 patienter med tilgængelige data og var inden for normalgrænserne hos 3 af 14 patienter.
English: As measured by motor performance age-equivalent scores of the Alberta Infant Motor Scale (AIMS), seven of the 18 patients made motor development gains during the study and were walking independently by the last study assessment (with individual patient treatment durations ranging from 52 to 130 weeks; mean follow-up period of 94 weeks).
Danish: Målt efter motorisk præstation i form af aldersækvivalente scorer på Alberta Infant Motor Scale (AIMS) opnåede syv af 18 patienter bedre motorisk funktion under studiet og kunne gå selv ved den sidste vurdering (med individuelle patientbehandlingsvarigheder fra 52 til 130 uger: gennemsnitlig opfølgningsperiode på 94 uger).
English: Patients who develop IgE antibodies to alglucosidase alfa appear to be at a higher risk for the occurrence of IARs when Myozyme is readministered (see section 4.8).
Danish: Patienter, der udvikler IgE-antistoffer med alglucosidase alfa, synes at have en forhøjet risiko for forekomst af infusionsrelaterede reaktioner, når Myozyme indgives igen (se pkt.
English: After 52 weeks of treatment, LVM decreased from baseline in all 12 patients with available data and was within normal limits in 6 of 12 patients.
Danish: Efter 52 ugers behandling faldt LVM fra baseline hos alle 12 patienter med tilgængelige data, og værdien var inden for normalgrænserne hos 6 af 12 patienter.
English: Six of the 11 patients with available data continued to make motor development gains beyond Week 52 (with individual patient treatment durations ranging from 58 to 168 weeks; mean follow-up period of 121 weeks), including 3 patients ambulatory and 3 patients with only functional sitting skills by the last study visit.
Danish: Seks af de 11 patienter med tilgængelige data fortsatte med at forbedre den motoriske funktion efter uge 52 (med individuelle patientbehandlingsvarigheder fra 58 til 168 uger: gennemsnitlig opfølgningsperiode på 121 uger), herunder 3 ambulante patienter og 3 patienter, der kun kunne sidde ved det sidste besøg i studiet.
English: In the rabbit embryofoetal development study, following administration of Myozyme (10-40 mg/kg/day) with coadministration of diphenhydramine, a treatment-related increase in the incidence of abortions and early delivery was observed.
Danish: I studiet af den embryoføtale udvikling hos kaniner blev der observeret en behandlingsrelateret forøgelse af incidensen af aborter og tidlig fødsel efter administration af Myozyme (10-40 mg/kg/dag) med samtidig administration af diphenhydramin.
English: The most common adverse reactions observed were IARs.
Danish: De almindeligste bivirkninger, der blev observeret, var infusionsrelaterede reaktioner.
English: • Protein loss in urine
Danish: • Proteintab via urinen
English: The infantile-onset form of Pompe disease is characterised by massive deposition of glycogen in the heart, and skeletal muscle always resulting in rapidly progressive cardiomyopathy, generalised muscle weakness and hypotonia.
Danish: Pompes sygdom, der debuterer i spædbørnsalderen, kendetegnes ved massiv aflejring af glykogen i hjertet og skeletmusklerne, hvilket altid medfører hurtigt progredierende kardiomyopati, general muskelsvækkelse og hypotoni.
English: Tachypnoea Cough common
Danish: Takypnø Hoste almindelig
English: If you have experienced life-threatening allergic (hypersensitive) reactions to alglucosidase alfa or any of the other ingredients of this medicine (listed in section 6) and re-administration of the medicine was not successful.
Danish: hvis De har oplevet livstruende allergiske (overfølsomheds) reaktioner over for alglucosidase alfa eller et af de øvrige indholdsstoffer i Myozyme (angivet i punkt 6.
English: Psychiatric disorders common Agitation not known
Danish: Psykiske forstyrrelser almindelig ikke kendt
English: At 104 weeks of treatment LVM assessments were available for 8 patients, of which 5 decreased to within normal limits.
Danish: Efter 104 ugers behandling var LVM-vurderingerne tilgængelige for 8 patienter, hvoraf værdien faldt til indenfor normalgrænserne hos 5.
English: Use of ITI and alglucosidase alfa has been evaluated in 1 clinical trial and a retrospective chart review of patients naïve to ERT at the initiation of treatment and 1 clinical trial of patients already receiving alglucosidase alfa at time of initiating ITI.
Danish: Immuntoleranceinduktion - Pompes sygdom med debut i spædbørnsalderen (IOPD) Anvendelse af ITI og alglucosidase alfa er blevet undersøgt i 1 klinisk studie og en retrospektiv journalgennemgang af patienter, som var naive overfor enzymerstatningsterapi ved behandlingsstart og 1 klinisk studie med patienter, der allerede fik alglucosidase alfa, da ITI initieredes.
English: • Bluish discolouration of the skin
Danish: • Blålig misfarvning af huden
English: A tendency was observed in infantile patients treated with a higher dose (40 mg/kg) to experience more symptoms when developing IARs.
Danish: Hos patienter med debut i spædbørnsalderen behandlet med en højere dosis (40 mg/kg) blev der observeret en tendens til at opleve flere symptomer ved udvikling af infusionsrelaterede reaktioner.
English: Nephrotic syndrome as well as severe cutaneous reactions, possibly immune mediated, have been reported with alglucosidase alfa including ulcerative and necrotizing skin lesions (see section 4.4).
Danish: Nefrotisk syndrom samt alvorlige kutane reaktioner (muligvis immunmedierede) er blevet rapporteret med alglucosidase alfa, inklusive ulcerøse og nekrotiske hudlæsioner (se pkt.
English: Vascular disorders very common
Danish: Vaskulære sygdomme meget almindelig
English: The recommended final concentration of alglucosidase in the infusion bags ranges from 0.5 mg/ml to 4 mg/ml.
Danish: Den anbefalede endelige koncentration af alglucosidase i infusionsposen varierer fra 0,5 mg/ml til 4 mg/ml.
English: Serious and life-threatening anaphylactic reactions, including anaphylactic shock, have been reported in infantile- and late-onset patients during Myozyme infusions (see section 4.8).
Danish: Alvorlige og livstruende, anafylaktiske reaktioner, herunder anafylaktisk shock, er indberettet fra patienter, hvor sygdommen debuterer i spædbørnsalderen, og fra sent debuterende patienter under Myozyme-infusioner (se pkt.
English: • Irritability
Danish: • Irritation
English: Symptoms of such reactions include low blood pressure, very fast heart rate, difficulty breathing, vomiting, facial, lip or tongue swelling, hives or rash.
Danish: Symptomer på sådanne reaktioner omfatter lavt blodtryk, meget hurtig hjertefrekvens (puls), besværet åndedræt, opkast, opsvulmet ansigt, læber eller tunge, nældefeber eller udslæt.
English: • Eyes tearing
Danish: • Øjne, der løber