FrancophonIA/ELRC3.0 huggingface dataset:
English: Myozyme should not be used during pregnancy unless clearly necessary.
Slovenian: Med nosečnostjo zdravila Myozyme ni dovoljeno uporabljati, razen če je to nujno potrebno.
English: Such a reaction comprises different symptoms like low blood pressure, chest discomfort, throat tightness, face, lips or tongue swelling (angioedema), hives (urticaria), dizziness, rash, itchy skin, nausea, vomiting, cough and bronchospasm (see section 4 for an overview of all infusion-associated reactions).
Slovenian: Takšne reakcije vključujejo različne simptome, kot so nizek krvni tlak, občutek nelagodja v prsih, stiskanje v vratu, zatekanje obraza, ustnic ali jezika (angioedem), koprivnica (urtikarija) , omotica, izpuščaji, srbeča koža, slabost, bruhanje, kašelj in bronhospazem (glejte poglavje 4 za pregled vseh infuzijskih reakcij).
English: Reactions in 2 of these patients were IgE-mediated hypersensitivity reactions.
Slovenian: Reakcije pri 2 izmed navedenih bolnikov so bile IgE posredovane preobčutljivostne reakcije.
English: At 104 weeks of treatment, all 13 patients with available data had maintained or improved weight-for-age percentiles (above the 3rd percentile), all 12 patients with available data were above the 3rd percentile for length and all 12 patients with available data were above the 3rd percentile for head circumference.
Slovenian: Pri 104 tednu zdravljenja je vseh 13 bolnikov, za katere so na voljo podatki, ohranilo ali izboljšalo odstotek teže glede na starost (nad 3 odstotke), vseh 12 bolnikov, za katere so na voljo podatki, je bilo nad 3 odstotke glede na višino ter vseh 12 bolnikov, za katere so na voljo podatki, je bilo nad 3 odstotke glede na obseg glave.
English: Adverse reactions reported in at least 2 patients are listed in Table 1 by System Organ Class.
Slovenian: Neželeni učinki, o katerih so poročali pri najmanj 2 bolnikih, so navedeni v tabeli 1 po organskih sistemih.
English: This medicine contains less than 1 mmol sodium (23 mg) per vial, i.e. essentially ‘sodium free'.
Slovenian: Zdravilo Myozyme vsebuje natrij To zdravilo vsebuje manj kot 1 mmol natrija (23 mg) na vialo, kar v bistvu pomeni „brez natrija“.
English: Pharmacotherapeutic group: Other alimentary tract and metabolism products, enzymes.
Slovenian: Farmakoterapevtska skupina: Druga zdravila za bolezni prebavil in presnove - encimi.
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).
Slovenian: V klinični študiji, kontrolirani s placebom, ki je trajala 78 tednov, so 90 bolnikov s poznim nastopom Pompejeve bolezni, v starosti od 10 do 70 let, zdravili z zdravilom Myozyme ali placebom; naključna razporeditev je bila v razmerju 2:1 (glejte poglavje 5.1).
English: Patients should be closely monitored and all cases of IARs, delayed reactions and possible immunological reactions should be reported to the marketing authorisation holder.
Slovenian: Bolnike je treba skrbno opazovati in poročati imetniku dovoljenja za promet z zdravilom o vseh primerih infuzijskih reakcij, zapoznelih reakcij in morebitnih imunoloških reakcij.
English: After 104 weeks of treatment, all 16 patients who enrolled in the extension study were alive and 10 of these 16 patients were free of invasive ventilatory support.
Slovenian: Po 104. tednu zdravljenja je bilo vseh 16 bolnikov, ki so se vključili v podaljšek študije živih, in 10 izmed navedenih 16 bolnikov ni potrebovalo podpore invazivnega predihavanja.
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.
Slovenian: Priporočljiva začetna hitrost infundiranja je 1 mg/kg/h in jo postopno povečujemo za 2 mg/kg/h vsakih 30 minut, če ni prisotnih znakov infuzijskih reakcij, dokler ni dosežena najvišja stopnja 7 mg/kg/h.
English: An open-label clinical trial assessed the safety and efficacy of Myozyme in 5 patients with late-onset Pompe disease who ranged in age from 5 to 15 years at initiation of treatment (AGLU02804).
Slovenian: Odprta klinična raziskava je ocenjevala varnost in učinkovitost zdravila Myozyme pri 5 bolnikih s poznim nastopom Pompejeve bolezni, ob začetku zdravljenja pa so bili stari od 5 do 15 let (AGLU02804).
English: Myozyme treatment should be supervised by a physician experienced in the management of patients with Pompe disease or other inherited metabolic or neuromuscular diseases.
