FrancophonIA/ELRC3.0 huggingface dataset:
English: • have sickle cell anaemia.
Croatian: • ako bolujete od anemije srpastih stanica.
English: Ristempa contains less than 1 mmol (23 mg) sodium per 6 mg dose, i.e. essentially ‘sodium-free'.
Croatian: Ristempa sadrži manje od 1 mmol natrija (23 mg) po dozi od 6 mg, tj.
English: Each pre-filled syringe contains 0.6 ml of solution for injection.
Croatian: Jedna napunjena štrcaljka sadrži 0,6 ml otopine za injekciju.
English: Back- Ristempa reference guide:
Croatian: Stražnja strana - Vodič za primjenu lijeka Ristempa:
English: The injections should be given into the thigh, abdomen or upper arm. For instructions on handling of the medicinal product before administration, see section 6.6.
Croatian: Za uputu o rukovanju s lijekom prije primjene, vidjeti dio 6.6.
English: Ristempa 6 mg injection Pegfilgrastim
Croatian: Ristempa 6 mg injekcija pegfilgrastim
English: Elevations in lactate dehydrogenase and alkaline phosphatase Transient elevations in LFT's for ALT or AST1
Croatian: Povišena razina laktatdehidrogenaze i alkalne fosfataze
English: If you are nursing during Ristempa treatment, you may be encouraged to enrol in Amgen's Lactation Surveillance programme.
Croatian: Ukoliko ipak dojite tijekom primjene Ristempe, možda će Vas liječnik potaknuti da se uključite u Amgenov program praćenja dojenja.
English: Unless your doctor directs you otherwise, you must stop breast feeding if you use Ristempa.
Croatian: Ako primjenjujete Ristempu, morate prekinuti s dojenjem, osim ako Vaš liječnik ne savjetuje drugačije.
English: Front - Ristempa reference guide:
Croatian: Prednja strana - Vodič za primjenu lijeka Ristempa:
English: Syringe safety guard
Croatian: Sigurnosna opruga za
English: Acute Respiratory Distress
Croatian: Plućne nuspojave
English: Nervous system disorders Vascular disorders
Croatian: Poremećaji živčanog sustava Krvožilni poremećaji
English: Allow the pre-filled syringe to reach room temperature before injecting.
Croatian: Prije injiciranje pričekajte da napunjena štrcaljka dosegne sobnu temperaturu.
English: Do not use it if the date has passed
Croatian: Ne koristite napunjenu štrcaljku ako je prošao posljednji dan navedenog mjeseca.
English: BLISTERED SYRINGE LABEL
Croatian: Naljepnica na štrcaljki
English: Injection site reactions2
Croatian: Reakcije na mjestu injiciranja
English: Injecting Ristempa yourself
Croatian: Samostalno davanje Ristempe
English: Each pack contains 1 pre-filled syringe of type I glass with an attached stainless steel needle and needle cap.
Croatian: I s pripojenom iglom od nehrđajućeg čelika i kapicom za iglu.
English: Ristempa 6 mg solution for injection in a pre-filled syringe pegfilgrastim
Croatian: Ristempa 6 mg otopina za injekciju u napunjenoj štrcaljki pegfilgrastim
English: Ristempa 6 mg solution for injection.
Croatian: NAZIV LIJEKA Ristempa 6 mg otopina za injekciju
English: Ristempa 6 mg solution for injection Pegfilgrastim
Croatian: Ristempa 6 mg otopina za injekciju pegfilgrastim
English: Ristempa 6 mg injection Pegfilgrastim SC
Croatian: Ristempa 6 mg injekcija pegfilgrastim s.c.
English: *Sodium acetate is formed by titrating glacial acetic acid with sodium hydroxide.
Croatian: 6.1 Popis pomoćnih tvari natrijev acetat* sorbitol (E420) polisorbat 20 voda za injekcije *Natrijev acetat nastaje titracijom ledene acetatne kiseline s natrijevim hidroksidom.
English: Medicine
Croatian: Dojenje
English: • spleen rupture.
Croatian: Ruptura slezene
English: Reporting of side effects
Croatian: Priručnik o dijelovima
English: OUTER CARTON FOR UNBLISTERED SYRINGE
Croatian: NALJEPNICA NA ŠTRCALJKI BEZ BLISTERA
English: OUTER CARTON FOR BLISTERED SYRINGE
Croatian: NALJEPNICA NA ŠTRCALJKI U BLISTERU
English: Uncommon events of cutaneous vasculitis have been reported in patients treated with Ristempa.
