FrancophonIA/ELRC3.0 huggingface dataset:
English: Baseline disease characteristics were largely similar in the Spinraza treated patients and sham-control patients except that Spinraza treated patients at baseline had a higher percentage compared to sham-control patients of paradoxical breathing (89% vs 66%), pneumonia or respiratory symptoms (35% vs 22%), swallowing or feeding difficulties (51% vs 29%) and requirement for respiratory support (26% vs 15%).
Maltese: pnewmonja jew sintomi respiratorji (35% vs 22%), diffikultajiet biex wieħed jibla' jew jiekol (51% vs 29%), u l-ħtieġa għal appoġġ respiratorju (26% vs 15%).
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.
Maltese: Minn dawn il-pazjenti, 6 (33%) fil-grupp ta' Spinraza u 0 (0%) fil-grupp tal-kontroll ikkurati bi plaċebo, kisbu l-kriterji definiti filprotokoll għal persuna li rrispondiet għal milestone tal-moviment.
English: Two previously non-independent ambulatory patients (Type III) achieved independent walking, and one non-ambulatory patient (Type II) achieved independent walking.
Maltese: Żewġ pazjenti ambulatorji li fil-passat ma kinux indipendenti (Tip III) kisbu mixi indipendenti, u pazjent wieħed mhux ambulatorju (Tip II) kiseb mixi indipendenti.
English: Change from baseline in total HFMSE score at 15 months1,2,3
Maltese: Bidla mil-linja bażi fil-punteġġ totali HFMSE wara 15-il xahar1,2,3
English: Patients who initiated Spinraza treatment in Study CS11 (n=20) had a median age of 4.0 years (range 3 - 8 years).
Maltese: Pazjenti li bdew it-trattament bi Spinraza fi Studju CS11 (n=20) kellhom età medjana ta' 4.0 snin (medda 3 - 8 snin).
English: Nusinersen is metabolized slowly and predominantly via exonuclease (3'- and 5') -mediated hydrolysis and is not a substrate for, or inhibitor or inducer of CYP450 enzymes.
Maltese: Nusinersen jiġi metabolizzat bil-mod u l-biċċa l-kbira permezz ta' idrolisi medjata minn exonuclease (3'- u 5'), u mhuwiex substrat għal, jew inibitur jew induttur ta', enzimi CYP450.
English: An analysis of the subset of patients in the ITT population who had observed values at Month 15 demonstrated consistent, statistically significant results.
Maltese: ITT li kellhom valuri osservati f'Xahar 15 uriet riżultati konsistenti u statistikament sinifikanti.
English: Later onset
Maltese: Bijotrasformazzjoni
English: The analysis was conducted in the ITT population (Spinraza: n=84; sham-control: n=42), and post-baseline HFMSE data for patients without a Month 15 visit were imputed using the multiple imputation method.
Maltese: Lanaliżi twettqet fil-popolazzjoni ITT (Spinraza: n=84; grupp tal-kontroll bi plaċebo: n=42), u dejta HFMSE ta' wara l-linja bażi għall-pazjenti mingħajr viżta f'Xahar 15 kienet imputata bl-użu talmetodu ta' imputazzjoni multipla.
English: Each 5 ml vial contains nusinersen sodium equivalent to 12 mg nusinersen.
Maltese: Kull kunjett ta' 5 ml fih nusinersen sodium, ekwivalenti għal 12 mg ta' nusinersen.
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.
Maltese: Il-pazjenti kollha kellhom il-ħila li jsoffu u jibilgħu fl-aħħar evalwazzjoni, bi 22 (88%) tarbija li kisbu punteġġ massimu fuq il-HINE Sezzjoni 1.
English: Patients who first initiated Spinraza treatment in Study CS11 (n=24; sham control in Study CS3B) were of a median age of 17.8 months (range 10 - 23 months) and had a mean CHOP INTEND score of 17.25 (range 2.0 - 46.0) at baseline in Study CS11.