Slovenian: Zdravljenje z zdravilom Myozyme mora potekati pod nadzorom zdravnika, ki ima izkušnje pri zdravljenju bolnikov s Pompejevo boleznijo ali drugimi dednimi presnovnimi ali živčnomišičnimi boleznimi.
English: Patients received 20 mg/kg Myozyme once every two weeks for 52 weeks except for 8 patients who received 40 mg/kg after at least 26 weeks of treatment.
Slovenian: Bolniki so prejemali 20 mg/kg zdravila Myozyme enkrat na vsaka dva tedna v obdobju 52 tednov, razen 8 bolnikov, ki so prejemali 40 mg/kg po najmanj 26 tednih zdravljenja.
English: Life threatening reactions, including very severe generalised allergic reactions and anaphylactic shock, have been reported in some patients.
Slovenian: Pri nekaterih bolnikih so poročali o življenjsko nevarnih reakcijah, vključno z zelo hudimi generaliziranimi alergijskimi reakcijami in anafilaktičnim šokom.
English: The time course of disease progression is extremely variable and not predictable, with some patients experiencing a rapid deterioration in skeletal and respiratory muscle function leading to loss of ambulation and respiratory failure, others progressing less rapidly, and yet others presenting with a dissociation in the progression of skeletal and respiratory muscle involvement.
Slovenian: Časovni potek bolezni je zelo spremenljiv in ga ni mogoče napovedati, saj pri nekaterih bolnikih pride do hitrega poslabšanja v delovanju skeletnih in dihalnih mišic, kar povzroči izgubo sposobnosti gibanja in dihalno odpoved, pri drugih bolezen napreduje počasneje, spet pri drugih pa se pojavi disociacija v napredovanju prizadetosti skeletnih in dihalnih mišic.
English: The powder is white to off-white.
Slovenian: Prašek je bel ali skoraj bel.
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).
Slovenian: Preostalih 7 bolnikov ni klinično značilno motorično napredovalo oz. ni bilo zmožno ohraniti motoričnega napredovanja in je imelo zelo omejeno motorično gibanje do zadnje ocene študije (s trajanjem zdravljenja pri posameznih bolnikih v razponu od 52 do 142 tednov; povprečno obdobje sledenja 103 tedne).
English: There are no data from the use of alglucosidase alfa in pregnant women.
Slovenian: Ni zadostnih podatkov o uporabi alglukozidaze alfa pri nosečnicah.
English: Patients with advanced Pompe disease may have compromised cardiac and respiratory function, which may predispose them to a higher risk of severe complications from infusion associated reactions.
Slovenian: Pri bolnikih z napredujočo obliko Pompejeve bolezni je lahko ogrožena srčna in dihalna funkcija, zaradi česar so lahko nagnjeni k povišanemu tveganju za hujše zaplete zaradi reakcij, povezanih z infuzijo.
English: This should then be further diluted as follows: Slowly withdraw the reconstituted solution from each vial until the volume for the patient's dose is obtained using a syringe with a needle diameter not larger than 20 gauge.
Slovenian: Nato je potrebno nadaljnje redčenje, kot je opisano v nadaljevanju: Počasi odvzemajte rekonstituirano raztopino iz vsake viale, dokler ne dosežete količine za bolnikov odmerek injekcije z uporabo injekcijske brizge z iglo premera največ 20 G (gauge).
English: Immunomodulation
Slovenian: Imunomodulacija
English: A. MANUFACTURERS OF THE BIOLOGICAL ACTIVE SUBSTANCE AND MANUFACTURERS RESPONSIBLE FOR BATCH RELEASE
Slovenian: A. PROIZVAJALCI BIOLOŠKE UČINKOVINE IN PROIZVAJALCI, ODGOVORNI ZA
English: There are no clinical data on the effects of alglucosidase alfa on fertility.
Slovenian: Ni kliničnih podatkov o učinkih alglukozidaze alfa na plodnost.
English: Reconstitute each 50 mg vial of Myozyme with 10.3 ml water for injections.
Slovenian: Vsako 50 mg vialo zdravila Myozyme rekonstituirajte z 10,3 ml vode za injekcije.
English: These proportions were maintained up to the end of the study (with individual patient treatment durations ranging from 1 to 168 weeks; mean follow-up period of 109 weeks).
Slovenian: Navedeni odstotki so se ohranili do konca študije (s trajanjem zdravljenja pri posameznih bolnikih v razponu 1 do 168 tednov; povprečno obdobje sledenja 109 tednov).
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).
