Buchumschlag

Short-term safety results from compassionate use of risdiplam in patients with spinal muscular atrophy in Germany

The oral, selective SMN2-splicing modifier risdiplam obtained European approval in March 2021 for the treatment of patients ≥ 2 months old with a clinical diagnosis of 5q-associated spinal muscular atrophy (SMA) 1/2/3 or with 1-4 SMN2 gene copies. For the preceding 12 months, this compassionate use...

Ausführliche Beschreibung

Gespeichert in:
Bibliographische Detailangaben
Hauptverfasser: Hahn, Andreas (VerfasserIn) , Thiemer, René (VerfasserIn) , Ludolph, Albert C. (VerfasserIn) , Schwartz, Oliver (VerfasserIn) , Trollmann, Regina (VerfasserIn) , Weydt, Patrick (VerfasserIn) , Weiler, Markus (VerfasserIn) , Mellert, Kathrin Gertrud (VerfasserIn) , Schwaderer, Martin Sebastian (VerfasserIn) , Hagenacker, Tim (VerfasserIn)
Dokumenttyp: Article (Journal)
Sprache:Englisch
Veröffentlicht: 19 July 2022
In: Orphanet journal of rare diseases
Year: 2022, Jahrgang: 17, Pages: 1-10
ISSN:1750-1172
DOI:10.1186/s13023-022-02420-8
Online-Zugang:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1186/s13023-022-02420-8
Volltext
Verfasserangaben:Andreas Hahn, René Günther, Albert Ludolph, Oliver Schwartz, Regina Trollmann, Patrick Weydt, Markus Weiler, Kathrin Neuland, Martin Sebastian Schwaderer, Tim Hagenacker and the Risdiplam Compassionate Use Program Group
Beschreibung
Zusammenfassung:The oral, selective SMN2-splicing modifier risdiplam obtained European approval in March 2021 for the treatment of patients ≥ 2 months old with a clinical diagnosis of 5q-associated spinal muscular atrophy (SMA) 1/2/3 or with 1-4 SMN2 gene copies. For the preceding 12 months, this compassionate use program (CUP) made risdiplam available to patients with SMA1/2 in Germany who could not receive any approved SMA therapy.
Beschreibung:Gesehen am 30.11.2022
Beschreibung:Online Resource
ISSN:1750-1172
DOI:10.1186/s13023-022-02420-8

Ähnliche Einträge