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| Kat. Nr. | Arten | Produktbeschreibung | Struktur | Reinheit | Merkmal |
|---|---|---|---|---|---|
| SM1-H5145 | Human | Human SMN1 Protein, His Tag |  |
|
| Name | Research Code | Research Phase | Company | First Brand Name | First Approved Country | First Indication | First Approved Company | First Approved Date | Indications | Clinical Trials |
|---|---|---|---|---|---|---|---|---|---|---|
| Risdiplam | RG-7916; RO-7034067 | Approved | Ptc Therapeutics Inc | EVRYSDI, 艾满欣 | United States | Muscular Atrophy, Spinal | Genentech Inc | 2020-08-07 | Muscular Atrophy, Spinal | Details |
| Nusinersen sodium | BIIB-058; ISIS-396443 | Approved | Ionis Pharmaceuticals Inc, Biogen Inc | Spinraza | United States | Muscular Atrophy, Spinal | Biogen Idec | 2016-12-23 | Muscular Atrophy, Spinal | Details |
| Onasemnogene abeparvovec | rAAV-9; AVXS-101; scAAV-9.CB.SMN; OAV101 | Approved | Novartis Gene Therapies Inc, Novartis Pharma Ag | Zolgensma | United States | Muscular Atrophy, Spinal | Novartis Gene Therapies Inc | 2019-05-24 | Spinal Muscular Atrophies of Childhood; Muscular Atrophy, Spinal | Details |
| Risdiplam | RG-7916; RO-7034067 | Approved | Ptc Therapeutics Inc | EVRYSDI, 艾满欣 | United States | Muscular Atrophy, Spinal | Genentech Inc | 2020-08-07 | Muscular Atrophy, Spinal | Details |
| Nusinersen sodium | BIIB-058; ISIS-396443 | Approved | Ionis Pharmaceuticals Inc, Biogen Inc | Spinraza | United States | Muscular Atrophy, Spinal | Biogen Idec | 2016-12-23 | Muscular Atrophy, Spinal | Details |
| Onasemnogene abeparvovec | rAAV-9; AVXS-101; scAAV-9.CB.SMN; OAV101 | Approved | Novartis Gene Therapies Inc, Novartis Pharma Ag | Zolgensma | United States | Muscular Atrophy, Spinal | Novartis Gene Therapies Inc | 2019-05-24 | Spinal Muscular Atrophies of Childhood; Muscular Atrophy, Spinal | Details |
| Name | Research Code | Research Phase | Company | Indications | Clinical Trials |
|---|---|---|---|---|---|
| Branaplam | LMI-070; NVS-SM1 | Phase 2 Clinical | Novartis Pharma Ag | Huntington Disease; Muscular Atrophy, Spinal | Details |
| vesemnogene lantuparvovec (Lantu Biopharma) | AAV-hSMN1 | Phase 2 Clinical | Lantu Biopharma Inc | Muscular Atrophy, Spinal | Details |
| EXG001-307 | EXG001-307 | Phase 2 Clinical | Hangzhou Jiayin Biotechnology Co Ltd | Muscular Atrophy, Spinal | Details |
| SMN-C3 | SMN-C3 | Phase 1 Clinical | Ptc Therapeutics Inc, F. Hoffmann-La Roche Ltd | Muscular Atrophy, Spinal | Details |
| SKG-0201 | SKG0201; SKG-0201 | Phase 1 Clinical | Skyline Therapeutics (Hong Kong) Ltd | Spinal Muscular Atrophies of Childhood; Muscular Atrophy, Spinal | Details |
| BIIB-115 | BIIB-115; ION-306 | Phase 1 Clinical | Ionis Pharmaceuticals Inc | Muscular Atrophy, Spinal | Details |
| Branaplam | LMI-070; NVS-SM1 | Phase 2 Clinical | Novartis Pharma Ag | Huntington Disease; Muscular Atrophy, Spinal | Details |
| vesemnogene lantuparvovec (Lantu Biopharma) | AAV-hSMN1 | Phase 2 Clinical | Lantu Biopharma Inc | Muscular Atrophy, Spinal | Details |
| EXG001-307 | EXG001-307 | Phase 2 Clinical | Hangzhou Jiayin Biotechnology Co Ltd | Muscular Atrophy, Spinal | Details |
| SMN-C3 | SMN-C3 | Phase 1 Clinical | Ptc Therapeutics Inc, F. Hoffmann-La Roche Ltd | Muscular Atrophy, Spinal | Details |
| SKG-0201 | SKG0201; SKG-0201 | Phase 1 Clinical | Skyline Therapeutics (Hong Kong) Ltd | Spinal Muscular Atrophies of Childhood; Muscular Atrophy, Spinal | Details |
| BIIB-115 | BIIB-115; ION-306 | Phase 1 Clinical | Ionis Pharmaceuticals Inc | Muscular Atrophy, Spinal | Details |
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