Maltese: Pazjenti li bdew trattament bi Spinraza l-ewwel fi Studju CS11 (n=24; kontroll bi plaċebo fi Studju CS3B) kienu ta' età medjana ta' 17.8 il xahar (medda 10 - 23 xahar) u kellhom punteġġ medju ta' CHOP INTEND ta' 17.25 (medda 2.0 - 46.0) fil-linja bażi fi Studju CS11. Fil-ħin tal-analiżi interim, 22 minn 24 pazjent (92%) kienu ħajjin.
English: Patients with profound hypotonia and respiratory failure at birth, where Spinraza has not been studied, may not experience a clinically meaningful benefit due to severe SMN protein deficiency.
Maltese: Pazjenti b'ipotonija u falliment respiratorju mit-twelid, fejn Spinraza ma ġietx studjata, jistgħu ma jesperjenzawx benefiċċju klinikament rilevanti minħabba nuqqas serju tal-proteina SMN.
English: Spinraza contains the active substance nusinersen which belongs to a group of medicines known as antisense oligonucleotides.
Maltese: Spinraza fih is-sustanza attiva nusinersen li jappartjeni għal grupp ta' mediċini magħrufa bħala antisense oligonucleotides.
English: The immunogenic response to nusinersen was determined in 346 patients with baseline and post-baseline plasma samples evaluated for anti-drug antibodies (ADA).
Maltese: Ir-rispons immunoġeniku għal nusinersen ġie stabbilit fi 346 pazjent b'kampjuni tal-plażma fil-linja bażi u wara l-linja bażi evalwati għal antikorpi kontra l-mediċina (ADA, anti-drug antibodies).
English: Spinraza is used to treat a genetic disease called spinal muscular atrophy (SMA).
Maltese: Spinraza jintuża biex jikkura marda ġenetika msejħa atrofija muskolari tas-sinsla (SMA, spinal muscular atrophy).
English: In these patients, a mean improvement of 28.6 meters (SD 47.22, n=12) at Day 253 and 86.5 metres (SD 40.58, n=8) at Day 1050.
Maltese: F'dawn il-pazjenti, titjib medju ta' 28.6 metru (SD 47.22, n=12) f'Jum 253 u 86.5 metru (SD 40.58, n=8) f'Jum 1050.
English: 5 ml in a Type I glass vial with bromobutyl rubber stopper and an aluminium over-seal and plastic cap.
Maltese: 5 ml f'kunjett tal-ħġieġ ta' Tip I, b'tapp tal-lastku tal-bromobutyl u over-seal tal-aluminju u għatu talplastik.
English: As a precautionary measure, it is preferable to avoid the use of Spinraza during pregnancy.
Maltese: Hu preferibbli li jiġi evitat l-użu ta' Spinraza waqt ittqala u t-treddigħ.
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.
Maltese: Fil-linja bażi, il-pazjenti kellhom punteġġ medju ta' 21.6 fuq il-Hammersmith Functional Motor Scale Expanded (HFMSE), modulu rivedut tad-dirgħajn (revised upper limb module;
English: Secondary endpoints including functional measures and WHO motor milestone achievement were formally statistically tested and are described in Table 3.
Maltese: Riżultati deskrittivi li jinkludu miżuri funzjonali u l-kisba ta' milestone tal-moviment WHO kienu formalment u statistikament ittestjati u huma deskritti f'Tabella 3.
English: Survival Event-free survival2
Maltese: Sopravivenza mingħajr avvenimenti2
English: Spinraza is administered as an intrathecal bolus injection over 1 to 3 minutes, using a spinal anaesthesia needle.
Maltese: Spinraza jingħata bħala injezzjoni bolus għal ġot-teka fuq perjodu ta' minn 1 sa 3 minuti, billi tintuża labra ta' anestesija fis-sinsla.