Slovenian: Preživetje pri zdravljenih bolnikih je bilo primerjano s preživetjem v podobni historični kohorti nezdravljenih oseb po Coxovi regresijski analizi sorazmernih tveganj (glejte Tabelo 3).
English: After the first and sixth infusion of Myozyme, mean maximum plasma concentrations (Cmax) ranged from 178.2 to 263.7 µg/ml for the 20 mg/kg and 40 mg/kg dose groups respectively.
Slovenian: Po prvi in šesti infuziji zdravila Myozyme je bila srednja vrednost maksimalne koncentracije v plazmi (Cmax) v razponu od 178,2 do 263,7 g/ml za skupini, ki sta prejemali odmerke 20 mg/kg oziroma 40 mg/kg.
English: Based on these results the 20 mg/kg qow dose is recommended.
Slovenian: Glede na te rezultate je priporočen odmerek 20 mg/kg.
English: A small number of patients (<1%) in clinical trials and in the commercial setting developed anaphylactic shock and/or cardiac arrest during Myozyme infusion that required life-support measures.
Slovenian: V kliničnih preskušanjih in ob komercialni uporabi je pri majhnem številu bolnikov (< 1 %) prišlo do anafilaktičnega šoka in/ali zaustavitve delovanja srca med infundiranjem zdravila Myozyme, kar je zahtevalo izvajanje postopkov oživljanja.
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).
Slovenian: V kliničnih preskušanjih so 39 bolnikov z zgodnjim nastopom bolezni zdravili z zdravilom Myozyme več kot tri leta (168 tednov z mediano 121 tednov; glejte poglavje 5.1).
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.
Slovenian: Po 52 tednih zdravljenja je 14 izmed 18 bolnikov (77,8 %) ohranilo ali izboljšalo odstotek telesne mase glede na starost (nad 3 odstotke), 14 izmed 15 bolnikov (93,3 %) je bilo nad 3 odstotke glede na višino ter 12 izmed 15 bolnikov (80,0 %) je bilo nad 3 odstotke glede na obseg glave.
English: Change from Baseline to Week 78/Last Observation*
Slovenian: 78. teden / zadnje opazovanje povprečna ± s.d. povprečje sprememba od izhodišča do 78. tedna / zadnje opazovanje*
English: 2 Reactions reported in 60 late-onset patients in a placebo-controlled clinical trial.
Slovenian: 2 O učinkih so poročali pri 60 bolnikih s poznim nastopom bolezni v kliničnem preskušanju, kontroliranem s placebom.
English: Infantile onset patients who develop high IgG antibody titres appear to be at higher risk for developing more frequent IARs.
Slovenian: Bolniki z zgodnjim nastopom bolezni, pri katerih se razvijejo visoki titri protiteles IgG, so izpostavljeni večjemu tveganju za razvoj pogostejših infuzijskih reakcij.
English: The recommended final concentration of alglucosidase in the infusion bags ranges from 0.5 mg/ml to 4 mg/ml.
Slovenian: Priporočljiva končna koncentracija alglukozidaze v infuzijskih vrečkah se giblje od 0,5 mg/ml do 4 mg/ml.
English: N=18 N=42
Slovenian: n=42
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.
Slovenian: Preostalih 5 bolnikov ni pokazalo nobenih bistvenih sprememb glede motoričnega razvoja po 52. tednu (s trajanjem zdravljenja pri posameznih bolnikih v razponu od 104 do 168 tednov; povprečno obdobje sledenja 140 tednov), vključno s 4 bolniki, ki niso imeli nobenih bistvenih motoričnih sposobnosti v nobenem od ocenjevanih stanj in 1 bolnikom, ki je imel le funkcionalno sposobnost sedenja do zadnjega obiska v študiji.
English: Hypersensitivity/Anaphylactic reactions
Slovenian: Preobčutljivostne/anafilaktične reakcije
English: Keep this medicine out of the sight and reach of children Do not use this medicine after the expiry date which is stated on the label after ‘EXP'.
Slovenian: Tega zdravila ne smete uporabljati po datumu izteka roka uporabnosti, ki je naveden na nalepki poleg oznake „Uporabno do“.
English: Only one patient became wheelchair-free.
Slovenian: Le en bolnik ni več potreboval invalidskega vozička.
English: No significant adverse findings on embryofoetal development were observed in a mouse and a rabbit embryofoetal study and no significant adverse findings were observed in a mouse fertility and early embryonic development study.
Slovenian: V študiji na zarodkih/plodovih miši in zajcev niso opazili pomembnih neželenih učinkov na razvoj zarodka/plodu, ravno tako tudi niso opazili nobenih pomembnih neželenih učinkov v študiji plodnosti in zgodnjega razvoja zarodka pri miših.
