natgillin/translations-raw huggingface dataset:
Fabrazyme should not be used in people who may be hypersensitive (allergic) to agalsidase beta or any of the other ingredients.
Zdravila Fabrazyme ne smejo uporabljati osebe, ki utegnejo biti preobčutljive za (alergične na) agalzidazo beta ali katerokoli drugo sestavino.
The Committee for Medicinal Products for Human Use (CHMP) decided that, for patients with Fabry disease, treatment with Fabrazyme might provide long-term clinical benefits.
Odbor za zdravila za uporabo v humani medicini (CHMP) je odločil, da lahko bolnikom s Fabryjevo boleznijo zdravljenje z zdravilom Fabrazyme zagotovi dolgoročne klinične koristi.
The CHMP decided that Fabrazyme’ s benefits are greater than its risks for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease.
CHMP je zaključil, da so koristi zdravila Fabrazyme za dolgotrajno encimsko nadomestno zdravljenje pri bolnikih s potrjeno diagnozo Fabryjeve bolezni večje od z njim povezanih tveganj.
The Committee recommended that Fabrazyme be given marketing authorisation.
Odbor je priporočil, da se za zdravilo Fabrazyme odobri dovoljenje za promet z zdravilom.
Fabrazyme was originally authorised under ‘ Exceptional Circumstances’, because, as the disease is rare, limited information was available at the time of approval.
Zdravilo Fabrazyme je bilo prvotno odobreno v „ izjemnih okoliščinah “, ker zaradi redkosti bolezni v času odobritve ni bilo mogoče pridobiti vseh informacij.
As the company had supplied the additional information requested, the ‘ Exceptional Circumstances ’ ended on 6 February 2008.
Ker je družba predložila dodatne zahtevane informacije, so se „ izjemne okoliščine “ preklicane 6. februarja 2008.
Other information about Fabrazyme:
Druge informacije o zdravilu Fabrazyme:
The European Commission granted a marketing authorisation valid throughout the European Union for Fabrazyme to Genzyme Europe B. V. on 3 August 2001.
Evropska komisija je dovoljenje za promet z zdravilom Fabrazyme, veljavno po vsej Evropski uniji, odobrila družbi Genzyme Europe B. V. dne 3. avgusta 2001.
The marketing authorisation was renewed on 3 August 2006.
Dovoljenje za promet z zdravilom je bilo podaljšano dne 3. avgusta 2006.
The record of Fabrazyme’ s designation as an orphan medicine is available here.
Zapis o določitvi zdravila Fabrazyme kot zdravila sirote najdete tukaj.
The full EPAR for Fabrazyme can be found here.
Celotno evropsko javno poročilo o oceni zdravila (EPAR) za zdravilo Fabrazyme je na voljo tukaj.
EU/ 1/ 01/ 188/ 001 EU/ 1/ 01/ 188/ 002 EU/ 1/ 01/ 188/ 003 EU/ 1/ 01/ 188/ 004 EU/ 1/ 01/ 188/ 005 EU/ 1/ 01/ 188/ 006
Številka EU EU/ 1/ 01/ 188/ 001 EU/ 1/ 01/ 188/ 002 EU/ 1/ 01/ 188/ 003 EU/ 1/ 01/ 188/ 004 EU/ 1/ 01/ 188/ 005 EU/ 1/ 01/ 188/ 006
Fabrazyme Fabrazyme Fabrazyme Fabrazyme Fabrazyme Fabrazyme
Izmišljeno ime Fabrazyme Fabrazyme Fabrazyme Fabrazyme Fabrazyme Fabrazyme
35 mg 35 mg 35 mg 5 mg 5 mg 5 mg
Jakost 35 mg 35 mg 35 mg 5 mg 5 mg 5 mg
Powder for concentrate for solution for infusion Intravenous use Powder for concentrate for solution for infusion Intravenous use Powder for concentrate for solution for infusion Intravenous use Powder for concentrate for solution for infusion Intravenous use Powder for concentrate for solution for infusion Intravenous use Powder for concentrate for solution for infusion Intravenous use
Farmacevtska oblika Prašek za koncentrat za raztopino za infundiranje Prašek za koncentrat za raztopino za infundiranje Prašek za koncentrat za raztopino za infundiranje Prašek za koncentrat za raztopino za infundiranje Prašek za koncentrat za raztopino za infundiranje Prašek za koncentrat za raztopino za infundiranje
Vial (glass) Vial (glass) Vial (glass) Vial (glass) Vial (glass) Vial (glass)
Ovojnina Viala (steklena) Viala (steklena) Viala (steklena) Viala (steklena) Viala (steklena) Viala (steklena)
1 vial 5 vials 10 vials 1 vial 5 vials 10 vials
Velikost pakiranja 1 viala 5 vial 10 vial 1 viala 5 vial 10 vial
Fabrazyme 35 mg, powder for concentrate for solution for infusion.
