L\u00e9k Zolgensma na spin\u00e1ln\u00ed svalovou atrofii obdr\u017eel od Evropsk\u00e9 komise (EK) podm\u00edne\u010dnou registraci, kter\u00e1 je podm\u00ednkou standardn\u00edho pod\u00e1v\u00e1n\u00ed l\u00e9ku pacient\u016fm v Evropsk\u00e9 unii. Podle St\u00e1tn\u00edho \u00fastavu pro kontrolu l\u00e9\u010div (S\u00daKL) automaticky neznamen\u00e1, \u017ee bude tato genov\u00e1 terapie ihned dostupn\u00e1 i v \u010cesku.
Informoval o tom v tiskov\u00e9 zpr\u00e1v\u011b. V \u010cesku byl l\u00e9k na z\u00e1klad\u011b mimo\u0159\u00e1dn\u00e9ho rozhodnut\u00ed ministerstva zdravotnictv\u00ed schv\u00e1len pro pod\u00e1n\u00ed t\u0159em d\u011btem, prvn\u00ed ho dostalo minul\u00fd t\u00fdden.<\/p>\n\n\n\n
„To, kdy a kam jej za\u010dne dr\u017eitel rozhodnut\u00ed o registraci dod\u00e1vat, je \u010dist\u011b na jeho rozhodnut\u00ed. St\u00e1tn\u00ed \u00fastav pro kontrolu l\u00e9\u010div obecn\u011b na takov\u00e1 obchodn\u00ed rozhodnut\u00ed soukrom\u00fdch subjekt\u016f nem\u00e1 \u017e\u00e1dn\u00fd vliv,“ uvedla \u0159editelka S\u00daKL Irena Storov\u00e1. Podle n\u00ed se ale t\u00edmto rozhodnut\u00edm p\u0159\u00edpravek pro \u010desk\u00e9 pacienty st\u00e1v\u00e1 dostupn\u011bj\u0161\u00edm ne\u017e doposud.
Podm\u00edne\u010dnou registraci EK ud\u011bluje v p\u0159\u00edpad\u011b, \u017ee u l\u00e9\u010div\u00e9ho p\u0159\u00edpravku p\u0159eva\u017euj\u00ed p\u0159\u00ednosy nad riziky a je \u017e\u00e1douc\u00ed, aby byl l\u00e9\u010div\u00fd p\u0159\u00edpravek dostupn\u00fd na trhu. Mus\u00ed j\u00edt z\u00e1rove\u0148 o l\u00e9k, k n\u011bmu\u017e nen\u00ed alternativa. Registrace plat\u00ed rok a prodlou\u017eena m\u016f\u017ee b\u00fdt, jen kdy\u017e v\u00fdrobce p\u0159edlo\u017e\u00ed dal\u0161\u00ed data.<\/p>\n\n\n\n
„S l\u00e9\u010dbou pacient\u016f star\u0161\u00edch dvou let nebo s hmotnost\u00ed nad 13,5 kilogramu jsou jen omezen\u00e9 zku\u0161enosti. Bezpe\u010dnost a \u00fa\u010dinnost tohoto p\u0159\u00edpravku u takov\u00fdch pacient\u016f nebyla dosud stanovena,“ vysv\u011btlila Storov\u00e1.<\/p>\n\n\n\n
\u010cesk\u00e9 ministerstvo zdravotnictv\u00ed povolilo pou\u017eit\u00ed l\u00e9ku na z\u00e1klad\u011b takzvan\u00e9ho specifick\u00e9ho l\u00e9\u010debn\u00e9ho programu. Prvn\u00ed ze t\u0159\u00ed d\u011bt\u00ed tuto genovou terapii dostalo ve Fakultn\u00ed nemocnici v Motole minul\u00fd t\u00fdden. Zda bude m\u00edt o\u010dek\u00e1van\u00fd efekt, se uk\u00e1\u017ee zhruba do t\u0159\u00ed m\u011bs\u00edc\u016f.