English: Higher mean clearance, lower mean AUC∞, and lower mean Cmax were observed in 5 patients who tested positive for inhibition of cellular uptake of enzyme.
Slovenian: Višjo povprečno vrednost klirensa CL, nižjo povprečno vrednost AUC∞ in nižjo povprečno vrednost Cmax so opazili pri 5 bolnikih s pozitivnim testom na zaviranje privzema encimov v celice.
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.
Slovenian: Zaradi možnosti hudih reakcij, povezanih z infuzijo, mora biti ob uporabi zdravila Myozyme takoj na voljo ustrezna zdravniška pomoč, vključno z opremo za srčno-pljučno oživljanje.
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).
Slovenian: Pri bolnikih, kjer se razvijejo protitelesa IgE proti alglukozidazi alfa, dozdevno obstaja večje tveganje za pojavnost IARs ob ponovnem dajanju zdravila Myozyme (glejte poglavje 4.8).
English: Patients were randomized to receive either 20 mg/kg or 40 mg/kg once every two weeks for a period of 52 weeks.
Slovenian: Bolniki so bili naključno izbrani za prejemanje 20 mg/kg ali 40 mg/kg enkrat na vsaka dva tedna v obdobju 52 tednov.
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.
Slovenian: Nekatere IgE pozitivne bolnike so uspešno ponovno zdravili z zdravilom Myozyme z uporabo manjše hitrosti infundiranja in manjšimi začetnimi odmerki, le-ti so nato nadaljevali s prejemanjem zdravila Myozyme ob skrbnem kliničnem nadzoru.
English: At 104 weeks of treatment LVM assessments were available for 10 patients, of which 9 decreased to within normal limits.
Slovenian: Pri 104. tednu zdravljenja so bile na voljo ocene LVM za 10 bolnikov, od katerih se je 9-im znižala na normalne vrednosti.
English: If immune-mediated reactions occur, discontinuation of the administration of alglucosidase alfa should be considered and appropriate medical treatment initiated.
Slovenian: Če pride do imunsko posredovanih reakcij, morate presoditi o prekinitvi dajanja alglukozidaze alfa in uvesti ustrezno zdravljenje.
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.
Slovenian: Deset bolnikov z napredujočo Pompejevo boleznijo s poznim nastopom (npr. na invalidskem vozičku 10/10 in odvisnih od respiratorja 9/10) starih od 9-54 let so zdravili v programih z razširjenim dostopom z alglukozidazo alfa v odmerkih 20-40 mg/kg enkrat na vsaka dva tedna v različnih časovnih obdobjih med 6 meseci in 2,5 leta.
English: Myozyme is given through a drip into a vein (by intravenous infusion).
Slovenian: Zdravilo Myozyme se daje v veno (z intravenskim infundiranjem).
English: Class, presented by frequency categories: very common (≥1/10), common (≥1/100 to<1/10), uncommon (≥1/1,000 to <1/100), rare (≥1/10,000 to <1/1,000), very rare (<1/10,000) and not known
Slovenian: Tabela 1: Neželeni učinki (o katerih so poročali najmanj pri 2 bolnikih) in neželeni učinki, o katerih so poročali v postmarketinških raziskavah, programih razširjenega dostopa in nenadzorovanih kliničnih preskušanjih, po organskih sistemih, predstavljeni po kategorijah pogostnosti: zelo pogosti (≥ 1/10), pogosti (≥ 1/100 do <1/10), občasni (≥ 1/1.000 do < 1/100), redki (≥ 1/10.000 do <1/1.000), zelo redki (<1/10.000) in neznana (ni mogoče oceniti iz razpoložljivih podatkov).
English: Because it is a recombinant human protein, alglucosidase alfa is an unlikely candidate for cytochrome P450 mediated drug-drug interactions.
Slovenian: Ker je alglukozidaza alfa rekombinantna humana beljakovina, je malo verjetno, da bi imela kakšno medsebojno delovanje z drugimi zdravili, ki se presnavljajo s pomočjo citokroma P450.
English: Therefore, a 0.2 micron low protein binding in-line filter should be used for administration.
Slovenian: Zato je treba na cevki uporabiti 0,2-mikronski filter, ki veže nizkomolekularne beljakovine.
English: Patients with Pompe disease treated with immunosuppressive agents maybe at further increased risk of developing severe infections and vigilance is recommended.
Slovenian: Bolniki s Pompejevo boleznijo, zdravljeni z imunosupresivnimi zdravili, imajo lahko dodatno povečano tveganje za pojav hudih okužb, zato je priporočljiva pazljivost.