Fabrazyme 35 mg prašek za koncentrat za raztopino za infundiranje
After reconstitution with 7.2 ml water for injections, each vial of Fabrazyme contains 5 mg/ ml (35 mg/ 7 ml) of agalsidase beta.
Po rekonstituciji s 7, 2 ml vode za injekcije vsebuje vsaka viala zdravila Fabrazyme 5 mg/ ml (35 mg/ 7 ml) agalzidaze beta.
The reconstituted solution must be diluted further (see section 6.6).
Rekonstituirano raztopino je treba dodatno redčiti (glejte poglavje 6. 6).
Agalsidase beta is a recombinant form of human α -galactosidase A and is produced by recombinant DNA technology using a mammalian Chinese Hamster Ovary (CHO) cell culture.
Agalzidaza beta je rekombinantna oblika človeškega encima α- galaktozidaze A, ki ga izdelujejo s tehnologijo rekombinantne DNA v sesalskih celičnih kulturah ovarijskih celic kitajskega hrčka (Chinese Hamster Ovary, CHO).
The amino acid sequence of the recombinant form, as well as the nucleotide sequence which encoded it, are identical to the natural form of α -galactosidase.
Aminokislinsko zaporedje rekombinantne oblike, kakor tudi nukleotidno zaporedje, ki ga kodira, sta popolnoma enaka naravni obliki encima α- galaktozidaze.
Powder for concentrate for solution for infusion White to off-white lyophilized cake or powder
Bela do sivkastobela, liofilizirana sredica ali prašek.
Fabrazyme is indicated for long-term enzyme replacement therapy in patients with a confirmed diagnosis of Fabry disease (α -galactosidase A deficiency).
Zdravilo Fabrazyme je indicirano za dolgoročno encimsko nadomestno terapijo pri bolnikih s potrjeno diagnozo Fabryjeve bolezni (pomanjkanje α- galaktozidaze A).
Fabrazyme treatment should be supervised by a physician experienced in the management of patients with Fabry Disease or other inherited metabolic diseases.
Zdravljenje z zdravilom Fabrazyme mora potekati pod nadzorom zdravnika, izkušenega v zdravljenju bolnikov s Fabryjevo boleznijo ali drugimi dednimi presnovnimi boleznimi.
The recommended dose of Fabrazyme is 1mg/ kg body weight administered once every 2 weeks as an intravenous infusion.
Priporočeni odmerek zdravila Fabrazyme je 1 mg/ kg telesne mase in ga dajemo enkrat na vsaka 2 tedna v obliki intravenske infuzije.
For further instructions see section 6.6.
Za nadaljnja navodila glejte poglavje 6. 6.
In one of these studies, after an initial dose of 1.0 mg/ kg every 2 weeks for 6 months, 0.3 mg/ kg every 2 weeks may maintain clearance of GL-3 in certain cell types in some patients; however, the long term clinical relevance of these findings has not been established (see section 5.1).
