FrancophonIA/ELRC3.0 huggingface dataset:
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.
Slovenian: Čas do smrti ali trajne ventilacije (≥ 16 ur ventilacije/dan neprekinjeno za > 21 dni v odsotnosti akutnega reverzibilnega dogodka ali traheostomije) je bil določen za primarni končni opazovani dogodek.
English: Animal studies do not indicate direct or indirect harmful effects with respect to reproductive toxicity (see section 5.3).
Slovenian: Študije na živalih ne kažejo neposrednih ali posrednih škodljivih učinkov v povezavi z reproduktivno toksičnostjo (glejte poglavje
English: At the time of a Study CS11 interim analysis, 61 out of 65 patients (94%) were alive.
Slovenian: V času vmesne analize študije CS11 je bilo 61 od 65 bolnikov (94 %) živih.
English: Spinraza is not metabolised via the cytochrome P450 enzyme system in the liver, therefore dose adjustment is unlikely to be required in patients with hepatic impairment (see sections 4.5 and 5.2).
Slovenian: Zdravilo Spinraza se ne presnavlja preko encimskega sistema citokroma P450, zato ni verjetno, da bo pri bolnikih z okvaro jeter potrebno prilagajanje odmerka (glejte poglavji 4.5 in 5.2).
English: Pre-symptomatic infants
Slovenian: Predsimptomatični dojenčki
English: There is no accumulation in plasma exposure measures (Cmax and AUC) after multiple doses.
Slovenian: Kazalca izpostavljenosti plazme (Cmax in AUC) se po večkratnih odmerkih ne nakopičita.
English: The remaining 2 events (rash and eosinophil count increased, both nonserious) were possibly related to nusinersen.
Slovenian: Preostala dva neželena učinka (izpuščaj in povečanje števila eozinofilcev, oba opredeljena kot neresna) sta bila morda povezana z nusinersenom.
English: Following intrathecal administration trough plasma concentrations of nusinersen were relatively low compared to the trough CSF concentration.
Slovenian: Po intratekalnem dajanju so bile najnižje plazemske koncentracije nusinersena relativno nizke v primerjavi z najnižjo koncentracijo v CSF.
English: A decision must be made whether to discontinue breast-feeding or to discontinue/abstain from Spinraza therapy taking into account the benefit of breast-feeding for the child and the benefit of therapy for the woman.
Slovenian: Odločiti se je treba med prenehanjem dojenja in prenehanjem/prekinitvijo zdravljenja z zdravilom Spinraza, pri čemer je treba pretehtati prednosti dojenja za otroka in prednosti zdravljenja za mater.
English: Of the 346 patients who received Spinraza up to a maximum of 5 years, 258 patients received treatment for at least 1 year.
Slovenian: Spinraza do največ 5 let, je 258 bolnikov prejemalo zdravljenje vsaj 1 leto.
English: What Spinraza is and what it is used for 2.
Slovenian: Kaj je zdravilo Spinraza in za kaj ga uporabljamo 2.
English: Spinraza works by helping the body to produce more of the SMN protein that people with SMA are lacking.
Slovenian: Zdravilo Spinraza deluje tako, da pomaga telesu izdelovati več beljakovine SMN, ki je ljudem s SMA primanjkuje.
English: Clear and colourless solution with pH of approximately 7.2.
Slovenian: raztopina za injiciranje Bistra in brezbarvna raztopina s pH približno 7,2.
English: Mean plasma Cmax and AUC values increased approximately dose proportionally over the evaluated dose range.
Slovenian: Srednje vrednosti plazemske Cmax in AUC so v ocenjenem razponu odmerkov naraščale približno proporcionalno odmerku.
English: 5 ml in a Type I glass vial with bromobutyl rubber stopper and an aluminium over-seal and plastic cap.
Slovenian: 5 ml v viali iz stekla tipa I z zamaškom iz bromobutilne gume in aluminijasto prekrivno zaporko in plastično zaporko.
English: In repeat-dose toxicity studies (14-weeks and 53-weeks) of intrathecal administration to juvenile cynomolgus monkeys, nusinersen was well tolerated.
Slovenian: V študijah toksičnosti s ponavljajočimi se odmerki (14-tedenskih in 53-tedenskih) intratekalnega dajanja nusinersena juvenilnim opicam Cynomolgus so živali nusinersen dobro prenašale.
English: Submission of study results:
Slovenian: Predložitev rezultatov študije: april
English: Twelve out of 20 patients (60%) in the study met the primary endpoint with improvement in mean motor milestone achievement over time.