English: Luxembourg/Luxemburg
Maltese: Luxembourg/Luxemburg Biogen Belgium N.V./S.
English: Of the 45 patients who had not met the definition of permanent ventilation in Study CS3B, 38 patients (84%) were alive without permanent ventilation in Study CS11 at the time of interim analysis.
Maltese: Mill-45 pazjent li ma ssodisfawx id-definizzjoni ta' ventilazzjoni permanenti fi Studju CS3B, 38 pazjent (84%) kienu ħajjin mingħajr ventilazzjoni permanenti fi Studju CS11 fil-ħin tal-analiżi interim.
English: Of the twelve patients (50%) who had not met the definition of permanent ventilation in Study CS3B, 7 patients (58%) were alive without permanent ventilation in Study CS11.
Maltese: Mit-tnax-il pazjent (50%) li ma ssodisfawx iddefinizzjoni ta' ventilazzjoni permanenti fi Studju CS3B, 7 pazjenti (58%) kienu ħajjin mingħajr ventilazzjoni permanenti fi Studju CS11.
English: Of those with observed values at Month 15, a higher proportion of Spinraza treated subjects had improvement (73% vs 41%, respectively) and a lower proportion of Spinraza treated subjects had worsening (23% vs 44%, respectively) in total HFMSE score compared to sham-control.
Maltese: Fost dawk b'valuri osservati f'Xahar 15, rata ogħla ta' pazjenti kkurati bi Spinraza kellhom titjib (73% vs 41% rispettivament) u rata aktar baxxa ta' pazjenti kkurati bi Spinraza kellhom rigress (23% vs 44% rispettivament) f'punteġġ totali HFSME meta mqabbel mal-grupp tal-kontroll bi plaċebo.
English: In patients with Type II SMA the Upper Limb Module test was conducted with mean improvement of 1.9 (SD 2.68, n=11) at Day 253 and 3.5 (SD 3.32, n=9) at Day 1050.
Maltese: F'pazjenti b'SMA tat-Tip II it-test tal-Modulu tal-Dirgħajn twettaq b'titjib medju ta' 1.9 (SD 2,68, n=11) f'Jum 253 u 3.5 (SD 3.32, n=9) f'Jum 1050.
English: - The active substance is nusinersen.
Maltese: X'fih Spinraza - Is-sustanza attiva hi nusinersen.
English: The mean 6MWT distance was 278.5 meters (SD 206.46) at Day 253 and 333.6 metres (SD 176.47) at Day 1050.
Maltese: 46) f'Jum 253 u 333.6 metru SD 176.47) f'Jum 1050.
English: Spinraza treatment should be initiated as early as possible after diagnosis with 4 loading doses on Days 0, 14, 28 and 63.
Maltese: Il-kura bi Spinraza għandha tinbeda kmieni kemm jista' jkun wara d-dijanjosi b'4 dożi għoljin tal-bidu f'Jiem 0, 14, 28 u 63.
English: The benefits and risks of continuing Spinraza whilst having a "ventriculo-peritoneal shunt" in place are not known at present.
Maltese: Il-benefiċċji u r-riskji li tkompli Spinraza waqt li jkollok “shunt ventrikulo-peritoneali” imwaħħal mhumiex magħrufa bħalissa.
English: These results are supported by an open-label Phase 2 study in symptomatic patients diagnosed with SMA (CS3A).
Maltese: 2 f'pazjenti sintomatiċi ddijanjostikati b'SMA (CS3A).
English: Proportion achieving pre-defined motor milestone responder criteria (HINE section 2)4,5
Maltese: Proporzjon li kisbu kriterji ta' persuna li rrispondiet għal milestone talmoviment definiti minn qabel (HINE sezzjoni 2)4,5
English: At the final analysis, a statistically significant greater percentage of patients achieved the definition of a motor milestone responder in the Spinraza group (51%) compared to the sham-control group (0%) (p<0.0001).
