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: | , , , , , , , , , |
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| Format: | Article (Journal) |
| Language: | English |
| Published: |
19 July 2022
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| 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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| 520 | |a 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. | ||
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