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69 Result(s)
Substance(s)
- 1-(4-(6-chloropyridazin-3-yl)piperazin-1-yl)-2-(4-cyclopropyl-3-fluorophenyl)ethan-1-one
- Adeno-associated viral vector 9 delivering human Glutaryl-CoA Dehydrogenase (GCDH) gene
- Adeno-associated viral vector LK03 encoding human methylmalonyl-CoA mutase
- Adeno-associated viral vector serotype 8 encoding human ornithine transcarbamylase
- Adeno-associated viral vector serotype LK03 encoding human ornithine transcarbamylase
- Adeno-associated virus serotype 9 containing the human ASPA gene
- Adeno-associated virus serotype HSC15 expressing human phenylalanine hydroxylase
- Adeno-associated virus serotype HSC15, containing human homology arms, expressing human phenylalanine hydroxylase
- Adeno-associated virus serotype rh79 containing the human OTC gene, adeno-associated virus serotype rh79 encoding a meganuclease for targeted editing of the human PCSK9 gene
- Allogeneic adult liver-derived stem cells
- Betaine anhydrous
- Carglumic acid
- Codon -optimised human ornithine transcarbamylase mRNA complexed with lipid -based nanoparticle
- Elamipretide
- Escherichia coli, strain Nissle 1917, expressing high affinity phenylalanine transporter, phenylalanine ammonia lyase and L-amino acid deaminase
- Fosdenopterin
- Glycerol phenylbutyrate
- Glycine, L-alanine, L-arginine, L-aspartic acid, L -cysteine, L-cystine, L- glutamic acid, L-histidine, L-lysine monohydrate, L -methionine, L- phenylalanine, L-proline, L-serine, L-threonine, L-tryptophan, L-tyrosine, taurine
- Heterologous human adult liver derived stem cells
- Heterologous human adult liver-derived progenitor cells
- Human heterologous liver cells (for infusion)
- L-cystine bis(N-methylpiperazide)
- L-ergothioneine
- Live biotherapeutic consisting of metP and metDC genes which are incorporated into the genome of a probiotic strain of E. coli Nissie 1917
- Methotrexate
- Modified mRNA encoding human methylmalonyl-coenzyme A mutase containing a polymorphism at position 671
- Modified mRNA encoding human methylmalonyl-coenzyme A mutase encapsulated into lipid nanoparticles
- Modified messenger ribonucleic acid encoding human argininosuccinate lyase enzyme encapsulated into lipid nanoparticles
- Modified messenger ribonucleic acid encoding human ornithine transcarbamylase enzyme encapsulated into lipid nanoparticles
- Modified messenger ribonucleic acid encoding human propionyl-coenzyme A carboxylase alpha and beta subunits encapsulated into lipid nanoparticle
- Modified version of a leucine decarboxylase enzyme from Planctomycetaceae bacterium
- Nitisinone
- Pegylated human recombinant arginase I
- Pegylated recombinant phenylalanine ammonia lyase
- Phenylalanine ammonia lyase
- Poly(oxy-1,2-ethanediyl), alpha-(carboxymethyl)-omega-methoxy-, amide with cystathionine gamma-lyase (Pyridoxal 5'-phosphate cofactor) (synthetic engineered human), tetramer
- Recombinant adeno-associated viral vector serotype HSC15, containing homology arms targeting the human phenylalanine hydroxylase genomic locus, and expressing human phenylalanine hydroxylase
- Recombinant adeno-associated virus Olig001 containing human aspartoacylase cDNA
- Recombinant adeno-associated virus containing human ASPA cDNA (rAAV-Olig001-ASPA)
- Recombinant adeno-associated virus serotype 8 vector encoding human phenylalanine hydroxylase (PAH)
- Sapropterin
- Sepiapterin
- Sodium benzoate
- Sodium phenylbutyrate
- Tetrahydrobiopterin
- Tiopronine
- Tripotassium citrate monohydrate and potassium hydrogen carbonate
- adeno-associated virus vector encoding human phenylalanine hydroxylase
- mRNA encoding modified human ornithine transcarbamylase
- pegzilarginase
- polyethylene glycol-modified human recombinant truncated cystathionine beta-synthase
- sodium phenylacetate / sodium benzoate