Maltese: Fl-analiżi finali, perċentwali statistikament sinifikanti akbar ta' pazjenti kisbu d-definizzjoni ta' persuna li rrispondiet għal milestone tal-moviment fil-grupp ta' Spinraza (51%) meta mqabbla malgrupp tal-kontroll ikkurati bi proċedura falza (0%) (p<0.0001).
English: An improvement in mean CHOP INTEND score over time was observed from baseline to day 1072 (mean change 21.30).
Maltese: Ġie osservat titjib fil-medja talpunteġġ maż-żmien CHOP INTEND mil-linja bażi sa jum 1072 (bidla medja ta' 21.30).
English: Single- and multiple-dose pharmacokinetics (PK) of nusinersen, administered via intrathecal injection, were determined in paediatric patients diagnosed with SMA.
Maltese: Il-farmakokinetika (PK) ta' dożi waħidhom u multipli farmakokinetiċi ta' nusinersen, mogħtija permezz ta' injezzjoni ġot-teka, ġew stabbiliti f'pazjenti pedjatriċi ddijanjostikati b'SMA.
English: RULM
Maltese: KUNJETT
English: At the final analysis, a statistically significant improvement in HFMSE score from baseline to Month 15 was seen in the Spinraza group compared to the sham-control group (Table 3, Figure 2).
Maltese: Fl-analiżi finali, titjib statistikament sinifikanti fil-punteġġ HFMSE mil-linja bażi għal Xahar 15 intwera fil-grupp Spinraza meta mqabbel mal-grupp tal-kontroll bi plaċebo (Tabella 3, Figura 2).
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.
Maltese: Xi avvenimenti avversi li komunement huma assoċjati ma' titqiba fis-sinsla (eż. uġigħ ras u tad-dahar) ma jistgħux jiġu evalwati fil-popolazzjoni ta' trabi esposti għal Spinraza minħabba komunikazzjoni limitata li hi adattata għal dak il-grupp ta' età.
English: Some patients were implanted with a ventriculo-peritoneal shunt.
Maltese: Xi pazjenti ġew impjantati b'shunt ventrikulo-peritoneali biex jikkura l-idroċefalu.
English: EU/1/17/1188/001
Maltese: TAL-AWTORIZZAZZJONI GĦAT-TQEGĦID FIS-SUQ EU/1/17/1188/001
English: It is unknown whether nusinersen/metabolites are excreted in human milk.
Maltese: Mhux magħruf jekk nusinersen/metaboliti jiġux eliminati mill-ħalib tas-sider tal-bniedem.
English: In Study CS11 all patients received Spinraza, with the length of treatment ranging from 65 to 592 days (median 289 days) at the time of interim analysis.
Maltese: Fi Studju CS11, il-pazjenti kollha rċivew Spinraza, bit-tul tat-trattament li varja minn 65 sa 592 jum (medjan ta' 289 jum) fil-ħin tal-analiżi interim.
English: Study CS4 (CHERISH) was a Phase 3, randomised, double-blind, sham-procedure controlled study conducted in 126 symptomatic patients with later-onset SMA (symptom onset after 6 months of age).
Maltese: Studju CS4 (CHERISH) kien studju kkontrollat ta' Fażi 3, li fih il-parteċipanti ntgħażlu b'mod każwali, double-blind, bi proċedura ta' plaċebo, li twettaq fuq 126 pazjent sintomatiku, bil-bidu ta' SMA li kien aktar tard (bidu tas-sintomi wara li l-pazjenti kellhom 6 xhur).
English: 1CS3B was stopped following positive statistical analysis on the primary endpoint at interim analysis (statistically significantly greater percentage of patients achieved the definition of a motor milestone responder in the Spinraza group (41%) compared to the sham-control group (0%), p<0.0001) 2At the final analysis, event-free survival and overall survival were assessed using the Intent to Treat population (ITT Spinraza n=80; Sham-control n=41).
