Vyhledat léčivý přípravek pro vzácné onemocnění
Další možnosti vyhledávání
45 Výsledků
Účinné látky
- (1S,3S)-3-amino-4-(difluoromethylene) cyclopentanecarboxylic acid hydrochloride
- (5R)-5-ethyl-3-[2-(7-methylspiro[2H-benzofuran-3,1'-cyclopropane]-4-yl)oxypyrimidin-5-yl]imidazolidine-2,4-dione
- 18-mer antisense oligonucleotide complementary to SCN1A mRNA, sodium salt
- 2'-O-(2-methoxyethyl) modified antisense oligonucleotide targeting glycogen synthase 1 pre-mRNA
- 26 base synthetic single-stranded fully phosphorothioated 2'-O-methyl-RNA and DNA mixmer oligonucleotide-based compound
- 3-(ethoxydifluoromethyl)-6-(5-fluoro-6-(2,2,2-trifluoroethoxy)pyridin-3-yl)-[1,2,4]triazolo[4,3-a]pyrazine
- 5'-moemC-(sp)-moemC-(p)-moeA-(p)-moemC-(p)-moeG-(p)-moeA-(p)-dmC-(sp)-dA-(sp)-dT-(sp)-dA-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dT-(sp)-dmC-(sp)-moeT-(p)-moeA-(sp)-moemC-(sp)-moeA 3'
- Adeno-associated viral vector serotype 9 containing the human SLC13A5 gene
- Adeno-associated virus serotype 9 expressing a transcription factor for the SCN1A gene
- Allopregnanolone
- Brivaracetam
- Cannabidiol
- Clemizole hydrochloride
- Felbamate
- Fenfluramine hydrochloride
- Flunarizine
- Ganaxolone
- Ketamine
- Lamotrigine
- Lorcaserin
- Metformin
- N-[1-((5-cyanopyridin-2-yl)methyl)-1H-pyrazol- 3-yl]-2-[4-(1-(trifluoromethyl)cyclopropyl) phenyl]acetamide
- Recombinant human tripeptidyl-peptidase 1
- Rufinamide
- Small molecule agonist of 5-hydroxytryptamine serotonergic receptors
- Soticlestat
- Stiripentol
- Tricaprilin
- Vigabatrin