V eni od študij lahko po začetnem odmerku 1, 0 mg/ kg vsaka 2 tedna v trajanju 6 mesecev, 0, 3 mg/ kg vsaka 2 tedna vzdržuje očistek GL- 3 pri nekaterih vrstah celic in pri nekaterih bolnikih; vendar pa dolgoročne klinične pomembnosti teh izsledkov niso ugotavljali (glejte poglavje 5. 1).
The initial infusion rate should be no more than 0.25 mg/ min (15 mg/ hour) to minimise the potential occurrence of infusion-associated reactions.
Začetna hitrost infundiranja ne sme preseči 0, 25 mg/ min (15 mg/ uro), da zmanjšamo pojavljanje morebitnih infuzijskih reakcij na najmanjšo možno mero.
2 Paediatric patients Studies in children 0-7 years have not been performed and no dosage regimen can presently be recommended in patients in this paediatric age group as safety and efficacy have not yet been established.
2 odmerjanja.
Life threatening hypersensitivity (anaphylactic reaction) to the active substance or any of the excipients.
Smrtno nevarna preobčutljivost (anafilaktična reakcija) za učinkovino ali katero koli pomožno snov.
Since agalsidase beta (r-hα GAL) is a recombinant protein, the development of IgG antibodies is expected in patients with little or no residual enzyme activity.
Ker je agalzidaza beta (r- hαGAL) rekombinantni protein, pričakujemo tvorjenje protiteles IgG pri bolnikih z majhno ali neobstoječo preostalo encimsko aktivnostjo.
The majority of patients developed IgG antibodies to r-hα GAL, typically within 3 months of the first infusion with Fabrazyme.
Večina bolnikov je protitelesa IgG proti r- hαGAL začela tvoriti v 3 mesecih od prve infuzije zdravila Fabrazyme.
Over time, the majority of seropositive patients in clinical trials demonstrated either a downward trend in titers (based on a ≥ 4-fold reduction in titer from the peak measurement to the last measurement) (40% of the patients), tolerised (no detectable antibodies confirmed by 2 consecutive radioimmunoprecipitation (RIP) assays) (14% of the patients) or demonstrated a plateau (35% of the patients).
Sčasoma je večina seropozitivnih bolnikov v kliničnih preskušanjih izkazala padajoči (na osnovi ≥ 4 kratnega zmanjšanja titra od največje meritve do zadnje meritve) (40% bolnikov) tolerirano (s pomočjo 2 zaporednih testov radioimunske percipitacije (RIP) ni bilo zaznati protiteles) (14% bolnikov) oziroma je bil prikazan plato (35% bolnikov).
Patients with antibodies to r-hα GAL have a greater potential to experience infusion-associated reactions (IARs), which are defined as any related adverse event occurring on the infusion day.
Bolniki s protitelesi proti r- hαGAL imajo večjo možnost, da bodo imeli reakcije, povezane z infuzijo, ki so opredeljene kot kakršenkoli neželeni učinek, ki se pojavi na dan infuzije.
These patients should be treated with caution when re-administering agalsidase beta (See section 4.8).
Te bolnike bi morali pri ponovnemu dajanju agalzidaze beta obravnavati previdno (glejte poglavje 4. 8).
In clinical trials, sixty seven percent (67%) of the patients experienced at least one infusion-associated reaction (See section 4.8).
V kliničnih preskušanjih je sedeminšestdeset odstotkov (67%) bolnikov imelo vsaj eno reakcijo, povezano z infuzijo (glejte poglavje 4. 8).
Patients experiencing mild or moderate infusion-associated reactions when treated with agalsidase beta during clinical trials have continued therapy after a reduction in the infusion rate (~0.15 mg/ min; 10 mg/ hr) and/ or pre-treatment with antihistamines, paracetamol, ibuprofen and/ or corticosteroids.