Maltese: 1CS3B twaqqaf wara analiżi statistikament pożittiva fuq il-punt aħħari primarju fl-analiżi interim (perċentwali statistikament sinifikanti akbar ta' pazjenti li kisbu d-definizzjoni ta' persuna li rrispondiet għal milestone tal-moviment fil-grupp ta' Spinraza (41%) meta mqabbla mal-grupp ta' pazjenti kkurati bi proċedura falza (0%) (p<0.0001).
English: There is a risk of adverse reactions occurring as part of the lumbar puncture procedure (e.g. headache, back pain, vomiting; see section 4.8).
Maltese: Hemm riskju ta' reazzjonijiet avversi li jseħħu bħala parti mill-proċedura tat-titqiba fis-sinsla (eż. uġigħ ta' ras, uġigħ fid-dahar, rimettar, ara sezzjoni 4.8).
English: 4 Assessed using the Month 15 Efficacy Set (Spinraza n=66; Sham control n=34); analyses are based on imputed data when there are missing data.
Maltese: 4L-analiżi saret bl-użu ta' Sett ta' Effikaċja ta' Xahar 15 (Spinraza n=66; grupp tal-kontroll bi plaċebo n=34); l-analiżi huma bbażati fuq dejta imputata f'każ ta' dejta nieqsa
English: Ewopharma AG pārstāvniecība
Maltese: Ewopharma AG Representative Office Tel: + 40
English: 4Assessed at the later of Day 183, Day 302, and Day 394 Study Visit 5According to Hammersmith Infant Neurological Examination (HINE) section 2: ≥2 point increase [or maximal score] in ability to kick, OR ≥1 point increase in the motor milestones of head control, rolling, sitting, crawling, standing or walking, AND improvement in more categories of motor milestones than worsening, defined as a responder for this primary analysis.
Maltese: 5Skont Hammersmith Infant Neurological Examination (HINE) sezzjoni 2: Żieda ta' ≥2 punti [jew fil-punteġġ massimu] filkapaċità li tagħti bis-sieq, JEW żieda ta' ≥1 punt f'milestones tal-moviment tal-kontroll tar-ras, li titgerbeb, li togħod bilqiegħda, li titkaxkar, li toqgħod bilwieqfa u li timxi, U titjib f'aktar kategoriji ta' milestones minn aggravament, definita bħala persuna li rrispondiet għal din l-analiżi primarja.
English: By binding, the ASO displaces splicing factors, which normally suppress splicing.
Maltese: Billi jeħel, l-ASO jisposta l-fatturi tal-isplicing, li normalment irażżnu l-isplicing.
English: Teл.
Maltese: Xhur
English: The usual dose of Spinraza is 12 mg.
Maltese: Id-doża tas-soltu ta' Spinraza hi ta' 12 mg.
English: Of the 20 subjects enrolled, 11 (55%) were alive and free of permanent ventilation at the last visit.
Maltese: Mill-20 individwu li rreġistraw, 11 (55%) kienu ħajjin u mingħajr ventilazzjoni permanenti fl-aħħar vista.
English: Overall, the incidence of ADAs was low, with 15 (4%) patients classified as ADA positive overall, of which 4 had a transient response, 5 had a persistent response, and 6 patients had responses which could not be classified as transient or persistent at the time of data cut off.
Maltese: Globalment, l-inċidenza ta' ADAs kienet baxxa, b'15 (4%) tal-pazjenti li ikklassifikaw bħala pożittivi għal ADAs b'mod globali, li minnhom 4 kellhom rispons temporanju, 5 li kellhom rispons persistenti, u 6 pazjenti kellhom risponsi li ma setgħux jiġu kklassifikati bħala temporanji jew persistenti fil-ħin tal-iskadenza tal-ġbir tad-dejta.
English: Study CS3B (ENDEAR) was a Phase 3, randomized, double-blind, sham-procedure controlled study conducted in 121 symptomatic infants ≤ 7 months of age, diagnosed with SMA (symptom onset before 6 months of age).
