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(+)-Epicatechin (1E,6E)-1,7-bis(3,4-dimethoxyphenyl)-4-cyclobutylmethyl-1,6-heptadiene-3,5-dione (6aS)-1,10-dimethoxy-6-methyl-5,6,6a,7-tetrahydro-4H-dibenzo[de,g]quinoline-2,9-diol (S)-6-idrossi-2,5,7,8-tetrametil-N-((R)-piperidina-3-il)croman-2-carbossamide cloridrato 1,5-(Butylimino)-1,5-dideoxy, D-glucitol 1-deoxynojirimycin hydrochloride 17alpha,21-diidrossi-16alpha-metil-pregna-1,4,9(11)-triene-3,20-dione 2'-O-metil-fosforotioato oligonucleotide 2-(4-(diethylamino) phenyl)-6-methyl-2H-benzo[d][1,2,3] triazol-5-amine 2-isopropyl-3H-naphtho[1,2-d]imidazole-4,5-dione 20- hydroxyecdysone 5'-cEtG-sp-cEt5MeU-sp-cEt5MeU-sp-dT-sp-dA-sp-dT-sp-dT-sp-dA-sp-dT-sp-dA-sp-dG-sp-dG-sp-dG-sp-cEt5MeC-sp-cEt5MeU-sp-cEt5MeU-3' 5-(ethylsulfonyl)-2-(naphthalen-2-yl)benzo[d]oxazole 5-[1-(2,6-diclorobenzil)piperidina-4-ilmetossi] quinazolin-2,4-diamin dicloridrato 505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase Acido sialico Adeno associated viral vector containing the human calpain 3 gene Adeno-associated viral vector containing a modified U7-snRNA gene Adeno-associated viral vector containing the human alpha-sarcoglycan gene Adeno-associated viral vector serotype 8 containing the human MTM1 gene Adeno-associated viral vector serotype 8 containing the human acid alpha-glucosidase gene Adeno-associated viral vector serotype 9 containing the human SMN gene Adeno-associated viral vector serotype 9 containing the human mini-dystrophin gene Adeno-associated viral vector serotype hu68 containing the human SMN1 gene Adeno-associated virus serotype 9 expressing the human Survival Motor Neuron gene Adeno-associated virus serotype rh74 containing the human micro-dystrophin 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 Alfa glucosidasi acida umana ricombinante GILT-marcata Alfa-glucosidasi umana ricombinante coniugata con copie multiple di bis-mannosio-6-fosfato-tetra mannosio glicano di sintesi Alglucosidase alfa Alofuginone idrobromuro Alpha-tocopherol Anticorpo monoclonale umanizzato anti-miostatina Ascorbic acid Asp-Arg-Val-Tyr-Ile-His-Pro Ataluren Brivaracetam Cellule progenitrici motoneuronali allogeniche derivate da cellule staminali embrionali umane Clenbuterol Colesten-3-one, ossima Dantrolene sodium Eteplirsen Ex vivo fused normal allogeneic human myoblast with another normal allogeneic human myoblast Ex vivo fused normal allogeneic human myoblast with autologous human myoblast derived from Duchenne muscular dystrophy affected donor Ex-vivo fused autologous human bone marrow-derived mesenchymal stem cell with allogenic human myoblast Exon 55 specific phosphorothioate oligonucleotide Fenilbutirrato di sodio Givinostat Gliceril-tri-(4-fenilbutirrato) Golodirsen Human anti-promyostatin monoclonal antibody Idebenone Kifunensine L carnitine L-selenomethionine LMI 070 Late Stage Human Motor Neutron Progenitors Losmapimod Metformin and L-citrulline Mexiletina cloridrato N-(2-((4Z,7Z,10Z,13Z,16Z,19Z)-docosa-4,7,10,13,16,19-hexaenamido)ethyl)-2-hydroxybenzamide N-acetyl-D-mannosamine monohydrate Naproxcinod Oligonucleotide antisenso mirato al gene SMN2 Oligonucleotide fosforotioato specifico per l'esone 44 Oligonucleotide fosforotioato specifico per l'esone 45 Oligonucleotide fosforotioato specifico per l'esone 51 Oligonucleotide fosforotioato specifico per l'esone 53 Oligonucleotide fosforotioato specifico per l'esone 55 Omigapil maleate Pamrevlumab Proteina ricombinante di fusione costituita dalla porzione extracellulare del recettore dell'activina umana di tipo IIB legata al dominio FC di IgG1 umana R, S-O-(3-piperidino-2-idrossi-1-propil) - acido nicotinico dicloridrato amidoxime RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a-->5') (C-m5U-C-C-A-A-C-A-m5U-C-A-A-G-G-A-A-G-A-m5U-G-G-C-A-m5U-m5U-m5U-C-m5U-A-G), P-[4[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl] N,N-dimethylaminophosphonamidate RNA, [P-deoxy-P-(dimethylamino)] (2',3'-dideoxy-2',3'-imino-2',3'-seco) (2'a-->5') (C-m5U-m5U-A-C-A-G-G-C-m5U-C-C-A-A-m5U-A-G-m5U-G-G-m5U-C-A-G-m5U), 5' [P-[4-[[2-[2-(2-hydroxyethoxy)ethoxy]ethoxy]carbonyl]-1-piperazinyl]-N,N-dimethylaminophosphonamidate], 3'-[2'a-[------], octahydrochloride Rasagiline Recombinant adeno-associated viral vector containing a bioengineered capsid serotype AAV-rh74 and a codon-optimised expression cassette to drive the expression of a secretable form of human acid alpha-glucosidase Recombinant adeno-associated viral vector containing human acid alfa-glucosidase-gene Recombinant adeno-associated viral vector encoding a human micro-dystrophin gene under the control of a muscle specific promoter Recombinant human acid alpha-glucosidase Recombinant human acid alpha-glucosidase conjugated with mannose-6-phosphate analogues Reldesemtiv Risdiplam Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA Tamoxifen citrate Thymidine and deoxycytidine Timidina fosforilasi ricombinante incapsulata in eritrociti autologhi Trehalose Trieptanoina Valproato di sodio Valproic acid, sodium Vettore basato sulla struttura di un virus adeno-associato sierotipo 2, pseudo-sierotipizzato con un capside di tipo 8, contenente la sequenza codificante del gene umano TYMP controllato dal promotore umano di globulina che lega la tiroxina Vettore derivato da virus adeno-associati contenente il gene del gamma-sarcoglicano umano Vettore virale contenente il DNA codificante per la proteina umana SMN Vettori virali associati all'adenovirus contenenti la versione modificata del gene U1-snRNA Viltolarsen Zinc gluconat deflazacort genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1 p38 mitogen-activated kinase inhibitor rimeporide tetracosactide