Slovenian: V študiji je 12 od 20 bolnikov (60 %) doseglo primarni končni opazovani dogodek s postopnim izboljševanjem povprečnih motoričnih mejnikov skozi čas.
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.
Slovenian: Če hlajenje ni na voljo, lahko zdravilo Spinraza shranjujete v originalni kartonski škatli, zaščiteno pred svetlobo, pri 30 °C ali nižji temperaturi do 14 dni.
English: The injection must not be administered in areas of the skin where there are signs of infection or inflammation.
Slovenian: Injekcije se ne sme dati v predelih kože, kjer so vidni znaki okužbe ali vnetja.
English: Pack size of one vial per carton.
Slovenian: Velikost pakiranja je ena viala na kartonsko škatlo.
English: The length of treatment ranged from 74 to 474 days (median 250 days) at the time of the interim analysis.
Slovenian: V času vmesne analize je zdravljenje trajalo od 74 do 474 dni (mediana 250 dni).
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.
Slovenian: V študiji CS11 so vsi bolniki prejemali zdravilo Spinraza, v času vmesne analize pa je zdravljenje trajalo od 65 do 592 dni (mediana 289 dni).
English: Upon completion of Study CS4 (CHERISH) 125 patients enrolled in Study CS11 (SHINE), where all patients received Spinraza.
Slovenian: Po zaključku študije CS4 (CHERISH) je bilo 125 bolnikov vključenih v študijo CS11 (SHINE), kjer so vsi prejemali zdravilo Spinraza.
English: Spinraza is given by injection into the lower back.
Slovenian: Zdravilo Spinraza se daje z injekcijo v spodnji del hrbta.
English: *Adverse events considered related to the lumbar puncture procedure.
Slovenian: *Neželeni učinki, za katere menijo, da so povezani s postopkom lumbalne punkcije.
English: Number of patients who died or received permanent ventilation Hazard ratio (95% CI) p-value6
Slovenian: Število bolnikov, ki so umrli ali prejeli trajno ventilacijo Razmerje ogroženosti (95-% IZ) Vrednost p6
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.
Slovenian: Vsi bolniki so bili ob zadnji oceni zmožni sesati in požirati, od teh je 22 (88 %) dojenčkov doseglo maksimalni rezultat v skladu s HINE, poglavje 1.
English: In vitro studies indicate that the likelihood for interactions with nusinersen due to competition for plasma protein binding, or competition with or inhibition of transporters is low.
Slovenian: Študije in vitro kažejo, da je medsebojno delovanje z nusinersenom malo verjetno zaradi kompeticije za vezavo na plazemske beljakovine ali zaradi kompeticije s prenašalci ali inhibicije prenašalcev.
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.
Slovenian: Uvedba zdravljenja kmalu po nastopu simptomov je posledično vodila v hitrejše in večje izboljšanje motorične funkcije v primerjavi s pozno uvedbo zdravljenja; vendar sta imeli obe skupini korist od zdravljenja v primerjavi s kontrolnimi bolniki na placebu.
English: Treatment should be administered by health care professionals experienced in performing lumbar punctures.
Slovenian: Zdravljenje smejo izvajati zdravstveni delavci, ki imajo izkušnje z opravljanjem lumbalnih punkcij.
English: Reproductive toxicology studies were conducted using subcutaneous administration of nusinersen in mice and rabbits.
Slovenian: Toksikološke študije vpliva na razmnoževanje so izvedli s subkutanim dajanjem nusinersena mišim in kuncem.
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.
Slovenian: SMA je klinični spekter bolezni, kjer je resnost bolezni povezana z manjšim številom genskih kopij SMN2 in nastopom simptomov pri nižji starosti.
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.
Slovenian: Nekaterih neželenih dogodkov, ki so pogosto povezani z lumbalno punkcijo (npr. glavobol in bolečina v hrbtu), v populaciji dojenčkov, izpostavljenih zdravilu Spinraza, niso mogli oceniti zaradi omejene komunikacije, značilne za to starostno skupino.
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.
Slovenian: V izhodišču so imeli bolniki povprečen rezultat razširjene Hammersmithove lestvice gibalnih sposobnosti (Hammersmith Functional Motor Scale Expanded - HFMSE) 21,6, povprečen revidirani test modula zgornjih udov (revised upper limb module - RULM) 19,1, vsi so dosegli neodvisno sedenje in noben bolnik ni dosegel neodvisne hoje.
English: The majority of these side effects are reported within 72 hours of the procedure.
Slovenian: O večini teh neželenih učinkov poročajo v 72 urah po postopku.