Bolniki, ki so bili med kliničnimi preskušanji zdravljeni z agalzidazo beta in so imeli blage do zmerne reakcije, povezane z infuzijo, so zdravljenje nadaljevali po zmanjšanju hitrosti infundiranja (~0, 15 mg/ min; 10 mg/ uro) in/ ali predhodnem zdravljenju z antihistaminiki, paracetamolom, ibuprofenom in/ ali kortikosteroidi.
As with any intravenous protein product, allergic-type hypersensitivity reactions are possible.
Kot pri vsakem intravenskem beljakovinskem zdravilu so tudi pri uporabi zdravila Fabrazyme možne alergijske preobčutljivostne reakcije.
If severe allergic or anaphylactic-type reactions occur, immediate discontinuation of the administration of Fabrazyme should be considered and appropriate treatment initiated.
Če nastopijo hude alergijske ali anafilaktične reakcije, pretehtajte, ali je potrebna takojšnja ustavitev aplikacije zdravila Fabrazyme in uvedite ustrezno zdravljenje.
In this trial, the initial rechallenge administration was at a low dose and a lower infusion rate (1/ 2 the therapeutic dose at 1/ 25 the initial standard recommended rate).
V tem preskušanju so začetno ponovno izzivalno dajanje izvajali ob manjšem odmerku in manjši hitrosti infundiranja (1/ 2 terapevtskega odmerka pri 1/ 25 začetne standardno priporočene hitrosti).
Once a patient tolerates the infusion, the dose may be increased to reach the therapeutic dose of 1 mg/ kg and the infusion rate may be increased by slowly titrating upwards, as tolerated.
Ko bolnik tako infuzijo dobro prenaša, lahko odmerek povečate do terapevtskega odmerka 1 mg/ kg, hitrost infundiranja pa lahko počasi večate s titracijo navzgor, v skladu s prenašanjem bolnika.
Fabrazyme should not be administered with chloroquine, amiodarone, benoquin or gentamycin due to a theoretical risk of inhibition of intra-cellular α -galactosidase activity.
Zdravila Fabrazyme ne smete uporabljati sočasno s klorokvinom, amiodaronom, benokvinom ali gentamicinom zaradi teoretičnega tveganja za inhibicijo znotrajcelične aktivnosti α- galaktozidaze.
3 4.6 Pregnancy and lactation
3 4. 6 Nosečnost in dojenje
Animal studies do not indicate direct or indirect harmful effects with respect to embryonal/ foetal development (See section 5.3).
Študije na živalih ne kažejo na neposredne ali posredne škodljive vplive na razvoj zarodka/ plodu (glejte poglavje 5. 3).
No studies on the ability to drive and use machines have been performed with Fabrazyme.
Študije z zdravilom Fabrazyme o vplivu na sposobnost vožnje in upravljanja s stroji niso bile opravljene.
Adverse drug reactions (ADRs) reported from clinical trials to be related to Fabrazyme administered at a dose of 1mg/ kg in a total of 168 patients (154 males and 14 females) treated with Fabrazyme for a minimum of one infusion up to a maximum of 5 years are listed by System Organ Class and frequency (very common ≥ 1/ 10; common ≥ 1/ 100 to < 1/ 10 and uncommon ≥ 1/ 1000 to < 1/ 100) in the table below.
Neželeni učinki zdravila (ADR – Adverse Drug Reaction), poročani med kliničnim preskušanjem v povezavi z zdravilom Fabrazyme pri odmerku 1 mg/ kg pri skupaj 168 bolnikih (154 moških in 14 žensk), zdravljenih z najmanj eno infuzijo zdravila Fabrazyme do največ 5 let, so navedeni v spodnji preglednici po organskih sistemih in po pogostnosti (zelo pogosti: > 1/ 10, pogosti: ≥ 1/ 100 do < 1/ 10 in občasni: ≥ 1/ 1. 000 do < 1/ 100).
The occurrence of an ADR in a single patient is defined as uncommon in light of the relatively small number of patients treated.
Glede na majhno število zdravljenih bolnikov se pojav neželenega učinka zdravila pri enem bolniku upošteva za občasnega.
