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(+)-Epicatechin (1E,6E)-1,7-bis(3,4-dimethoxyphenyl)-4-cyclobutylmethyl-1,6-heptadiene-3,5-dione (6R)-4, 5, 6, 7-tetrahydro-N6-propyl-2, 6-benzothiazolediamine dihydrochloride monohydrate (6aS)-1,10-dimethoxy-6-methyl-5,6,6a,7-tetrahydro-4H-dibenzo[de,g]quinoline-2,9-diol (R)-troloxamide quinone (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-[(2-Chloro-4-methoxyphenoxy)methyl]-4-[(2,6-dichlorophenoxy)methyl]benzene 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 3,4-diaminopyridine phosphate 5'-CTG CCA CGT TCT CCT GC-(2' methoxy)A-(2' methoxy)C-(2' methoxy)C-3' 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 6-etinil-1-(pentan-3-il)-1H-imidazo[4,5-b]pirazina-2(3H)-one Lactobacillus plantarum Acetazolamide 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 Ambroxol hydrochloride Anticorpo monoclonale umanizzato anti-miostatina Anticorpo monoclonale umanizzato ricombinante della classe IgG1/kappa anti proteina umana Nogo-A Arimoclomol Arimoclomol citrate Ascorbic acid Asp-Arg-Val-Tyr-Ile-His-Pro Ataluren Autologous Olfactory Neural Progenitors Autologous adipose derived mesenchymal stromal cells Brivaracetam Cellule progenitrici motoneuronali allogeniche derivate da cellule staminali embrionali umane Clenbuterol Clorito di sodio Colesten-3-one, ossima DNA plasmid vector (pCK-HGFX7) expressing human hepatocyte growth factor Dantrolene sodium Diclofenamide Eculizumab Edaravone Efgartigimod alfa Enoxacin 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 Fattore di crescita endoteliale ricombinante umano Fenilbutirrato di sodio Filgrastim Fusion proteins composed by a genetically modified cholera toxin subunit A1, peptides from the acetylcholine receptor alpha chain and a dimer of the D fragment from Staphylococcus aureus protein A Givinostat Gliceril-tri-(4-fenilbutirrato) Golodirsen H-Leu-Pro-Pro-Leu-Pro-Tyr-Pro-OH H-Val-Ile-Val-Lys-Leu-Ile-Pro-Ser-Thr-Ser-Ser-Ala-Val-Asp-Thr-Pro-Tyr-Leu-Asp-Ile-Thr-Tyr-His-Phe-Val-Ala-Gln-Arg-Leu-Pro-Leu-OH Human anti-promyostatin monoclonal antibody Human culture expanded autologous mesenchymal stromal cells Human normal immunoglobulin Hydrocinnamate-[Orn-Pro-dCha-Trp-Arg]acetate Ibudilast Idebenone Ifosfamide Kifunensine L carnitine L-selenomethionine LMI 070 Late Stage Human Motor Neutron Progenitors Levosimendan Losmapimod Masitinib mesilato Metformin and L-citrulline Methotrexate Mexiletina cloridrato Monarsen 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 Oligopeptide containing 6 amino acids (H-Phe-Ser-Arg-Tyr-Ala-Arg-OH) Omigapil maleate Osseo autologo derivate dal midollo mesenchimali stromali cellule che secernono fattori neurotrofici Pamrevlumab Peptidomimetici del recettore per l'antigene di cellule B e T autoimmuni associate a miastenia grave 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 Human Alpha-Fetoprotein (Rhafp) 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 Recombinant human antibody directed against misfolded human superoxide dismutase 1 Recombinant human cerebral dopamine neurotrophic factor (CDNF) Recombinant human monoclonal antibody against activin receptor type IIB Reldesemtiv Resunab Riluzole Risdiplam Rozanolixizumab S[+] apomorfina Sarsapogenin Smilagenin Sodium phenylbutyrate, tauroursodeoxycholic acid Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA Synthetic ribonucleic acid oligonucleotide directed against superoxide dismutase 1 (SOD-1) messenger ribonucleic acid Talampanel Tamoxifen citrate Tauroursodeoxycholic acid (TUDCA) Thymidine and deoxycytidine Timidina fosforilasi ricombinante incapsulata in eritrociti autologhi Tiosolfato di sodio 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 Xaliproden hydrochloride Zinc gluconat adeno-associated viral vector serotype 9 encoding miRNA against human superoxide dismutase 1 caprine hyperimmune serum against HIV lysate deflazacort genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1 recombinant mutated extracellular domain of the human acetylcholine receptor subunit alpha1 rimeporide siponimob tetracosactide
