Pharmacological management of spinal muscular atrophy
| dc.contributor.advisor | Pórszász, Róbert | |
| dc.contributor.advisordept | Debreceni Egyetem::Általános Orvostudományi Kar::Farmakológiai és Farmakoterápiai Intézet | |
| dc.contributor.author | Le, Ngo Minh Tran | |
| dc.contributor.department | DE--Általános Orvostudományi Kar | |
| dc.contributor.opponent | Szentmiklósi, József András | |
| dc.contributor.opponent | Drimba, László | |
| dc.contributor.opponentdept | Debreceni Egyetem::Általános Orvostudományi Kar::Farmakológiai és Farmakoterápiai Intézet | |
| dc.contributor.opponentdept | Debreceni Egyetem::Általános Orvostudományi Kar::Aneszteziológiai és Intenzív Terápiás Tanszék | |
| dc.date.accessioned | 2023-08-29T09:15:07Z | |
| dc.date.available | 2023-08-29T09:15:07Z | |
| dc.date.created | 2023-06-23 | |
| dc.description.abstract | Spinal muscular atrophy is an autosomal recessive inherited disease, which associate with SMN gene mutation. It presents with a wide range of symptoms from weakness, and fatigue of muscles to non-ambulatory, and death. Currently, there is no complete cure therapy. However, FDA has approved Nusinersen as a standard treatment. Additionally, with direct-to-genetic treatments, pharmacological management for spinal muscular atrophy has shown a promising future. | |
| dc.description.course | általános orvos | |
| dc.description.courselang | angol | |
| dc.description.degree | egységes, osztatlan | |
| dc.format.extent | 44 | |
| dc.identifier.uri | https://hdl.handle.net/2437/358540 | |
| dc.language.iso | en | |
| dc.subject | spinal muscular atrophy | |
| dc.subject | smn1 gene | |
| dc.subject | nusinersen | |
| dc.subject | zolgensma | |
| dc.subject.dspace | DEENK Témalista::Orvostudomány::Farmakológia | |
| dc.title | Pharmacological management of spinal muscular atrophy |