JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam
- Location
-
Deutsche Nationalbibliothek Frankfurt am Main
- Extent
-
1 Online-Ressource.
- Language
-
Englisch
- Bibliographic citation
-
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam ; day:11 ; month:5 ; year:2024 ; pages:1-14
Journal of neurology ; (11.5.2024), 1-14
- Creator
-
Chiriboga, Claudia A.
Bruno, Claudio
Duong, Tina
Fischer, Dirk
Mercuri, Eugenio
Kirschner, Janbernd
Kostera-Pruszczyk, Anna
Jaber, Birgit
Gorni, Ksenija
Kletzl, Heidemarie
Carruthers, Imogen
Martin, Carmen
Scalco, Renata S.
Fontoura, Paulo
Muntoni, Francesco
- Contributor
-
SpringerLink (Online service)
- DOI
-
10.1007/s00415-024-12318-z
- URN
-
urn:nbn:de:101:1-2407221025540.031058189330
- Rights
-
Open Access; Der Zugriff auf das Objekt ist unbeschränkt möglich.
- Last update
-
14.08.2025, 10:47 AM CEST
Data provider
This object is provided by:
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Deutsche Nationalbibliothek. If you have any questions about the object, please contact the data provider.
Associated
- Chiriboga, Claudia A.
- Bruno, Claudio
- Duong, Tina
- Fischer, Dirk
- Mercuri, Eugenio
- Kirschner, Janbernd
- Kostera-Pruszczyk, Anna
- Jaber, Birgit
- Gorni, Ksenija
- Kletzl, Heidemarie
- Carruthers, Imogen
- Martin, Carmen
- Scalco, Renata S.
- Fontoura, Paulo
- Muntoni, Francesco
- SpringerLink (Online service)
Other Objects (12)
Nusinersen - praktische Aspekte der Therapie bei Spinaler Muskelatrophie im klinischen Allltag
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam
Advances in treatment of spinal muscular atrophy – new phenotypes, new challenges, new implications for care
Die Hospizbewegung in Deutschland am Beispiel Recklinghausen
Diagnostik und Therapie der Spinalen Muskelatrophie
Untersuchung von altersabhängigen Veränderungen der paraspinalen Muskulatur mit monoklonalen Antikörpern gegen Myosin-heavy-chain Muskelfaserstrukturproteine mittels Immunfluoreszenz
Correction to: Risdiplam inpatients previously treated with other therapies for spinal muscular atrophy: an interim analysis from the JEWELFISH study
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
The TREAT‐NMD DMD global database: analysis of more than 7,000 duchenne muscular dystrophy mutations
Clinical outcomes in Duchenne Muscular Dystrophy: a study of 5345 patients from the TREAT-NMD DMD global database
Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study
Nusinersen - praktische Aspekte der Therapie bei Spinaler Muskelatrophie im klinischen Allltag
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam
Advances in treatment of spinal muscular atrophy – new phenotypes, new challenges, new implications for care
Die Hospizbewegung in Deutschland am Beispiel Recklinghausen
Diagnostik und Therapie der Spinalen Muskelatrophie
Untersuchung von altersabhängigen Veränderungen der paraspinalen Muskulatur mit monoklonalen Antikörpern gegen Myosin-heavy-chain Muskelfaserstrukturproteine mittels Immunfluoreszenz
Correction to: Risdiplam inpatients previously treated with other therapies for spinal muscular atrophy: an interim analysis from the JEWELFISH study
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
The TREAT‐NMD DMD global database: analysis of more than 7,000 duchenne muscular dystrophy mutations
Clinical outcomes in Duchenne Muscular Dystrophy: a study of 5345 patients from the TREAT-NMD DMD global database
Experiences of caregivers of children with spinal muscular atrophy participating in the expanded access program for nusinersen: a longitudinal qualitative study
Nusinersen - praktische Aspekte der Therapie bei Spinaler Muskelatrophie im klinischen Allltag
JEWELFISH: 24-month results from an open-label study in non-treatment-naïve patients with SMA receiving treatment with risdiplam
Advances in treatment of spinal muscular atrophy – new phenotypes, new challenges, new implications for care
Die Hospizbewegung in Deutschland am Beispiel Recklinghausen
Diagnostik und Therapie der Spinalen Muskelatrophie
Untersuchung von altersabhängigen Veränderungen der paraspinalen Muskulatur mit monoklonalen Antikörpern gegen Myosin-heavy-chain Muskelfaserstrukturproteine mittels Immunfluoreszenz
Correction to: Risdiplam inpatients previously treated with other therapies for spinal muscular atrophy: an interim analysis from the JEWELFISH study
Nusinersen initiated in infants during the presymptomatic stage of spinal muscular atrophy: Interim efficacy and safety results from the Phase 2 NURTURE study
A checklist for clinical trials in rare disease: obstacles and anticipatory actions-lessons learned from the FOR-DMD trial
The TREAT‐NMD DMD global database: analysis of more than 7,000 duchenne muscular dystrophy mutations
Clinical outcomes in Duchenne Muscular Dystrophy: a study of 5345 patients from the TREAT-NMD DMD global database