Croatian: U bolesnika liječenih Ristempom zabilježeni su manje česti slučajevi kožnog vaskulitisa.
English: 9) was observed in younger children aged 0-5 yrs (8.9 days) compared to older children aged 6-11 years and 12-21 years (6 days and 3.7 days, respectively) and adults.
Croatian: 6-11 i 12-21 godine (6 odnosno 3,7 dana) te odrasle.
English: A Hold the pinch.
Croatian: Držite nabor kože.
English: You can use: • Upper part of your thigh.
Croatian: • Gornji dio bedra.
English: As the company had supplied the additional information requested, the ‘exceptional circumstances' ended on 27 May 2015.
Croatian: Budući da je tvrtka dostavila dodatne zatražene informacije, „iznimne okolnosti” prestale su 27. svibnja 2015.
English: ‘Advanced' means that the cancer has started to spread.
Croatian: "Uznapredovano" znači da se rak počeo širiti.
English: In both studies, the main measure of effectiveness was how long the patients lived without their disease getting worse.
Croatian: U oba ispitivanja, glavna mjera djelotvornosti bilo je koliko su dugo bolesnici živjeli, a da se njihova bolest nije pogoršala.
English: • ovarian cancer;
Croatian: • rak jajnika;
English: Treatment carries on for as long as the patient shows a benefit.
Croatian: Liječenje se nastavlja tako dugo dok bolesnik pokazuje koristi.
English: This can prevent the cells from dividing too quickly, slowing down the growth of various types of cancer.
Croatian: Ovo može spriječiti stanice od prebrzog dijeljenja, što usporava rast različitih tipova raka.
English: The study compared two different dosing schedules of Yondelis: three times per month, or once every three weeks.
Croatian: Ispitivanje je usporedilo dva različita rasporeda doziranja lijeka Yondelis: tri puta na mjesec ili jednom svaka tri tjedna.
English: Cancer is a disease where cells divide too quickly, usually because the way their genes work is faulty.
Croatian: Rak je bolest uslijed koje se stanice prebrzo dijele, najčešće zato što njihovi geni ne funkcioniraju ispravno.
English: The European Commission granted a marketing authorisation valid throughout the European Union for Yondelis on 17 September 2007.
Croatian: Europska komisija izdala je odobrenje za stavljanje u promet koje je za lijek Yondelis na snazi u Europskoj uniji od 17. rujna 2007.
English: Around two-thirds of the patients had cancer that was sensitive to platinum-containing medicines.
Croatian: Otprilike dvije trećine bolesnika imalo je rak koji je osjetljiv na lijekove koji sadrže platinu.
English: Patients receiving it once every three weeks lived for an average of 3.8 months without their disease getting worse, compared with 2.1 months in those receiving Yondelis three times per month.
Croatian: Bolesnici koji su ga primali jednom svaka tri tjedna živjeli su prosječno 3,8 mjeseci bez pogoršanja bolesti, u usporedbi s 2,1 mjeseca u onih koji su primali Yondelis tri puta mjesečno.
English: Yondelis was originally authorised under ‘exceptional circumstances', because, limited information was available at the time of approval on soft-tissue sarcoma.
Croatian: Yondelis je izvorno odobren u "iznimnim okolnostima", budući da su bile dostupne ograničene informacije u trenutku izdavanja odobrena vezane za sarkom mekanog tkiva.
English: Trabectedin works by attaching to the DNA, the chemical molecule that makes up genes, and preventing some genes in human cells from increasing their activity.
Croatian: Trabectedin djeluje vezujući se na DNK, kemijske molekule koja proizvodi gene, i sprječava pojedine gene u ljudskim stanicama da povećaju svoju aktivnost.
English: Yondelis is used to treat adults with two types of cancer:
Croatian: Yondelis se koristi za liječenje odraslih osoba s dva tipa raka:
English: All of the patients had been treated previously with an anthracycline and ifosfamide but this treatment had stopped working.
Croatian: Svi su bolesnici prethodno liječeni antraciklinom i ifosfamidom, no ova je terapija prestala djelovati.
