Přehled nových a ještě novějších léků na Duchennovu svalovou dystrofii a spinální svalovou atrofii.
| Title in English | Overview of new and even newer drugs for Duchenne muscular dystrophy and spinal muscular atrophy |
|---|---|
| Authors | |
| Year of publication | 2019 |
| Type | Article in Periodical |
| Magazine / Source | Výsledky hledání Výsledky vyhledávání na webu Česko-slovenská pediatrie |
| MU Faculty or unit | |
| Citation | |
| Web | https://www.prolekare.cz/casopisy/cesko-slovenska-pediatrie/2019-3-6/prehled-novych-a-jeste-novejsich-leku-na-duchennovu-svalovou-dystrofii-a-spinalni-svalovou-atrofii-112987 |
| Keywords | Duchenne muscular dystrophy; spinal muscular atrophy; Spinraza; Translarna; gene therapy; exon skipping |
| Description | Duchenne muscular dystrophy (DMD and spinal muscular atrophy (SMA) are the most common neuromuscular disorders of childhood. Both of them are progressive and significantly affects the length and quality of life. There are currently more than 200 active clinical trials investigating new drugs for DMD patients and over 100 for patients with SMA. For both DMD and SMA patients several drugs have already been approved by FDA and EMA. This article focuses on selected therapies in the clinical phase of testing. |