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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...

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Main Authors: Hahn, Andreas (Author) , Thiemer, René (Author) , Ludolph, Albert C. (Author) , Schwartz, Oliver (Author) , Trollmann, Regina (Author) , Weydt, Patrick (Author) , Weiler, Markus (Author) , Mellert, Kathrin Gertrud (Author) , Schwaderer, Martin Sebastian (Author) , Hagenacker, Tim (Author)
Format: Article (Journal)
Language:English
Published: 19 July 2022
In: Orphanet journal of rare diseases
Year: 2022, Volume: 17, Pages: 1-10
ISSN:1750-1172
DOI:10.1186/s13023-022-02420-8
Online Access:Verlag, lizenzpflichtig, Volltext: https://doi.org/10.1186/s13023-022-02420-8
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Author Notes: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
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Summary: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.
Item Description:Gesehen am 30.11.2022
Physical Description:Online Resource
ISSN:1750-1172
DOI:10.1186/s13023-022-02420-8

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