English: 1171 LP Badhoevedorp The Netherlands
Slovenian: Prins Mauritslaan 13 1171 LP Badhoevedorp
English: No serious complications of lumbar puncture, such as serious infections, have been observed in the clinical trials of Spinraza.
Slovenian: Resnih zapletov pri lumbalni punkciji, na primer resnih okužb, v kliničnih preskušanjih zdravila Spinraza niso opazili.
English: The benefits and risks of continuing Spinraza whilst having a "ventriculo-peritoneal shunt" in place are not known at present.
Slovenian: Koristi in tveganja nadaljevanja zdravljenja z zdravilom Spinraza, tačas ko ima bolnik nameščen “ventrikulo-peritonealni spoj”, trenutno niso znana.
English: This injection, called a lumbar puncture, is done by inserting a needle into the space around the spinal cord.
Slovenian: To injekcijo, ki se imenuje lumbalna punkcija, damo tako, da zabodemo iglo v prostor okrog hrbtenjače.
English: Among patients treated with Spinraza by lumbar puncture, serious infection, such as meningitis, has been observed.
Slovenian: Med bolniki, zdravljenimi z zdravilom Spinraza z lumbalno punkcijo, so opazili resne okužbe, kot je meningitis.
English: Each carton of Spinraza contains one vial.
Slovenian: Ena kartonska škatla zdravila Spinraza vsebuje eno vialo.
English: Don't stop treatment with Spinraza unless your doctor tells you to.
Slovenian: Ne prenehajte se zdraviti z zdravilom Spinraza, če vam tega ne naroči zdravnik.
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.
Slovenian: Intratekalno injicirani nusinersen v CSF je popolnoma na voljo za porazdelitev iz CSF v tarčna tkiva centralnega živčnega sistema (CŽS).
English: Communicating hydrocephalus, aseptic meningitis and hypersensitivity (e.g. angioedema, urticaria and rash) have also been reported.
Slovenian: Poročali so tudi o komunikantnem hidrocefalusu, aseptičnem meningitisu in preobčutljivosti (npr. angioedem, urtikarija in izpuščaji).
English: Proportion of patients who achieved at least a 3 point improvement from baseline to month 152
Slovenian: Delež bolnikov, ki so dosegli izboljšanje od izhodišča za vsaj 3 točke do 15. meseca2
English: Potential difficulties with this route of administration may be seen in very young patients and those with scoliosis.
Slovenian: Možne težave pri tej poti uporabe zdravila se lahko pojavljajo pri zelo mladih bolnikih in pri tistih s skoliozo.
English: Your doctor will tell you how long you or your child need to receive Spinraza.
Slovenian: Zdravnik vam bo povedal, kako dolgo morate ali mora vaš otrok prejemati zdravilo Spinraza.
English: Before you or your child are given Spinraza your doctor may decide to do a blood test to check that your or your child's blood can clot properly.
Slovenian: Preden bodo vam ali vašemu otroku dali zdravilo Spinraza, se lahko zdravnik odloči, da bo naredil krvno preiskavo, s katero bo preveril, ali se vaša kri ali kri vašega otroka pravilno strjuje.
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.
Slovenian: Obstaja majhno število poročil o bolnikih, pri katerih se je po dajanju zdravila Spinraza razvil hidrocefalus (kopičenje prevelike količine tekočine okrog možganov).
English: 1 out of 28 patients had 2 SMN2 gene copies, 21 had 3 copies, and 6 had 4 copies.
Slovenian: Eden od 28 bolnikov je imel 2 kopiji gena za SMN2, 21 jih je imelo po 3 kopije, 6 pa po 4 kopije.
English: No impact on male or female fertility, or embryo-foetal development, or pre/post-natal development was observed.
Slovenian: Vpliva na plodnost samcev ali samic, na razvoj zarodka in ploda ali na pred/poporodni razvoj niso opazili.
English: Spinraza contains the active substance nusinersen which belongs to a group of medicines known as antisense oligonucleotides.
Slovenian: Zdravilo Spinraza vsebuje učinkovino nusinersen, ki pripada skupini zdravil, ki so znana kot protismiselni oligonukleotidi.
English: Spinraza is indicated for the treatment of 5q Spinal Muscular Atrophy.
Slovenian: Zdravilo Spinraza je indicirano za zdravljenje 5q spinalne mišične atrofije.
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.
Slovenian: Od teh bolnikov jih je 6 (33 %) v skupini z zdravilom Spinraza in 0 (0 %) v kontrolni skupini na placebu izpolnjevalo s protokolom opredeljena merila za bolnika, odzivnega z motoričnimi mejniki.