ADRs only reported during the Post Marketing period are also included in the table below at a frequency category of “ unknown”.
V preglednici spodaj so v kategoriji » neznano « zajeti tudi neželeni učinki zdravila, o katerih se je poročalo samo med obdobjem po začetku trženja.
ADRs were mostly mild to moderate in severity:
Po jakosti so bili neželeni učinki zdravila večinoma blagi do zmerni:
Incidence of Related Adverse Events with Fabrazyme Treatment
Pogostnost neželenih učinkov pri zdravljenju z zdravilom Fabrazyme
Frequency
Pogostost neznana
Class Infections and infestations
Infekcijske in parazitske bolezni
common
-
nasopharyngitis
nazofaringitis
rhinitis
rinitis
unknown
-
anaphylactoid reaction
anafilaktična reakcija
Headache, paraesthesia
glavobol,
dizziness, somnolence, hypoaesthesia, burning sensation, lethargy, syncope
omotica,
Eye disorders
parestezija
tinnitus, vertigo
zatekanje ušes,
auricular swelling, ear pain
bolečina v ušesih sinusna
sinus bradycardia
bradikardija
4 Incidence of Related Adverse Events with Fabrazyme Treatment (continued)
4 Pogostnost neželenih učinkov pri zdravljenju z zdravilom Fabrazyme (nadaljevanje)
peripheral coldness
periferna hladnost bronhospazem,
---
--- ---
bronchospasm, pharyngolaryngeal pain, rhinnorhoea, tachypnoea, upper respiratory tract congestion
bolečine v požiralniku in grlu, rinoreja, tahipneja, kongestija zgornjega dela dihalnih poti
---
navzea, bruhanje
Gastrointestinal Disorders
trebušne bolečine,
nausea, vomiting
disepsija,
abdominal pain, abdominal pain upper, abdominal discomfort, stomach discomfort, hypoaesthesia oral, diarrhoea
bolečine v zgornjem trebušnem predelu, neugodje v trebuhu, neugodje v želodcu, oralna hipestezija, diareja
dyspepsia, dysphagia
Bolezni kože in
Skin and subcutaneous tissue disorders
livedo reticularis,
---
podkožja
pruritus, urticaria, rash, erythema, pruritus generalized, angioneurotic oedema, swelling face, rash maculo- papular
urtikarija, izpuščaj, eritem, generaliziran
livedo reticularis, rash erythematous, rash pruritic, skin discolouration, skin discomfort
pruritus, angionevrotični edem, zatekanje obraza, makulopapulozni izpuščaj
leukocytoclastic vasculitis
izpuščaj, obarvanje kože, neugoden občutek na koži
pain in extremity, myalgia, back pain, muscle spasms, arthralgia, muscle tightness, musculoskeletal stiffness
bolečine v udih, mialgija, bolečine v hrbtu, mišični krči, artralgija, zategnjenost mišic, mišično - skeletna togost
musculoskeletal pain
mišično- skeletne bolečine občutek vročine
chills, pyrexia,
mrzlica, pireksija,
fatigue, chest discomfort, feeling
utrujenost,
feeling hot and cold, influenza-like
in mraza, gripi
hot, oedema peripheral, pain, asthenia, chest pain, face oedema, hyperthermia
neugodje v prsih, občutek vročine, periferni edem, bolečina, astenija, bolečine v prsih, obrazni edem, hipertermija
illness, infusion site pain, infusion site reaction, injection site thrombosis, malaise, oedema
podobna bolezen, bolečina na mestu infundiranja, reakcija na mestu infundiranja, tromboza na mestu injiciranja, bolehnost, edem
For the purpose of this table, ≥ 1% is defined as events occurring in 2 or more patients.