English: If you experience swelling of your lower limbs or generalized swelling, please inform your doctor.
Slovenian: Če opazite otekanje spodnjih okončin ali otekanje po vsem telesu, obvestite zdravnika.
English: Serious infusion reactions including urticaria, rales, tachycardia, decreased oxygen saturation, bronchospasm, tachypnea, periorbital edema and hypertension have been reported.
Slovenian: Resne infuzijske reakcije so vključevale urtikarijo, hropenje, tahikardijo, zmanjšano saturacijo s kisikom, bronhospazem, tahipnejo, periorbitalni edem in visok krvni tlak.
English: There is no experience with overdose of alglucosidase alfa.
Slovenian: Ni izkušenj s prevelikim odmerjanjem alglukozidaze alfa.
English: If severe hypersensitivity or anaphylactic reactions occur, immediate discontinuation of Myozyme infusion should be considered and appropriate medical treatment should be initiated.
Slovenian: Če pride do hudih preobčutljivostnih ali anafilaktičnih reakcij, je treba z infundiranjem zdravila Myozyme takoj prekiniti in začeti z ustreznim zdravljenjem.
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.
Slovenian: Uporabo ITI in alglukozidaze alfa so ocenili v enem kliničnem preskušanju ter z retrospektivnim pregledom dokumentacije bolnikov, ki do uvedbe zdravljenja še niso prejemali encimskega nadomestnega zdravljenja, in v enem kliničnem preskušanju bolnikov, ki so do uvedbe ITI že prejemali alglukozidazo alfa.
English: The majority of patients were successfully re-challenged with alglucosidase alfa using lower doses and/or pretreatment with antiinflammatory drugs and/or corticosteroids and have continued to receive treatment under close clinical supervision.
Slovenian: Pri večini bolnikov je bila ponovna uporaba alglukozidaze alfa ob uporabi manjših odmerkov in/ali predhodnem zdravljenju s protivnetnimi zdravili in/ali kortikosteroidi uspešna in so še naprej prejemali alglukozidazo alfa pod skrbnim kliničnim nadzorom.
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.
Slovenian: Dodatni 4 bolniki so med študijo motorično napredovali in so samostojno sedeli do zadnje ocene študije (s trajanjem zdravljenja pri posameznih bolnikih v razponu od 78 do 130 tednov; povprečno obdobje sledenja 110 tednov), čeprav nog niso funkcionalno uporabljali.
English: Patients who experience hypersensitivity reactions may also be tested for IgE antibodies to alglucosidase alfa and other mediators of anaphylaxis.
Slovenian: Pri bolnikih s preobčutljivostnimi reakcijami lahko izvedete testiranja za protitelesa IgE na alglukozidazo alfa in druge posrednike anafilakse.
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.
Slovenian: V študiji razvoja zarodka/plodu pri zajcih so po dajanju zdravila Myozyme (1040 mg/kg/dan) skupaj z difenhidraminom opazili z zdravljenjem povezan porast pogostosti splava in predčasne kotitve.
English: Also remove an equal volume of sodium chloride 9 mg/ml (0.9%) solution for injection, that will be replaced with reconstituted Myozyme.
Slovenian: Odstranite tudi enako količino natrijevega klorida 9 mg/ml (0,9 %), raztopine za injiciranje, ki ga bo nadomestilo rekonstituirano zdravilo Myozyme.
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.
Slovenian: Opisana je bila netipična, počasneje napredujoča oblika Pompejeve bolezni z zgodnjim nastopom, za katero je značilna blažja kardiomiopatija in posledično podaljšano preživetje.
English: Some patients have been successfully rechallenged and continued to receive alglucosidase alfa under close clinical supervision.
Slovenian: Pri nekaterih bolnikih je bila ponovna uporaba uspešna in so še naprej prejemali alglukozidazo alfa pod skrbnim kliničnim nadzorom.
English: Patients with renal and hepatic impairment
Slovenian: Bolniki z ledvično ali jetrno okvaro
English: Perform an immediate inspection of the reconstituted vials for particulate matter and discoloration.
Slovenian: Rekonstituirane viale takoj preglejte zaradi morebitnih delcev in spremembe barve.
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.
Slovenian: Pri bolnikih s poznim nastopom Pompejeve bolezni, ki jo običajno spremlja progresivna miopatija pretežno proksimalnih mišic v medeničnem in ramenskem obroču in različne stopnje dihalne prizadetosti, na koncu stanje napreduje do hude nesposobnosti in/ali potrebe po podpori respiratorja.