English: This is a summary of the European public assessment report (EPAR) for Yondelis.
Croatian: Ovo je sažetak europskoga javnog izvješća o ocjeni dokumentacije (EPAR) o lijeku Yondelis.
English: To prevent vomiting and to protect the liver, patients must receive an infusion of corticosteroids such as dexamethasone before treatment.
Croatian: Kako bi se spriječilo povraćanje i zaštitila jetra, bolesnik mora primiti infuziju kortikosteroidima poput deksametazona prije terapije.
English: It is available as a powder that is made up into a solution for infusion (drip into a vein).
Croatian: Dostupan je kao prašak koji se koristi za pripremu otopine za infuziju (drip u venu).
English: For ovarian cancer, it is given at a dose of 1.1 mg/m2 every three weeks as an infusion lasting three hours, immediately after the PLD infusion.
Croatian: U slučaju raka jajnika, primjenjuje se u dozi od 1,1 mg/m2 svaka tri tjedna kao infuzija koja traje tri sata, odmah nakon PLD infuzije.
English: • ovarian cancer (cancer of the ovaries) that has relapsed (come back after previous treatment) and is sensitive to medicines containing platinum.
Croatian: • rakom jajnika koji je u relapsu (vratio je nakon prethodne terapije) i osjetljiv je na lijekove koji sadrže platinu.
English: What measures are being taken to ensure the safe and effective use of Yondelis?
Croatian: Koje se mjere poduzimaju kako bi se osigurala sigurna i učinkovita primjena lijeka Yondelis?
English: The most common side effects of any severity were neutropenia (low levels of neutrophils, a type of white blood cell), nausea (feeling sick), vomiting, increase in liver enzymes, anaemia (low red blood cell counts), tiredness, thrombocytopenia (low blood platelet counts), loss of appetite and diarrhoea.
Croatian: Najčešće nuspojave bilo kojeg stupnja ozbiljnosti su neutropenija (niske razine neutrofila, tipa bijelih krvnih stanica), mučnina (osjećaj slabosti), povraćanje, povećanje jetrenih enzima, anemija (malibroj crvenih krvnih stanica), umor, trombocitopenija (mali broj trombocita u krvi), gubitak apetita i proljev.
English: Yondelis must not be used in patients who have any serious or uncontrolled infection, in combination with the vaccine for yellow fever, or in breast-feeding women.
Croatian: Yondelis se ne smije koristiti u bolesnika koji imaju bilo kakve ozbiljne ili nekontrolirane infekcije, u kombinaciji s cjepivom za žutici ili u dojilja.
English: It explains how the Committee for Medicinal Products for Human Use (CHMP) assessed the medicine to reach its opinion in favour of granting a marketing authorisation and its recommendations on the conditions of use for Yondelis.
Croatian: Objašnjava kako je Odbor za lijekove za humanu uporabu (CHMP) ocijenio lijek da bi donio svoje mišljenje u korist izdavanja odobrenja za stavljanje lijeka u promet te preporuke o uvjetima za primjenu lijeka Yondelis.
English: For soft-tissue sarcoma, Yondelis was more effective when it was given once every three weeks than when it was given with the alternative dosing schedule.
Croatian: U slučaju sarkoma mekanog tkiva, Yondelis je bio djelotvorniji kada se primjenjivao jednom u tri tjedna u odnosno na primjenu prema alternativnom rasporedu doziranja.
English: If the patient's blood counts are abnormal, infusion with Yondelis should be delayed, the dose of Yondelis should be reduced or other medicines can be used to treat the blood problems.
Croatian: Ako su vrijednosti krvi bolesnika abnormalne, infuziju lijekom Yondelis treba odgoditi, dozu lijeka Yondelis treba smanjiti ili se može primijeniti drugi lijek za terapiju problema krvi.
English: The summary of the opinion of the Committee for Orphan Medicinal Products for Yondelis can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/Rare disease designation
Croatian: Sažetak mišljenja Odbora za lijekove za rijetke bolesti o lijeku Yondelis dostupan je na internetskim stranicama Agencije: ema.europa.eu/Find medicine/Human medicines/Rare disease designations:
English: Yondelis is used when treatment with anthracyclines and ifosfamide (other cancer medicines) have stopped working, or in patients who cannot be given these medicines;
Croatian: Yondelis se koristi kada je liječenje antraciklinima i ifosfamidom (drugim antitumorskim lijekovima) prestalo djelovati ili u bolesnika koji ne mogu primiti ove lijekove;
English: Fatal side effects have occurred in 1.9% and 0.9% of patients treated with Yondelis as a single agent and combination therapy, respectively.
