Vyhledat léčivý přípravek pro vzácné onemocnění
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93 Výsledků
Účinné látky
- (1E,6E)-1,7-bis(3,4-dimethoxyphenyl)-4-cyclobutylmethyl-1,6-heptadiene-3,5-dione
- (R)-2-amino-N-((S)-1-(((S)-5-amino-1-(3-benzyl-1,2,4-oxadiazol-5-yl)pentyl)amino)-3-(4-hydroxy-2,6-dimethylphenyl)-1-oxopropan-2-yl)-5-guanidinopentanamide, tris hydrochloride
- (R)-troloxamide quinone
- 1-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy)methyl]benzene
- 2,3,5-Trimethyl-6-nonyl-2,5-cyclohexadiene-1,4-dione
- 2-(4-Tert-butylphenyl)-1H-benzimidazole
- 4,4',4''-(Ethane-1,1,1- triyl)tris(2,6-diethoxyphenol)
- 5-((S)-1-(3,5-bis(trifluoromethyl)phenoxy)ethyl)-3-hydroxycyclohex-2-en-1-one
- 5-[1-(2,6-dichlorobenzyl)piperidin-4-ylmethoxy]quinazoline-2,4-diamine dihydrochloride
- Lactobacillus plantarum
- AAV9-Synapsin-Caveolin-1
- Adeno-associated viral vector serotype hu68 containing the human SMN1 gene
- Adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene
- Aldesleukin
- Allogeneic motor neuron progenitor cells derived from human embryonic stem cells
- Ambroxol hydrochloride
- Anti-CD38 IgG4 human monoclonal antibody
- Apitegromab
- Arimoclomol
- Autologous adipose derived mesenchymal stromal cells
- Autologous bone marrow-derived mesenchymal stromal cells secreting neurotrophic factors
- Autologous olfactory neural progenitors
- Brivaracetam
- Caprine hyperimmune serum against HIV lysate
- Celecoxib , Ciprofloxacin
- Crisdesalazine
- D-lactic acid, glycolic acid
- DNA plasmid vector (pCK-HGFX7) expressing human hepatocyte growth factor
- Dexpramipexole
- Edaravone
- Enoxacin
- Fasudil hydrochloride
- Filgrastim
- Ganglioside GM1
- Glycerol phenylbutyrate
- Gold
- H-L-tryphophanyl-L-seryl-glycyl-L-tryptophanyl-L-seryl-L-seryl-L-cysteinyl-L-seryl-L-arginyl-L-seryl-L-cysteinyl-glycyl-OH (disulfide bond)
- H-Leu-Pro-Pro-Leu-Pro-Tyr-Pro-OH
- Human culture expanded autologous mesenchymal stromal cells
- Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate
- Ibudilast
- Icerguastat acetate
- Insulin-like Growth Factor-1
- L-pyroglutamyl-L-asparaginyl-L-prolyl-D-tyrosyl-D-tryptophan amide
- Late Stage Human Motor Neutron Progenitors
- Levosimendan
- Low molecular weight dextran sulfate
- Masitinib mesilate
- Nusinersen
- Olesoxime
- Oligopeptide containing 6 amino acids (H-Phe-Ser-Arg-Tyr-Ala-Arg-OH)
- Onasemnogene abeparvovec
- Oxygen, sodium chloride solution 0.9%
- Pegcetacoplan
- Pridopidine hydrochloride
- Recombinant human antibody directed against misfolded human superoxide dismutase 1
- Recombinant human cerebral dopamine neurotrophic factor (CDNF)
- Recombinant human vascular endothelial growth factor
- Recombinant humanised monoclonal antibody to human Nogo-A protein of the IgG1/kappa class
- Reldesemtiv
- Riluzole
- Risdiplam
- S[+] apomorphine
- Sarsasapogenin
- Smilagenin
- Sodium chlorite
- Sodium phenylbutyrate
- Sodium phenylbutyrate, tauroursodeoxycholic acid
- Sodium valproate
- Sotuletinib
- Talampanel
- Talditercept alfa
- Tauroursodeoxycholic acid (TUDCA)
- Tirasemtiv
- Tofersen
- Trehalose
- Utreloxastat
- Viral vector containing DNA encoding the human SMN protein
- Xaliproden hydrochloride
- adeno-associated viral vector serotype 9 encoding miRNA against human superoxide dismutase 1
- branaplam
- siRNA oligonucleotide designed to target and knockdown the expression of SOD1