Maltese: Studju CS3B (ENDEAR) kien studju kkontrollat ta' Fażi 3, li fih il-parteċipanti ntgħażlu b'mod każwali, double-blind, bi proċedura falza, li twettaq fuq 121 tarbija sintomatika li kellhom ≤ 7 xhur, iddijanjostikati b'SMA (bidu tas-sintomi qabel ma kellhom 6 xhur).
English: Population PK analyses revealed no apparent correlation between hepatic and renal clinical chemistry markers and inter-subject variability.
Maltese: Analiżijiet PK tal-popolazzjoni ma żvelaw l-ebda korrelazzjoni apparenti bejn markaturi ta' kimika klinika tal-fwied u tal-kliewi u varjabilità bejn l-individwi.
English: If clinically indicated, platelet and coagulation laboratory testing is recommended prior to administration of Spinraza.
Maltese: Jekk ikun indikat klinikament, ittestjar fil-laboratorju tal-plejtlits u tal-koagulazzjoni hu rakkomandat qabel l-għoti ta' Spinraza.
English: • Hydrocephalus (a build-up of too much fluid around the brain)
Maltese: • Idroċefalu (jinġabar fluwidu żejjed madwar il-moħħ)
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).
Maltese: Il-proporzjon ta' pazjenti li kienu qed jiżviluppaw SMA klinikament manifestata ġie evalwat fost pazjenti evalwati li laħqu l-viżta ta' Jum 700 fl-analiżi interim (n=16).
English: Study CS5 (NURTURE) is an open-label study in pre-symptomatic infants genetically diagnosed with SMA, who were enrolled at 6 weeks of age or younger.
Maltese: Studju CS5 (NURTURE) huwa studju open-label fi trabi qabel saru sintomatiċi, ġenetikament iddijanjostikati b'SMA, li ġew irreġistrati meta kellhom 6 ġimgħat jew inqas.
English: In repeat-dose toxicity studies (14-weeks and 53-weeks) of intrathecal administration to juvenile cynomolgus monkeys, nusinersen was well tolerated.
Maltese: Fi studji dwar dożi ripetuti (14-il ġimgħa u 53 ġimgħa) tal-għoti minn ġot-teka lil xadini cynomolgus ġuvenili, nusinersen kien ittollerat tajjeb.
English: In the 53-week intrathecal dosing study in cynomolgus monkeys no toxicity effects were seen at levels up to 14-fold the recommended annual clinical maintenance dose.
Maltese: Fl-istudju dwar id-dożaġġ mogħti minn ġot-teka li dam 53 ġimgħa, li twettaq f'xadini cynomolgus, ma ġew osservati l-ebda effetti tossiċi f'livelli sa 14-il darbiet tad-doża ta' manteniment kliniku annwali rakkomandata.
English: METHOD AND ROUTE(S) OF ADMINISTRATION
Maltese: MANIFATTUR(I) GĦALL- RESPONSABBLI
English: Spinraza treated Patients
Maltese: TAL-PRODOTT MEDIĊINALI
English: out of 20 patients (55%) met the endpoint of an increase in total CHOP INTEND score of ≥4 points as of the last study visit.
Maltese: Globalment, 11 minn 20 pazjent (55%), kisbu l-punt aħħari ta' żieda fil-punteġġ totali CHOP INTEND ta' ≥4 punti sal-aħħar viżta tal-istudju tagħhom.
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.
Maltese: Jekk l-ebda refriġerazzjoni ma tkun disponibbli, Spinraza jista' jinħażen fil-kartuna oriġinali tiegħu, protett mid-dawl, f'temperatura sa 30°C ' jew inqas għal sa 14-il jum.
English: Thrombocytopenia and coagulation abnormalities
Maltese: Anormalitajiet fil-koagulazzjoni u tromboċitopenija
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.