Croatian: Smrtonosne su posljedice nastupile u 1,9% i 0,9% bolesnika liječenih lijekom Yondelis kao jedinim sredstvom, odnosno kombiniranom terapijom.
English: Other information about Yondelis
Croatian: Ostale informacije o lijeku Yondelis
English: It is recommended that Yondelis be given through a central venous line (a thin tube leading from the skin into the large veins just above the heart).
Croatian: Preporučuje se primjena lijeka Yondelis kroz središnju vensku liniju (tanku cijev koja vodi od kože u velike vene neposredno iznad srca).
English: • advanced soft-tissue sarcoma, a type of cancer that develops from the soft, supporting tissues of the body.
Croatian: • uznapredovalim sarkomom mekanog tkiva, tipom raka koji se razvija iz mekanog, potpornog tkiva tijela.
English: Around 10% of those patients treated with Yondelis as a single agent and 25% treated with Yondelis in combination therapy can be expected to have serious side effects.
Croatian: Otprilike u 10% bolesnika liječenih lijekom Yondelis kao samostalnom tvari, te u 25% liječenih Yondelisom u kombiniranoj terapiji mogu se očekivati ozbiljna nuspojave.
English: It should only be used by qualified oncologists (cancer specialists) or other health professionals who are specialised in giving cytotoxic (cell-killing) medicines.
Croatian: Smije se koristiti samo od strane kvalificiranih onkologa (specijalista liječnika za rak) ili drugih zdravstvenih djelatnika koji su specijalizirani za primjenu citotoksika (lijekova koji ubijaju stanice).
English: A risk management plan has been developed to ensure that Yondelis is used as safely as possible.
Croatian: Pripremljen je plan upravljanja rizikom kako bi se osigurala što sigurnija primjena lijeka Yondelis.
English: Based on this plan, safety information has been included in the summary of product characteristics and the package leaflet for Yondelis, including the appropriate precautions to be followed by healthcare professionals and patients.
Croatian: Na temelju tog plana, u sažetku opisa svojstava lijeka kao i u uputi o lijeku za Yondelis nalaze se sigurnosne informacije, uključujući odgovarajuće mjere opreza kojih se zdravstveni djelatnici i bolesnici trebaju pridržavati.
English: The active substance in Yondelis, trabectedin, is a synthetic version of a substance that was originally extracted from a species of tunicate or ‘sea squirt' (a marine animal).
Croatian: Djelatna tvar lijeka Yondelis, trabectedin, je sintetska verzija tvari koja je izvorno ekstrahirana iz vrsta tunicata ili "plaštenjaka" (morska životinja).
English: For soft-tissue sarcoma, the recommended dose of Yondelis is 1.5 mg per square metre of body surface area (calculated using the patient's height and weight), given as a single infusion lasting 24 hours every three weeks.
Croatian: U slučaju sarkoma mekanog tkiva, preporučena doza lijeka Yondelis iznosi 1,5 mg po metru kvadratnom tjelesne površine (izračunato primjenom visine i tjelesne težine bolesnika), a primjenjuje se kao jedna infuzija koja traje 24 sata svaka tri tjedna.
English: Yondelis must be given under the supervision of a doctor who is experienced in the use of chemotherapy (medicines to treat cancer).
Croatian: Yondelis treba primjenjivati uz nadzor liječnika koji je iskusan u primjeni kemoterapije (lijekova za liječenje raka).
English: Because the numbers of patients with soft-tissue sarcoma and ovarian cancer are low, the diseases are considered ‘rare', and Yondelis was designated an ‘orphan medicine' (a medicine used in rare diseases) on 30 May 2001 (for soft-tissue sarcoma) and on 17 October 2003 (for ovarian cancer).
Croatian: Budući da je broj bolesnika sa sarkomom mekanog tkiva i raka jajnika malen, te se bolesti smatraju „rijetkima” te je lijek Yondelis uvršten u skupinu lijekova za rijetke bolesti dana 30. svibnja 2001. (za sarkom mekanog tkiva) i 17. listopada 2003. (za rak jajnika).
