Wirkstoff(e)

• 'Glykosylierungsunabhängiges lysosomales targeting'-getaggte rekombinante humane lyosomale alpha-Glucosidase
• (+)-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-Hydroxy-2,5,7,8-tetramethyl-N-((R)-piperidin-3-yl)chroman-2-carboxamidhydrochlorid
• 1,5-(Butylimino)-1,5-dideoxy, D-glucitol
• 1-deoxynojirimycin hydrochloride
• 17alpha,21-Dihydroxy-16alpha-methyl-pregna-1,4,9(11)-trien-3,20-dion
• 2'-O-methyl-phosphorothioate Oligonucleotid
• 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-Dichlorobenzyl)piperidin-4-ylmethoxy]Quinazolin-2,4-Diamin-Dihydrochlorid
• 505 amino acid protein, corresponding to amino acids 2-506 of the wild type human histidyl-tRNA synthetase
• 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
• Adeno-assoziierte virale Vektoren, welche ein modifiziertes U1-snRNA Gen enthalten
• Adeno-assoziierter viraler vektor, der das human gamma-sarkoglykan-gen enthält
• 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
• Alglucosidase alfa
• Allogene Progenitorzellen für Motorneurone, abgeleitet von menschlichen embryonalen Stammzellen
• Alpha-tocopherol
• Antisense-Oligonukleotid gegen das SMN2-Gen
• Ascorbic acid
• Asp-Arg-Val-Tyr-Ile-His-Pro
• Ataluren
• Auf einem Adeno-assoziierten Virus serotyp 2 Plasmid basierender Vektor mit pseudo-serotyp Typ 8 Kapsid, der unter der Kontrolle des menschlichen Thyroxin-bindenden Globulin Promoters fuer das menschliche TYMP Gen kodiert
• Brivaracetam
• Cholest-4-in-3-one, Oxime
• Clenbuterol
• 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
• Givinostat
• Glyceryl-tri-4-phenylbutyrat
• Golodirsen
• Halofuginon hydrobromid
• Human anti-promyostatin monoclonal antibody
• Humanes rekombinantes Fusionsprotein, das aus dem extrazellulären Anteil des Activin-Rezeptors IIB besteht, der an die Fc-Domäne von humanem IgG1 angekoppelt wurde
• Humanisierter monoklonaler Antikörper gegen Myostatin
• Idebenon
• Kifunensin
• L-selenomethionine
• LMI 070
• Late Stage Human Motor Neutron Progenitors
• Losmapimod
• Metformin and L-citrulline
• Mexiletine hydrochloride
• 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
• Natriumphenylbutyrat
• Natriumvalproat
• Omigapil maleate
• Pamrevlumab
• Phosphorothioat-Oligonukleotid spezifisch für Exon 45
• Phosphorothioat-Oligonukleotid spezifisch für Exon 53
• Phosphorothioate-Oligonukleotid spezifisch für Exon 44
• Phosphorothioate-Oligonukleotid spezifisch für Exon 51
• Phosphorothioate-Oligonukleotid spezifisch für Exon 52
• R,S-O-(3-Piperidino-2-hydroxy-1-propyl) - Nikotinsäure Amidoxim Dihydrochlorid
• 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
• Rekombinante Thymidinphosphorylase eingekapselt in autologe Erythrozyten
• Rekombinante humane alpha-Glukosidase konjugiert mit mehreren Einheiten synthetischer bis-Mannose-6-phosphat-tetra-Mannose-Glykane
• Reldesemtiv
• Risdiplam
• Sialinsäure
• Synthetic antisense oligonucleotide directed against human dystrophin pre-mRNA
• Tamoxifen citrate
• Thymidine and deoxycytidine
• Trehalose
• Triheptanoin
• Valproic acid, sodium
• Viltolarsen
• Viraler Vektor mit für humanes SMN-Protein kodierender DNA
• Zinc gluconat
• deflazacort
• genetically modified adeno-associated viral vector serotype 9 expressing shRNA as well as a codon-optimised shRNA-insensitive wildtype PABPN1
• levocarnitin
• p38 mitogen-activated kinase inhibitor
• rimeporide
• tetracosactide

Handelsname(n)

• CARNITOR
• DANTRIUM
• EXONDYS 51
• Emflaza
• KEVEYIS
• LEVOCARNIL
• LUMIZYME
• MYOZYME
• NAMUSCLA
• RYANODEX
• SPINRAZA
• TRANSLARNA
• Vyondys 53
• ZOLGENSMA