5 Za to preglednico je vrednost ≥1% opredeljena kot dogodek, do katere je prišlo pri dveh ali več pacientih.
AE terminology is based upon the Medical Dictionary for Regulatory Activities (MedDRA)
Terminologija neželenega dogodka (AE - Adverse Event) temelji na medicinskem slovarju za regulatorne aktivnosti (Medical Dictionary for Regulatory Activities, MedDRA)
Additional symptoms included mild or moderate dyspnoea, throat tightness, chest discomfort, flushing, pruritus, urticaria, face oedema, angioneurotic oedema, rhinitis, bronchospasm, tachypnoea, wheezing, hypertension, hypotension, tachycardia, palpitations, abdominal pain, nausea, vomiting, infusion-related pain including pain at the extremities, myalgia, and headache.
Dodatni simptomi so bili blaga ali zmerna dispneja, stiskanje v grlu, neugodje v prsih, navali vročine, pruritus, urtikarija, obrazni edem, angionevrotični edem, rinitis, bronhospazem, tahipneja, sopihajoče in piskajoče dihanje, hipertenzija, hipotenzija, tahikardija, palpitacije, trebušne bolečine, navzea, bruhanje, bolečine zaradi infuzije, vključno z bolečinami v udih, mialgijo in glavobolom.
The infusion-associated reactions were managed by a reduction in the infusion rate together with the administration of non-steroidal anti-inflammatory medicinal products, antihistamines and/ or corticosteroids.
Reakcije, povezane z infuzijo, so zdravili z zmanjšanjem hitrosti infundiranja in sočasno uporabo nesteroidnih protivnetnih zdravil, antihistaminikov in/ ali kortikosteroidov.
Sixty seven percent (67%) of the patients experienced at least one infusion-associated reaction.
Sedeminšestdeset odstotkov (67%) bolnikov je imelo vsaj eno reakcijo, povezano z infuzijo.
5 attributed to the formation of IgG antibodies and/ or complement activation.
Pogostnost teh reakcij je sčasoma upadla. Večino teh reakcij lahko pripišemo nastanku protiteles IgG in/ ali aktivaciji komplementa.
In a limited number of patients IgE antibodies were demonstrated (see Section 4.4).
Pri omejenem številu bolnikov so dokazali protitelesa IgE (glejte poglavje 4. 4).
Paediatric patients Limited information suggests that the safety profile of Fabrazyme treatment in paediatric patients (above the age of 7) is not different with that seen in adults.
Pediatrični bolniki Omejeni podatki nakazujejo, da varnostni profil zdravljenja z zdravilom Fabrazyme pri pediatričnih bolnikih (nad starostjo 7 let) ni drugačen od tistega, ki je bil opažen pri odraslih.
In clinical trials doses up to 3mg/ kg body weight were used.
V kliničnih preskušanjih so uporabljali odmerke do 3 mg/ kg telesne mase.
Alimentary tract and metabolism products – enzymes.
Druga zdravila za bolezni prebavil in presnove – encimi.
A16AB04 agalsidase beta.
A16AB04 agalzidaza beta.
It is characterised by the deficiency of α -galactosidase.
Zanjo je značilno pomanjkanje encima α- galaktozidaze.
Reduced or absent α - galactosidase activity results in the accumulation of GL-3 in the lysosomes of many cell types including the endothelial and parenchymal cells, ultimately leading to life-threatening clinical deteriorations as a result of renal, cardiac and cerebrovascular complications.
Zmanjšana aktivnost ali odsotnost α- galaktozidaze povzroči kopičenje GL- 3 v lizosomih številnih vrst celic, vključno z endotelijskimi in parenhimskimi celicami, kar na koncu privede do življenjsko nevarnih kliničnih poslabšanj, ki so posledica ledvičnih, srčnih ali cerebrovaskularnih zapletov.
After intravenous infusion, agalsidase beta is rapidly removed from the circulation and taken up by vascular endothelial and parenchymal cells into lysosomes, likely through the mannose-6 phosphate, mannose and asialoglycoprotein receptors.
Po intravenskem infundiranju se agalzidaza beta hitro odstrani iz krvnega obtoka, ker jo absorbirajo celice žilnega endotelija in parenhimske celice v svoje lizosome, verjetno prek manoza- 6- fosfatnih, manoznih in asialoglikoproteinskih receptorjev.