L\u00e9\u010dbu v\u0161ech t\u0159\u00ed pacient\u016f uhrad\u00ed zdravotn\u00ed poji\u0161\u0165ovny, pro jednoho stoj\u00ed 54 milion\u016f korun, podle motolsk\u00e9 nemocnice jde o nejdra\u017e\u0161\u00ed jednor\u00e1zov\u00fd l\u00e9k, kter\u00fd podala. M\u011bl by jednor\u00e1zov\u011b opravit genovou poruchu, kter\u00e1 nemoc zp\u016fsobuje.<\/p>\n\n\n\n
St\u00e1t \u017e\u00e1dal da\u0148 z vybran\u00fdch pen\u011bz na l\u00e9\u010dbu d\u011bt\u00ed<\/p>\n\n\n\n
P\u016fvodn\u011b rodi\u010de vyb\u00edrali na l\u00e9\u010dbu sv\u00fdch d\u011bt\u00ed ve ve\u0159ejn\u00e9 sb\u00edrce, proto\u017ee poji\u0161\u0165ovny l\u00e9\u010dbu hradit odm\u00edtly. St\u00e1t po rodin\u011b, kter\u00e9 se poda\u0159ilo vybrat celou cenu, \u017e\u00e1dal zaplacen\u00ed dan\u011b z vybran\u00fdch pen\u011bz, zhruba 5,5 milionu korun.<\/p>\n\n\n\n
Spin\u00e1ln\u00ed svalov\u00e1 atrofie je vrozen\u00e9 onemocn\u011bn\u00ed, p\u0159i kter\u00e9m postupn\u011b ub\u00fdv\u00e1 svalstvo, a t\u00edm se zhor\u0161uje schopnost pohybu. Posti\u017een\u00e9 b\u00fdvaj\u00ed hlavn\u011b nohy, nemocn\u00ed ale tak\u00e9 h\u016f\u0159e polykaj\u00ed a pozd\u011bji maj\u00ed pot\u00ed\u017ee i s d\u00fdch\u00e1n\u00edm. Postup nemoci je individu\u00e1ln\u00ed, pacienti se ale v\u011bt\u0161inou nedo\u017e\u00edvaj\u00ed vysok\u00e9ho v\u011bku.<\/p>\n\n\n\n
den\u00edk.cz<\/p>\n\n\n\n
https:\/\/www.denik.cz\/<\/a> L\u00e9k Zolgensma na spin\u00e1ln\u00ed svalovou atrofii obdr\u017eel od Evropsk\u00e9 komise (EK) podm\u00edne\u010dnou registraci, kter\u00e1 je podm\u00ednkou standardn\u00edho pod\u00e1v\u00e1n\u00ed l\u00e9ku pacient\u016fm v Evropsk\u00e9 unii. Podle St\u00e1tn\u00edho […]<\/a><\/p>\n<\/div>","protected":false},"author":1,"featured_media":15438,"comment_status":"open","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[9],"tags":[],"class_list":["post-34424","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-zpravy"],"_links":{"self":[{"href":"https:\/\/www.invarena.cz\/index.php?rest_route=\/wp\/v2\/posts\/34424","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/www.invarena.cz\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/www.invarena.cz\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/www.invarena.cz\/index.php?rest_route=\/wp\/v2\/users\/1"}],"replies":[{"embeddable":true,"href":"https:\/\/www.invarena.cz\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=34424"}],"version-history":[{"count":0,"href":"https:\/\/www.invarena.cz\/index.php?rest_route=\/wp\/v2\/posts\/34424\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/www.invarena.cz\/index.php?rest_route=\/wp\/v2\/media\/15438"}],"wp:attachment":[{"href":"https:\/\/www.invarena.cz\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=34424"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/www.invarena.cz\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=34424"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/www.invarena.cz\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=34424"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}
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