Maltese: Titjib fil-funzjoni tal-moviment ġie osservat fost pazjenti li komplew bi Spinraza minn Studju CS3B, kif ukoll f'dawk li bdew Spinraza fi Studju CS11 (Figura 3), bl-akbar benefiċċju li ġie osservat f'dawk li bdew it-trattament aktar kmieni.
English: In vitro studies indicated that nusinersen is not an inducer or inhibitor of CYP450-mediated oxidative metabolism and therefore should not interfere with other medicinal products for these metabolic pathways.
Maltese: Studji in vitro indikaw li nusinersen mhuwiex induttur jew inibitur tal-metaboliżmu ossidattiv medjat minn CYP450, u għalhekk m'għandux jinterferixxi ma' prodotti mediċinali oħrajn għal dawn ilpassaġġi metaboliċi.
English: Proportion achieving a 4-point improvement
Maltese: Parametru ta' Effikaċja
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.
Maltese: SMA hu spectrum kliniku ta' mard, bis-severità tal-marda marbuta ma' numri aktar baxxi ta' kopji tal-ġeni SMN2 u età iżgħar meta jibdew is-sintomi.
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.
Maltese: Studju kliniku addizzjonali, CS7 (EMBRACE) infetaħ għal pazjenti li ma kinux eliġibbli biex jipparteċipaw fi Studju CS3B jew Studju CS4 minħabba l-età waqt l-iscreening jew numru ta' kopji ta' SMN2.
English: Patients achieved milestones unexpected in Type I or II SMA and more consistent with normal development.
Maltese: Il-pazjenti laħqu milestones mhux mistennija f'SMA ta' Tip I jew II u aktar konsistenti ma' żvilupp normali.
English: Post-authorisation efficacy study (PAES): In order to evaluate the long term efficacy and safety of nusinersen in symptomatic patients with spinal muscular atrophy, the MAH should conduct and submit the results of the Phase 3, open-label extension study (SHINE, CS11)
Maltese: B'mod li tkun evalwata leffikaċja u s-sigurtà fit-tul ta' nusinersen f'pazjenti sintomatiċi f'atrofija muskolari tas-sinsla, il-MAH għandha twettaq u tissottometti r-riżultati ta' studju open-label ta' estensjoni ta' Fażi 3. (SHINE, CS11)
English: Initiation of treatment sooner after symptom onset resulted in earlier and greater improvement in motor function than those with delayed treatment initiation; however, both groups experienced benefit compared to sham-control.
Maltese: Il-bidu tal-kura aktar qabel wara l-bidu tas-sintomi, irriżulta f'titjib akbar u aktar kmieni fil-funzjoni tal-moviment minn dawk b'bidu tal-kura li ġie ttardjat; madankollu, iż-żewġ gruppi kisbu benefiċċju meta mqabbla mal-pazjenti fil-grupp tal-kontroll ikkurati bi plaċebo.
English: • Hypersensitivity (an allergic or allergic-like reaction that may include swelling of your face, lips or tongue, rash, or itching)
Maltese: • Sensittività eċċessiva (reazzjoni allerġika jew bħal dawk tal-allerġija li tista' tinkludi nefħa talwiċċ, xufftejn jew ilsien, raxx, jew ħakk)
English: The MAH shall perform the required pharmacovigilance activities and interventions detailed in the agreed RMP presented in Module 1.8.2 of the marketing authorisation and any agreed subsequent updates of the RMP.
Maltese: L-MAH għandu jwettaq l-attivitajiet u l-interventi meħtieġa ta' farmakoviġilanza dettaljati flRMP maqbul ippreżentat fil-Modulu 1.8.2 tal-awtorizzazzjoni għat-tqegħid fis-suq u kwalunkwe aġġornament sussegwenti maqbul tal-RMP.
English: Biogen Idec Limited Tel: +44 (0) 1628 50 1000
Maltese: United Kingdom Biogen Idec Limited Tel: +44