English: For ovarian cancer, Yondelis in combination with PLD was compared with PLD alone in one main study involving 672 women whose disease had come back or got worse after previous treatment.
Croatian: U slučaju raka jajnika, Yondelis u kombinaciji s PLD-om uspoređen je s monoterapijom PLD-om u jednom glavnom ispitivanju koje je obuhvatilo 672 žena čija se bolest vratila ili pogoršala nakon prethodne terapije.
English: For soft-tissue sarcoma, Yondelis has been studied in one main study involving 266 patients with liposarcoma (a sarcoma originating in fat cells) or leiomyosarcoma (a sarcoma originating in ‘smooth' or involuntary muscle cells) that was advanced or metastatic (had spread to other parts of the body).
Croatian: U slučaju sarkoma mekanog tkiva, Yondelis je ispitan u jednom glavnom ispitivanju koje je obuhvatilo 266 bolesnika s liposarkomo (sarkomom koji potječe iz stanica masti) ili leiomiosarkomom (sarkomom koji potječe iz "glatkih" ili nedobrovoljnih mišićnih stanica), a uznapredovao je ili metastazirao (raširio se u druge dijelove tijela).
English: The CHMP concluded that Yondelis's benefits are greater than its risks and recommended that it be given marketing authorisation.
Croatian: CHMP je odlučio da koristi od lijeka Yondelis nadmašuju s njim povezane rizike te je preporučio izdavanje odobrenja za stavljanje lijeka u promet.
English: Yondelis is used in combination with pegylated liposomal doxorubicin (PLD, another cancer medicine).
Croatian: Yondelis se koristi u kombinaciji s pegiliranim liposomalnim doksorubicinom (PLDom, drugim antitumorskim lijekom).
English: • soft-tissue sarcoma.
Croatian: • sarkom mekog tkiva.
English: Yondelis is a cancer medicine that contains the active substance trabectedin.
Croatian: Yondelis je lijek protiv karcinoma koji sadrži djelatnu tvar trabectedin.
English: Why has Yondelis been approved?
Croatian: Zašto je Yondelis odobren?
English: What is the risk associated with Yondelis?
Croatian: Koji su rizici povezani s lijekom Yondelis?
English: EPAR summary for the public trabectedin
Croatian: EPAR, sažetak za javnost
English: For ovarian cancer, the combination of Yondelis and PLD was more effective than PLD alone: patients receiving the combination treatment lived for an average of 7.3 months without their disease getting worse, compared with 5.8 months in those receiving PLD alone.
Croatian: U slučaju raka jajnika, kombinacija lijeka Yodelis i PLD-a je bila djelotvornija od primjene samo PLD-a: bolesnici koji su primali kombiniranu terapiju živjeli su prosječno 7,3 mjeseci bez pogoršanja bolesti, u usporedbi s 5,8 mjeseca u onih koji su primali monoterapiju PLD-om. djelovanje lijeka Yodelis bilo je izraženije u žena čiji je rak bio osjetljiv na lijekove koji su sadržavali platinu.
English: What is Yondelis?
Croatian: Što je Yondelis?
English: For more information, see the summary of product characteristics (also part of the EPAR).
Croatian: Dodatne informacije pročitajte u sažetku opisa svojstava lijeka (također dio EPAR-a).
English: Most patients treated with Yondelis can be expected to have side effects.
Croatian: Nuspojave se mogu očekivati u većine bolesnika liječenih lijekom Yondelis.
English: What is Yondelis used for?
Croatian: Za što se Yondelis koristi?
English: The full EPAR for Yondelis can be found on the Agency's website: ema.europa.eu/Find medicine/Human medicines/European public assessment reports.
Croatian: Cjeloviti EPAR za lijek Yondelis nalazi se na internetskim stranicama Agencije: ema.europa.eu/Find medicine/Human medicines/European public assessment reports Više informacija o terapiji lijekom Yondelis pročitajte u uputi o lijeku (također dio EPAR-a) ili se obratite svom liječniku ili ljekarniku.
English: For the full list of all side effects reported with Yondelis, see the package leaflet.
Croatian: Potpuni popis nuspojava zabilježenih pri primjeni lijeka Yondelis potražite u uputi o lijeku.
English: What benefit has Yondelis shown during the studies?
