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32 Result(s)
Substance(s)
- (S)-6-hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamide hydrochloride
- 18-mer antisense oligonucleotide complementary to OPA1 pre-mRNA
- 2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione
- 4,5-dihydro-2-(2,4-dihydroxyphenyl)-4-methylthiazole-4(S)-carboxylic acid
- Adeno-associated viral vector containing the human NADH dehydrogenase 4 gene (rAAV2/2-ND4)
- Adeno-associated viral vector serotype 9 containing the human SURF1 gene
- Adeno-associated virus vector serotype 2 expressing the human MT-ND4 codon-optimised gene
- Adenoassociated virus vector (AAV) carrying a modified AAV serotype 2 backbone and coding sequence of human thymidine phosphorylase preceded by a human thyroxin-binding globulin promoter
- Autologous CD34+ cells edited with CRISPR/Cas9 and transduced with an adeno-associated virus vector serotype 6 containing the codon-optimized version of PKLR gene
- Brivaracetam
- Cyclocreatine
- Deferiprone
- Dodecyl creatine ester, dodecyl creatine ester hydrochloride
- Elamipretide
- L carnitine
- Lentiviral vector containing the human liver and erythroid pyruvate kinase (PKLR) gene
- Mitapivat sulfate
- Recombinant adeno-associated virus serotype 2 containing human mitochondrial ND1 codon optimized gene (rAAV2-ND1)
- Recombinant thymidine phosphorylase encapsulated in autologous erythrocytes
- Sodium (4-{(E)-3-(4-fluorophenyl)-3-[4-(3-morpholin-4-yl-prop1ynyl)phenyl]allyloxy}-2-methylphenoxy)acetate
- Sodium phenylbutyrate
- Thymidine and deoxycytidine
- Triheptanoin
- Ubiquinol
- Vatiquinone
- Vector based on an adeno-associated virus serotype 2 backbone, pseudo-serotyped with a type 8 capsid, which carries the coding sequence of the human TYMP gene under the control of the human thyroxine binding globulin promoter
- idebenone