English: Just prior to administration, remove the plastic cap and insert the syringe needle into the vial through the centre of the over-seal to remove the appropriate volume.
Slovenian: Tik pred uporabo odstranite plastično zaporko in zabodite iglo brizge v vialo skozi sredino prekrivne zaporke, da boste odstranili ustrezni volumen.
English: Population PK analyses revealed no apparent correlation between hepatic and renal clinical chemistry markers and inter-subject variability.
Slovenian: Populacijske farmakokinetične analize niso razkrile očitne korelacije med označevalci klinične kemije jeter in ledvic in variabilnostjo med preiskovanci.
English: How long to use Spinraza
Slovenian: Kako dolgo se zdravilo Spinraza uporablja
English: Study CS11 is an open label extension study for SMA patients who previously participated in the other Spinraza clinical studies.
Slovenian: Pri študiji CS11 gre za odprto podaljšano študijo za bolnike s SMA, ki so prej sodelovali v drugih kliničnih študijah z zdravilom Spinraza.
English: A majority of Spinraza treated patients experienced stabilization or improvement in motor function, with the greatest benefit observed in those with earlier treatment initiation.
Slovenian: Pri večini bolnikov, zdravljenih z zdravilom Spinraza, so opazili stabilizacijo ali izboljšanje motorične funkcije, največjo korist pa so opazili pri bolnikih, pri katerih je bilo zdravljenje uvedeno zgodaj.
English: Biogen (Denmark) Manufacturing ApS Biogen Allé 1
Slovenian: Izdelovalec Biogen (Denmark) Manufacturing ApS
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.
Slovenian: Nusinersen se presnavlja počasi, in sicer pretežno s hidrolizo, ki jo posreduje eksonukleaza (3'- in 5'-), in ni substrat, inhibitor ali induktor encimov CYP450.
English: The immunogenic response to nusinersen was determined in 346 patients with baseline and post-baseline plasma samples evaluated for anti-drug antibodies (ADA).
Slovenian: Imunogeni odziv na nusinersen so ugotavljali pri 346 bolnikih z izhodiščnimi in poizhodiščnimi vzorci plazme, v katerih so izmerili protitelesa proti zdravilu (anti-drug antibodies - ADA).
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).
Slovenian: Delež bolnikov, pri katerih se je razvila klinično izražena SMA, so ocenili pri bolnikih, ki so v času vmesne analize imeli pregled na 700. dan (n = 16).
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).
Slovenian: Nusinersen je protismiselni oligonukleotid (antisense oligonucleotide - ASO), ki zvečuje delež vključitve eksona 7 v kopije informacijske ribonukleinske kisline (messenger RNA - mRNA) za beljakovino preživetja motoričnega nevrona 2 (survival motor neuron 2 - SMN2), tako da se veže na mesto utišanja izrezovanja intronov (intronic splice silencing site - ISS-N1), ki ga najdemo v intronu 7 predinformacijske ribonukleinske kisline (pre-mRNA) SMN2.
English: Other medicines and Spinraza
Slovenian: Druga zdravila in zdravilo Spinraza
English: Survival Event-free survival2
Slovenian: Preživetje Preživetje brez dogodkov2
English: If removed from the original carton, the total combined time out of refrigeration should not exceed 30 hours, at a temperature that does not exceed 25°C.
Slovenian: Če ste jih vzeli iz originalne kartonske škatle, celotni skupen čas brez hlajenja ne sme preseči 30 ur pri temperaturi, ki ni višja od 25 °C.
English: Spinraza is a clear colourless solution for injection.
Slovenian: Zdravilo Spinraza je bistra, brezbarvna raztopina za injiciranje.
English: • Hypersensitivity (an allergic or allergic-like reaction that may include swelling of your face, lips or tongue, rash, or itching)
Slovenian: • preobčutljivost (alergijska ali alergiji podobna reakcija, ki lahko vključuje otekanje obraza,
English: Spinal muscular atrophy is caused by a shortage of a protein called survival motor neuron (SMN) in the body.
Slovenian: Spinalno mišično atrofijo povzroča pomanjkanje beljakovine, ki se imenuje beljakovina preživetja motoričnega nevrona (survival motor neuron - SMN) v telesu.
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).