In the dose finding study, the effects of 0.3, 1.0 and 3.0 mg/ kg once every 2 weeks and 1.0 and 3.0 mg/ kg once every 2 days were evaluated.
V študijah za ugotavljanje odmerka so ovrednotili učinke odmerkov 0, 3, 1, 0 in 3, 0 mg/ kg enkrat na vsaka 2 tedna in odmerkov 1, 0 in 3, 0 mg/ kg enkrat na vsaka 2 dni.
A reduction in GL-3 was observed in kidney, heart, skin and plasma at all doses.
Pri vseh odmerkih so ugotavljali zmanjšanje količine GL- 3 v ledvicah, srcu, koži in plazmi.
Plasma GL-3 was cleared in a dose dependent manner, but was less consistent at the dose of 0.3 mg/ kg.
Iz plazme se je GL- 3 odstranjeval v odvisnosti od odmerka, vendar pa je bil ta učinek manj konsistenten pri odmerku 0, 3 mg/ kg.
In the first placebo-controlled clinical trial, Fabrazyme was effective in clearing GL-3 from the vascular endothelium of the kidney after 20 weeks of treatment.
6 V prvem kliničnem preskušanju, nadzorovanem s placebom, je bilo zdravilo Fabrazyme učinkovito pri odstranjevanju GL- 3 iz ledvičnega žilnega endotelija po 20 tednih zdravljenja.
This clearance was achieved in 69% (20/ 29) of the Fabrazyme treated patients, but in none of the placebo patients (p < 0.001).
Očistek je bil dosežen pri 69% (20/ 29) bolnikov, zdravljenih z zdravilom Fabrazyme, in pri nobenem od tistih, ki so prejemali placebo (p < 0, 001).
This finding was further supported by a statistically significant decrease in GL-3 inclusions in kidney, heart and skin combined and in the individual organs in patients treated with agalsidase beta compared to placebo patients (p < 0.001).
Ta izsledek je bil podprt tudi s statistično občutnim zmanjšanjem skupnega števila inkluzij GL- 3 v ledvicah, srcu in koži ter v posameznih organih bolnikov, zdravljenih z agalzidazo beta, v primerjavi s tistimi, ki so prejemali placebo (p < 0, 001).
Sustained clearance of GL-3 from kidney vascular endothelium upon agalsidase beta treatment was demonstrated further in the open label extension of this trial.
Trajen očistek GL- 3 iz žilnega endotelija ledvic po zdravljenju z agalzidazo beta so nadalje dokazali v razširitvi te študije odprtega tipa.
This was achieved in 47 of the 49 patients (96%) with available information at month 6, and in 8 of the 8 patients (100%) with available information at the end of the study (up to a total of 5 years of treatment).
To je bilo doseženo pri 47 od 49 bolnikov (96%), s podatki, ki so bili na voljo ob koncu 6. meseca, in pri 8 od 8 bolnikov (100%) s podatki, ki so bili na voljo ob koncu študije (do največ 5 let zdravljenja).
Clearance of GL-3 was also achieved in several other cell types from the kidney.
Očistek GL- 3 je bil dosežen tudi pri nekaterih drugih vrstah ledvičnih celic.
Plasma GL-3 levels rapidly normalised with treatment and remained normal through 5 years.
Nivoji plazemskega GL- 3 so se z zdravljenjem hitro normalizirali in so ostali normalni tekom 5 let.
6 Although no specific study has been conducted to assess the effect on the neurological signs and symptoms, the results also indicate that patients may achieve reduced pain and enhanced quality of life upon enzyme replacement therapy.
Čeprav ni bilo izvedene posebne študije za ocenitev učinka na nevrološke znake in simptome, rezultati kažejo, da po encimskem nadomestnem zdravljenju pri bolnikih lahko pride do zmanjšanja bolečin in izboljšanja kvalitete življenja.