Croatian: Koje su koristi lijeka Yondelis utvrđene u ispitivanjima?
English: How is Yondelis used?
Croatian: Kako se Yondelis koristi?
English: How has Yondelis been studied?
Croatian: Kako je Yondelis ispitivan?
English: How does Yondelis work?
Croatian: Kako djeluje Yondelis?
English: The recommended starting dose is 1 capsule a day.
Croatian: Preporučena početna doza lijeka je 1 kapsula na dan.
English: The dose can be increased up to 3 capsules a day, depending on the intensity of the symptoms and how the patient responds to treatment.
Croatian: Doza se može povećati do 3 kapsule na dan, ovisno o intenzitetu simptoma i o tome kako bolesnik reagira na liječenje.
English: Namuscla (mexiletine)
Croatian: Namuscla (meksiletin)
English: What measures are being taken to ensure the safe and effective use of Namuscla?
Croatian: Koje se mjere poduzimaju kako bi se osigurala sigurna i učinkovita primjena lijeka Namuscla?
English: Further information on the orphan designation can be found here: ema.europa.eu/medicines/human/orphan-designations/EU3141353.
Croatian: Više informacija o statusu lijeka za rijetke bolesti možete naći ovdje: ema.europa.eu/medicines/human/orphan-designations/EU3141353.
English: Why is Namuscla authorised in the EU?
Croatian: Zašto je Namuscla odobren u EU-u?
English: Because it has a similar effect on the heart muscle, mexiletine has also been used for many years in patients with abnormal heart rhythm.
Croatian: Budući da ima sličan učinak na srčani mišić, meksiletin se godinama upotrebljava u bolesnika s abnormalnim srčanim ritmom.
English: Further information on Namuscla can be found on the Agency's website: ema.europa.eu/medicines/human/EPAR/Namuscla.
Croatian: Više informacija o lijeku Namuscla nalazi se na internetskim stranicama Agencije: ema.europa.eu/medicines/human/EPAR/Namuscla.
English: After 18 days of treatment, the average score for patients treated with Namuscla decreased from 66 to 24, while the score for patients given placebo went from 75 to 66.
Croatian: Nakon 18 dana liječenja prosječna ocjena za bolesnike koji su primali lijek Namuscla smanjila se sa 66 na 24, a ocjena za bolesnike koji su primali placebo smanjila se sa 75 na
English: By blocking them, the medicine helps to reduce the stiffness that occurs when the contractions are prolonged.
Croatian: Njihovom blokadom lijek pomaže u smanjenju ukočenosti koja nastupa kada se kontrakcije produže.
English: The company that markets Namuscla will provide educational material for doctors and a ‘patient alert card' with important information about the risks associated with the medicine, in particular the risk of arrhythmias.
Croatian: Tvrtka koja lijek Namuscla stavlja u promet dostavit će edukacijske materijale za liječnike i „karticu s upozorenjima za bolesnike” s važnim informacijama o rizicima povezanima s lijekom, osobito o riziku od pojave aritmija.
English: The company also provided supportive data on the effectiveness of Namuscla from the literature.
Croatian: Tvrtka je dostavila i popratne podatke o učinkovitosti lijeka Namuscla iz literature.
English: Patients should have tests to check how well their heart is working before beginning treatment and regularly during treatment.
Croatian: Bolesnici bi prije početka liječenja trebali napraviti pretrage za provjeru funkcije srca i obavljati ih redovito tijekom samog liječenja.
English: The most serious side effects reported (which may affect up to 1 in 10,000 people) are arrhythmias (disturbances of heart rhythm) and a severe reaction affecting skin, blood and internal organs, known as drug reaction with eosinophilia and systemic symptoms (DRESS).
Croatian: Najteže prijavljene nuspojave (koje se mogu javiti u više od 1 u 10 000 osoba) jesu aritmije (poremećaji srčanog ritma) i teška reakcija koja zahvaća kožu, krv i unutarnje organe, poznata kao reakcija na lijek s eozinofilijom i sistemskim simptomima (DRESS).
English: Myotonic disorders are rare, and Namuscla was designated an ‘orphan medicine' (a medicine used in rare diseases) on 19 November 2014.
Croatian: Poremećaji miotonije su rijetki, a lijek Namuscla dobio je status „lijeka za rijetku bolest” 19. studenoga 2014.