Slovenian: Izhodiščne značilnosti bolezni so bile na splošno podobne, z izjemo neravnovesja v deležu bolnikov, ki so sploh kdaj dosegli sposobnost stati brez opore (13 % bolnikov v skupini z zdravilom Spinraza in 29 % kontrolnih bolnikov na placebu) ali hoditi z oporo (24 % bolnikov v skupini z zdravilom Spinraza in 33 % kontrolnih bolnikov na placebu).
English: Figure 3: Change in HINE Motor Milestones versus Study days for Study CS3B (treated and sham-control), CS3A, CS5 and CS11
Slovenian: Slika 3: Sprememba motoričnih mejnikov HINE v primerjavi s številom dni trajanja študije za študijo CS3B (zdravljeni in kontrolni na placebu), CS3A, CS5 in CS11
English: Once drawn into the syringe, if the solution is not used within 6 hours, it must be discarded.
Slovenian: Če raztopine, ki ste jo vsrkali v brizgo, ne porabite v 6 urah, jo morate zavreči.
English: Store in the original carton in order to protect from light.
Slovenian: Shranjujte v originalni kartonski škatli za zagotovitev zaščite pred svetlobo.
English: Read all of this leaflet carefully before you or your child receives this medicine because it contains important information for you.
Slovenian: Preden prejmete ali preden vaš otrok prejme to zdravilo, natančno preberite navodilo, ker vsebuje za vas pomembne podatke!
English: Aseptic technique should be used when preparing and administering Spinraza; see instructions for use in section 6.6.
Slovenian: Spinraza se mora uporabljati aseptično tehniko; glejte navodila za uporabo v poglavju 6.6. 4.3 Kontraindikacije
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.
Slovenian: Dodatna klinična študija CS7 (EMBRACE) je bila odprta za bolnike, ki niso izpolnjevali meril za sodelovanje v študiji CS3B ali študiji CS4 zaradi starosti ob presejanju ali števila kopij gena za SMN2.
English: There are no or limited amount of data from the use of nusinersen in pregnant women.
Slovenian: Podatkov o uporabi nusinersena pri nosečnicah ni oziroma so omejeni.
English: As of 19 October 2018, 100 AEs of hypersensitivity due to the administration of nusinersen were identified from the clinical trial database.
Slovenian: 19. oktobra 2018 je bilo iz zbirke podatkov kliničnih preskušanj ugotovljenih 100 neželenih učinkov preobčutljivosti zaradi uporabe nusinersena.
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.
Slovenian: Izboljšanje motorične funkcije so opazili pri bolnikih, ki so nadaljevali zdravljenje z zdravilom Spinraza iz študije CS3B, in pri tistih, pri katerih je bilo zdravljenje z zdravilom Spinraza uvedeno v študiji CS11 (slika 3), največjo korist pa so opazili pri bolnikih, pri katerih je bilo zdravljenje uvedeno zgodaj.
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.
Slovenian: Trenutno so koristi in tveganja zdravljenja z nusinersenom pri bolnikih z ventrikulo peritonealnim spojem neznana in nadaljevanje zdravljenja je treba skrbno pretehtati.
English: Figure 2: Mean change from baseline in HFMSE score over time at final analysis (ITT)Study CS4 1,2
Slovenian: Slika 2: Povprečna sprememba rezultata HFMSE od izhodišča v času pri končni analizi (ITT)študija CS4 1,2
English: The safety assessment of Spinraza was based on two Phase 3 clinical studies in infants (CS3B) and children (CS4) with SMA, together with one Phase 2 study in infants and children with SMA (CS7) and open-label studies including pre-symptomatic infants (CS5) genetically diagnosed with SMA and infants and children with SMA.
Slovenian: Ocena varnosti zdravila Spinraza temelji na dveh kliničnih študijah 3. faze pri dojenčkih (CS3B) in otrocih (CS4) s SMA, skupaj z eno študijo 2. faze, v katero so bili vključeni dojenčki in otroci s SMA (CS7), in odprtimi študijami, ki so vključevale predsimptomatične dojenčke (CS5), ki so jim genetsko postavili diagnozo SMA, in dojenčke in otroke s SMA.
English: Some patients were implanted with a ventriculo-peritoneal shunt.
Slovenian: Nekaterim bolnikom so vstavili ventrikulo-peritonealni spoj.
English: Ninety-eight of these were considered not or unlikely related to nusinersen.
Slovenian: 98 izmed teh ni povezanih oziroma je povezanost z uporabo nusinersena malo verjetna.
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.
Slovenian: Študija CS5 (NURTURE) je odprta študija pri predsimptomatičnih dojenčkih z genetsko diagnozo SMA, ki so jih vključili pri 6 tednih starosti ali še prej.